Difference between revisions of "Immune thrombocytopenia"

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=Guidelines=
 
=Guidelines=
 
==ASH==
 
==ASH==
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===Older===
 
===Older===
 
*'''2011:''' Neunert et al. [http://www.bloodjournal.org/content/117/16/4190 The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia]
 
*'''2011:''' Neunert et al. [http://www.bloodjournal.org/content/117/16/4190 The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia]
 
 
==BSH==
 
==BSH==
 
*'''2019:''' Hill et al. [https://doi.org/10.1111/bjh.15735 The prevention of glucocorticoid-induced osteoporosis in patients with immune thrombocytopenia receiving steroids: a British Society for Haematology Good Practice Paper]
 
*'''2019:''' Hill et al. [https://doi.org/10.1111/bjh.15735 The prevention of glucocorticoid-induced osteoporosis in patients with immune thrombocytopenia receiving steroids: a British Society for Haematology Good Practice Paper]
 
 
=="How I Treat"==
 
=="How I Treat"==
 
*'''2021:''' Ghanima et al. [https://doi.org/10.1182/blood.2021010968 How I treat primary ITP in adult patients who are unresponsive to or dependent on corticosteroid treatment]
 
*'''2021:''' Ghanima et al. [https://doi.org/10.1182/blood.2021010968 How I treat primary ITP in adult patients who are unresponsive to or dependent on corticosteroid treatment]
 
 
=Initial therapy=
 
=Initial therapy=
 
 
==Dexamethasone monotherapy {{#subobject:7c8c62|Regimen=1}}==
 
==Dexamethasone monotherapy {{#subobject:7c8c62|Regimen=1}}==
 
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<div class="toccolours" style="background-color:#eeeeee">
 
 
 
===Regimen variant #1 {{#subobject:666055|Variant=1}}===
 
===Regimen variant #1 {{#subobject:666055|Variant=1}}===
 
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|-
 
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<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Dexamethasone (Decadron)]] 40 mg/day PO on days 1 to 4
 
*[[Dexamethasone (Decadron)]] 40 mg/day PO on days 1 to 4
 
 
'''4-day course'''
 
'''4-day course'''
 +
</div>
 +
<div class="toccolours" style="background-color:#cbd5e7">
 
====Subsequent treatment====
 
====Subsequent treatment====
 
*If platelets remained below 30 x 10<sup>9</sup>/L or bleeding by day 10, course is repeated once
 
*If platelets remained below 30 x 10<sup>9</sup>/L or bleeding by day 10, course is repeated once
 
+
</div></div><br>
 +
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen variant #2 {{#subobject:678fa4|Variant=1}}===
 
===Regimen variant #2 {{#subobject:678fa4|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
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|-
 
|-
 
|}
 
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<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy, part 1====
 
====Immunosuppressive therapy, part 1====
 
*[[Prednisone (Sterapred)]] 1 mg/kg/day PO for 7 days
 
*[[Prednisone (Sterapred)]] 1 mg/kg/day PO for 7 days
 
 
====Immunosuppressive therapy, part 2====
 
====Immunosuppressive therapy, part 2====
 
*[[Dexamethasone (Decadron)]] 0.6 mg/kg/day (route not specified) for days 1 to 4
 
*[[Dexamethasone (Decadron)]] 0.6 mg/kg/day (route not specified) for days 1 to 4
 
 
'''21-day cycle for 6 cycles'''
 
'''21-day cycle for 6 cycles'''
 
+
</div></div><br>
 +
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen variant #3 {{#subobject:19f4e9|Variant=1}}===
 
===Regimen variant #3 {{#subobject:19f4e9|Variant=1}}===
 
{| class="wikitable" style="width: 40%; text-align:center;"  
 
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|-
 
|-
 
|}
 
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<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Dexamethasone (Decadron)]] 40 mg PO once per day on days 1 to 4
 
*[[Dexamethasone (Decadron)]] 40 mg PO once per day on days 1 to 4
 
 
'''4-day course'''
 
'''4-day course'''
 
 
''Patients who had an initial response, but whose platelets dropped below 30 x 10<sup>9</sup>/L within 6 months received:''
 
''Patients who had an initial response, but whose platelets dropped below 30 x 10<sup>9</sup>/L within 6 months received:''
 
 
*[[Dexamethasone (Decadron)]] 40 mg PO once per day on days 1 to 4
 
*[[Dexamethasone (Decadron)]] 40 mg PO once per day on days 1 to 4
 
*[[Prednisone (Sterapred)]] 15 mg PO once per day, starting on day 5, "with gradual tapering"
 
*[[Prednisone (Sterapred)]] 15 mg PO once per day, starting on day 5, "with gradual tapering"
 
+
</div></div><br>
 +
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen variant #4, monthly dexamethasone {{#subobject:f892de|Variant=1}}===
 
===Regimen variant #4, monthly dexamethasone {{#subobject:f892de|Variant=1}}===
 
{| class="wikitable" style="width: 40%; text-align:center;"  
 
{| class="wikitable" style="width: 40%; text-align:center;"  
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|-
 
|-
 
|}
 
|}
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<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Dexamethasone (Decadron)]] 40 mg PO once per day on days 1 to 4
 
*[[Dexamethasone (Decadron)]] 40 mg PO once per day on days 1 to 4
 
*Patients who had platelet counts of less than or equal to 20 x 10<sup>9</sup>/L between cycles received [[Prednisone (Sterapred)]] 0.25 mg/kg PO once per day "between courses"
 
*Patients who had platelet counts of less than or equal to 20 x 10<sup>9</sup>/L between cycles received [[Prednisone (Sterapred)]] 0.25 mg/kg PO once per day "between courses"
 
 
'''28-day cycle for 6 cycles'''
 
'''28-day cycle for 6 cycles'''
 
+
</div></div><br>
 +
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen variant #5, bi-weekly dexamethasone {{#subobject:8ac5fe|Variant=1}}===
 
===Regimen variant #5, bi-weekly dexamethasone {{#subobject:8ac5fe|Variant=1}}===
 
{| class="wikitable" style="width: 40%; text-align:center;"  
 
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|-
 
|-
 
|}
 
|}
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<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Dexamethasone (Decadron)]] by the following criteria:
 
*[[Dexamethasone (Decadron)]] by the following criteria:
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**Patients less than 15 years old: 20 mg/m<sup>2</sup> (maximum dose of 40 mg) IV or PO once per day on days 1 to 4
 
**Patients less than 15 years old: 20 mg/m<sup>2</sup> (maximum dose of 40 mg) IV or PO once per day on days 1 to 4
 
**Patients who had platelet counts of less than or equal to 30 x 10<sup>9</sup>/L between cycles and/or who had bleeding related to thrombocytopenia received 0.035 mg/kg PO once per day "between courses"
 
**Patients who had platelet counts of less than or equal to 30 x 10<sup>9</sup>/L between cycles and/or who had bleeding related to thrombocytopenia received 0.035 mg/kg PO once per day "between courses"
 
 
'''14-day cycle for 4 cycles'''
 
'''14-day cycle for 4 cycles'''
 
+
</div></div>
 
===References===
 
===References===
 
# Cheng Y, Wong RS, Soo YO, Chui CH, Lau FY, Chan NP, Wong WS, Cheng G. Initial treatment of immune thrombocytopenic purpura with high-dose dexamethasone. N Engl J Med. 2003 Aug 28;349(9):831-6. [https://doi.org/10.1056/NEJMoa030254 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/12944568 PubMed] content property of [http://hemonc.org HemOnc.org]
 
# Cheng Y, Wong RS, Soo YO, Chui CH, Lau FY, Chan NP, Wong WS, Cheng G. Initial treatment of immune thrombocytopenic purpura with high-dose dexamethasone. N Engl J Med. 2003 Aug 28;349(9):831-6. [https://doi.org/10.1056/NEJMoa030254 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/12944568 PubMed] content property of [http://hemonc.org HemOnc.org]
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# '''Abstract:''' Johannes Matschke, Hannes Müller-Beißenhirtz, Ilona Vester, Bernd Hertenstein, Lewin Eisele, Hildegard Lax, Claudia Ose, Ulrich Dührsen. A Randomized Trial Of Daily Prednisone Versus Pulsed Dexamethasone In Treatment Naïve Patients With Idiopathic Thrombocytopenic Purpura. Blood Nov 2013,122(21)325. [http://www.bloodjournal.org/content/122/21/325 link to abstract]
 
# '''Abstract:''' Johannes Matschke, Hannes Müller-Beißenhirtz, Ilona Vester, Bernd Hertenstein, Lewin Eisele, Hildegard Lax, Claudia Ose, Ulrich Dührsen. A Randomized Trial Of Daily Prednisone Versus Pulsed Dexamethasone In Treatment Naïve Patients With Idiopathic Thrombocytopenic Purpura. Blood Nov 2013,122(21)325. [http://www.bloodjournal.org/content/122/21/325 link to abstract]
 
# Wei Y, Ji XB, Wang YW, Wang JX, Yang EQ, Wang ZC, Sang YQ, Bi ZM, Ren CA, Zhou F, Liu GQ, Peng J, Hou M. High-dose dexamethasone vs prednisone for treatment of adult immune thrombocytopenia: a prospective multicenter randomized trial. Blood. 2016 Jan 21;127(3):296-302. Epub 2015 Oct 19. [http://www.bloodjournal.org/content/127/3/296.long link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/26480931 PubMed]
 
# Wei Y, Ji XB, Wang YW, Wang JX, Yang EQ, Wang ZC, Sang YQ, Bi ZM, Ren CA, Zhou F, Liu GQ, Peng J, Hou M. High-dose dexamethasone vs prednisone for treatment of adult immune thrombocytopenia: a prospective multicenter randomized trial. Blood. 2016 Jan 21;127(3):296-302. Epub 2015 Oct 19. [http://www.bloodjournal.org/content/127/3/296.long link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/26480931 PubMed]
 
 
==Dexamethasone & Eltrombopag {{#subobject:910687|Regimen=1}}==
 
==Dexamethasone & Eltrombopag {{#subobject:910687|Regimen=1}}==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
 
 
===Regimen {{#subobject:470504|Variant=1}}===
 
===Regimen {{#subobject:470504|Variant=1}}===
 
{| class="wikitable" style="width: 40%; text-align:center;"  
 
{| class="wikitable" style="width: 40%; text-align:center;"  
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|-
 
|-
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Dexamethasone (Decadron)]] 40 mg/day PO on days 1 to 4
 
*[[Dexamethasone (Decadron)]] 40 mg/day PO on days 1 to 4
 
 
====Growth factor therapy====
 
====Growth factor therapy====
 
*[[Eltrombopag (Promacta)]] 50 mg PO once per day on days 5 to 33
 
*[[Eltrombopag (Promacta)]] 50 mg PO once per day on days 5 to 33
 
 
'''5-week course'''
 
'''5-week course'''
 
+
</div></div>
 
===References===
 
===References===
 
<!-- Presented in an abstract form at the 55th meeting of the American Society of Hematology, New Orleans, LA, December 2013. -->
 
<!-- Presented in an abstract form at the 55th meeting of the American Society of Hematology, New Orleans, LA, December 2013. -->
 
# Gómez-Almaguer D, Herrera-Rojas MA, Jaime-Pérez JC, Gómez-De León A, Cantú-Rodríguez OG, Gutiérrez-Aguirre CH, Tarín-Arzaga L, Hernández-Reyes J, Ruiz-Arguelles GJ. Eltrombopag and high-dose dexamethasone as frontline treatment of newly diagnosed immune thrombocytopenia in adults. Blood. 2014 Jun 19;123(25):3906-8. Epub 2014 May 6. [http://www.bloodjournal.org/content/123/25/3906.long link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/24802773 PubMed]
 
# Gómez-Almaguer D, Herrera-Rojas MA, Jaime-Pérez JC, Gómez-De León A, Cantú-Rodríguez OG, Gutiérrez-Aguirre CH, Tarín-Arzaga L, Hernández-Reyes J, Ruiz-Arguelles GJ. Eltrombopag and high-dose dexamethasone as frontline treatment of newly diagnosed immune thrombocytopenia in adults. Blood. 2014 Jun 19;123(25):3906-8. Epub 2014 May 6. [http://www.bloodjournal.org/content/123/25/3906.long link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/24802773 PubMed]
 
 
==Dexamethasone & Mycophenolate mofetil {{#subobject:5f56yh|Regimen=1}}==
 
==Dexamethasone & Mycophenolate mofetil {{#subobject:5f56yh|Regimen=1}}==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
 
 
===Regimen {{#subobject:5yh112|Variant=1}}===
 
===Regimen {{#subobject:5yh112|Variant=1}}===
 
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|-
 
|-
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Dexamethasone (Decadron)]]
 
*[[Dexamethasone (Decadron)]]
 
*[[Mycophenolate mofetil (CellCept)]]
 
*[[Mycophenolate mofetil (CellCept)]]
 +
</div></div>
 
===References===
 
===References===
 
#'''FLIGHT:''' Bradbury CA, Pell J, Hill Q, Bagot C, Cooper N, Ingram J, Breheny K, Kandiyali R, Rayment R, Evans G, Talks K, Thomas I, Greenwood R. Mycophenolate Mofetil for First-Line Treatment of Immune Thrombocytopenia. N Engl J Med. 2021 Sep 2;385(10):885-895. [https://doi.org/10.1056/nejmoa2100596 link to original article] [https://pubmed.ncbi.nlm.nih.gov/34469646/ PubMed] NCT03156452
 
#'''FLIGHT:''' Bradbury CA, Pell J, Hill Q, Bagot C, Cooper N, Ingram J, Breheny K, Kandiyali R, Rayment R, Evans G, Talks K, Thomas I, Greenwood R. Mycophenolate Mofetil for First-Line Treatment of Immune Thrombocytopenia. N Engl J Med. 2021 Sep 2;385(10):885-895. [https://doi.org/10.1056/nejmoa2100596 link to original article] [https://pubmed.ncbi.nlm.nih.gov/34469646/ PubMed] NCT03156452
 
 
==Dexamethasone & Rituximab {{#subobject:b9a6ee|Regimen=1}}==
 
==Dexamethasone & Rituximab {{#subobject:b9a6ee|Regimen=1}}==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen ("R+3Dex") {{#subobject:d05adc|Variant=1}}===
 
===Regimen ("R+3Dex") {{#subobject:d05adc|Variant=1}}===
 
{| class="wikitable" style="width: 40%; text-align:center;"  
 
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|-
 
|-
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Dexamethasone (Decadron)]] 28 mg/m<sup>2</sup>/day (maximum dose of 40 mg) on days 1 to 4
 
*[[Dexamethasone (Decadron)]] 28 mg/m<sup>2</sup>/day (maximum dose of 40 mg) on days 1 to 4
 
*[[Rituximab (Rituxan)]] as follows:
 
*[[Rituximab (Rituxan)]] as follows:
 
**Cycles 1 & 2: 375 mg/m<sup>2</sup> IV once per day on days 1 & 8
 
**Cycles 1 & 2: 375 mg/m<sup>2</sup> IV once per day on days 1 & 8
 
 
'''14-day cycle for 3 cycles'''
 
'''14-day cycle for 3 cycles'''
 +
</div></div>
 
===References===
 
===References===
 
# '''Abstract: Retrospective:''' Allison Imahiyerobo, Micha Thompson, Marina Izak Karaev, Waleed Ghanima, James B Bussel. Rituximab Combined With Three Cycles Of High Dose Dexamethasone Provides a Long Term Response Rate Similar To That Of Splenectomy In Patients With Immune Thrombocytopenia (ITP) Of Duration Less Than 2 Years. Blood Nov 2013,122(21)2310. [http://www.bloodjournal.org/content/122/21/2310 link to abstract]
 
# '''Abstract: Retrospective:''' Allison Imahiyerobo, Micha Thompson, Marina Izak Karaev, Waleed Ghanima, James B Bussel. Rituximab Combined With Three Cycles Of High Dose Dexamethasone Provides a Long Term Response Rate Similar To That Of Splenectomy In Patients With Immune Thrombocytopenia (ITP) Of Duration Less Than 2 Years. Blood Nov 2013,122(21)2310. [http://www.bloodjournal.org/content/122/21/2310 link to abstract]
 
 
==Intravenous immunoglobulin monotherapy {{#subobject:ea894c|Regimen=1}}==
 
==Intravenous immunoglobulin monotherapy {{#subobject:ea894c|Regimen=1}}==
 
 
IVIG: '''<u>I</u>'''ntra'''<u>V</u>'''enous '''<u>I</u>'''mmuno'''<u>G</u>'''lobulin
 
IVIG: '''<u>I</u>'''ntra'''<u>V</u>'''enous '''<u>I</u>'''mmuno'''<u>G</u>'''lobulin
 +
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen {{#subobject:9134b2|Variant=1}}===
 
===Regimen {{#subobject:9134b2|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
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| style="background-color:#1a9850" |Superior response rate
 
| style="background-color:#1a9850" |Superior response rate
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Supportive therapy====
 
====Supportive therapy====
 
*[[Intravenous immunoglobulin (IVIG)]] 1000 mg/kg IV once on day 1
 
*[[Intravenous immunoglobulin (IVIG)]] 1000 mg/kg IV once on day 1
 
 
'''One dose'''
 
'''One dose'''
 
+
</div></div>
 
===References===
 
===References===
 
# Imbach P, Wagner HP, Berchtold W, Gaedicke G, Hirt A, Joller P, Mueller-Eckhardt C, Müller B, Rossi E, Barandun S. Intravenous immunoglobulin versus oral corticosteroids in acute immune thrombocytopenic purpura in childhood. Lancet. 1985 Aug 31;2(8453):464-8. [https://doi.org/10.1016/s0140-6736(85)90400-3 link to original article] [https://pubmed.ncbi.nlm.nih.gov/2863492 PubMed]
 
# Imbach P, Wagner HP, Berchtold W, Gaedicke G, Hirt A, Joller P, Mueller-Eckhardt C, Müller B, Rossi E, Barandun S. Intravenous immunoglobulin versus oral corticosteroids in acute immune thrombocytopenic purpura in childhood. Lancet. 1985 Aug 31;2(8453):464-8. [https://doi.org/10.1016/s0140-6736(85)90400-3 link to original article] [https://pubmed.ncbi.nlm.nih.gov/2863492 PubMed]
Line 219: Line 212:
 
# Godeau B, Chevret S, Varet B, Lefrère F, Zini JM, Bassompierre F, Chèze S, Legouffe E, Hulin C, Grange MJ, Fain O, Bierling P; French ATIP Study Group. Intravenous immunoglobulin or high-dose methylprednisolone, with or without oral prednisone, for adults with untreated severe autoimmune thrombocytopenic purpura: a randomised, multicentre trial. Lancet. 2002 Jan 5;359(9300):23-9. [https://doi.org/10.1016/S0140-6736(02)07275-6 link to original article] [https://pubmed.ncbi.nlm.nih.gov/11809183 PubMed]
 
# Godeau B, Chevret S, Varet B, Lefrère F, Zini JM, Bassompierre F, Chèze S, Legouffe E, Hulin C, Grange MJ, Fain O, Bierling P; French ATIP Study Group. Intravenous immunoglobulin or high-dose methylprednisolone, with or without oral prednisone, for adults with untreated severe autoimmune thrombocytopenic purpura: a randomised, multicentre trial. Lancet. 2002 Jan 5;359(9300):23-9. [https://doi.org/10.1016/S0140-6736(02)07275-6 link to original article] [https://pubmed.ncbi.nlm.nih.gov/11809183 PubMed]
 
# '''TIKI:''' Heitink-Pollé KMJ, Uiterwaal CSPM, Porcelijn L, Tamminga RYJ, Smiers FJ, van Woerden NL, Wesseling J, Vidarsson G, Laarhoven AG, de Haas M, Bruin MCA; TIKI Investigators. Intravenous immunoglobulin vs observation in childhood immune thrombocytopenia: a randomized controlled trial. Blood. 2018 Aug 30;132(9):883-891. Epub 2018 Jun 26. [http://www.bloodjournal.org/content/132/9/883.long link to original article] [https://pubmed.ncbi.nlm.nih.gov/29945954 PubMed]
 
# '''TIKI:''' Heitink-Pollé KMJ, Uiterwaal CSPM, Porcelijn L, Tamminga RYJ, Smiers FJ, van Woerden NL, Wesseling J, Vidarsson G, Laarhoven AG, de Haas M, Bruin MCA; TIKI Investigators. Intravenous immunoglobulin vs observation in childhood immune thrombocytopenia: a randomized controlled trial. Blood. 2018 Aug 30;132(9):883-891. Epub 2018 Jun 26. [http://www.bloodjournal.org/content/132/9/883.long link to original article] [https://pubmed.ncbi.nlm.nih.gov/29945954 PubMed]
 
 
==Mycophenolate mofetil & Prednisolone {{#subobject:5f5dab|Regimen=1}}==
 
==Mycophenolate mofetil & Prednisolone {{#subobject:5f5dab|Regimen=1}}==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
 
 
===Regimen {{#subobject:dacj12|Variant=1}}===
 
===Regimen {{#subobject:dacj12|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
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|-
 
|-
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Mycophenolate mofetil (CellCept)]]
 
*[[Mycophenolate mofetil (CellCept)]]
 
*[[Prednisolone (Millipred)]]
 
*[[Prednisolone (Millipred)]]
 +
</div></div>
 
===References===
 
===References===
 
#'''FLIGHT:''' Bradbury CA, Pell J, Hill Q, Bagot C, Cooper N, Ingram J, Breheny K, Kandiyali R, Rayment R, Evans G, Talks K, Thomas I, Greenwood R. Mycophenolate Mofetil for First-Line Treatment of Immune Thrombocytopenia. N Engl J Med. 2021 Sep 2;385(10):885-895. [https://doi.org/10.1056/nejmoa2100596 link to original article] [https://pubmed.ncbi.nlm.nih.gov/34469646/ PubMed] NCT03156452
 
#'''FLIGHT:''' Bradbury CA, Pell J, Hill Q, Bagot C, Cooper N, Ingram J, Breheny K, Kandiyali R, Rayment R, Evans G, Talks K, Thomas I, Greenwood R. Mycophenolate Mofetil for First-Line Treatment of Immune Thrombocytopenia. N Engl J Med. 2021 Sep 2;385(10):885-895. [https://doi.org/10.1056/nejmoa2100596 link to original article] [https://pubmed.ncbi.nlm.nih.gov/34469646/ PubMed] NCT03156452
 
 
==Prednisone monotherapy {{#subobject:5f27f2|Regimen=1}}==
 
==Prednisone monotherapy {{#subobject:5f27f2|Regimen=1}}==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
 
 
===Regimen variant #1 {{#subobject:d3c7eb|Variant=1}}===
 
===Regimen variant #1 {{#subobject:d3c7eb|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
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|-
 
|-
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Prednisone (Sterapred)]] as follows:
 
*[[Prednisone (Sterapred)]] as follows:
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**Day 29 onwards: tapered over 4 to 6 weeks with a goal of maintenance dosing less than 15 mg/day or "complete termination"
 
**Day 29 onwards: tapered over 4 to 6 weeks with a goal of maintenance dosing less than 15 mg/day or "complete termination"
 
***Taper schedule determined by treating physician
 
***Taper schedule determined by treating physician
 
+
</div></div><br>
 +
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen variant #2 {{#subobject:7c003e|Variant=1}}===
 
===Regimen variant #2 {{#subobject:7c003e|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
Line 277: Line 270:
 
|-
 
|-
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Prednisone (Sterapred)]] as follows:
 
*[[Prednisone (Sterapred)]] as follows:
 
**Weeks 1 to 2: 1 mg/kg/day PO
 
**Weeks 1 to 2: 1 mg/kg/day PO
 
**Weeks 3 to 19: Tapered over 19 weeks with a goal of maintenance dosing less than 7.5 mg/day at the end of week 19
 
**Weeks 3 to 19: Tapered over 19 weeks with a goal of maintenance dosing less than 7.5 mg/day at the end of week 19
 
+
</div></div>
 
===References===
 
===References===
 
# Blanchette VS, Luke B, Andrew M, Sommerville-Nielsen S, Barnard D, de Veber B, Gent M. A prospective, randomized trial of high-dose intravenous immune globulin G therapy, oral prednisone therapy, and no therapy in childhood acute immune thrombocytopenic purpura. J Pediatr. 1993 Dec;123(6):989-95. [https://doi.org/10.1016/s0022-3476(05)80400-7 link to original article] [https://pubmed.ncbi.nlm.nih.gov/8229536 PubMed]
 
# Blanchette VS, Luke B, Andrew M, Sommerville-Nielsen S, Barnard D, de Veber B, Gent M. A prospective, randomized trial of high-dose intravenous immune globulin G therapy, oral prednisone therapy, and no therapy in childhood acute immune thrombocytopenic purpura. J Pediatr. 1993 Dec;123(6):989-95. [https://doi.org/10.1016/s0022-3476(05)80400-7 link to original article] [https://pubmed.ncbi.nlm.nih.gov/8229536 PubMed]
Line 287: Line 281:
 
# '''Abstract:''' Johannes Matschke, Hannes Müller-Beißenhirtz, Ilona Vester, Bernd Hertenstein, Lewin Eisele, Hildegard Lax, Claudia Ose, Ulrich Dührsen. A Randomized Trial Of Daily Prednisone Versus Pulsed Dexamethasone In Treatment Naïve Patients With Idiopathic Thrombocytopenic Purpura. Blood Nov 2013,122(21)325. [http://www.bloodjournal.org/content/122/21/325 link to abstract]
 
# '''Abstract:''' Johannes Matschke, Hannes Müller-Beißenhirtz, Ilona Vester, Bernd Hertenstein, Lewin Eisele, Hildegard Lax, Claudia Ose, Ulrich Dührsen. A Randomized Trial Of Daily Prednisone Versus Pulsed Dexamethasone In Treatment Naïve Patients With Idiopathic Thrombocytopenic Purpura. Blood Nov 2013,122(21)325. [http://www.bloodjournal.org/content/122/21/325 link to abstract]
 
# Wei Y, Ji XB, Wang YW, Wang JX, Yang EQ, Wang ZC, Sang YQ, Bi ZM, Ren CA, Zhou F, Liu GQ, Peng J, Hou M. High-dose dexamethasone vs prednisone for treatment of adult immune thrombocytopenia: a prospective multicenter randomized trial. Blood. 2016 Jan 21;127(3):296-302. Epub 2015 Oct 19. [http://www.bloodjournal.org/content/127/3/296.long link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/26480931 PubMed]
 
# Wei Y, Ji XB, Wang YW, Wang JX, Yang EQ, Wang ZC, Sang YQ, Bi ZM, Ren CA, Zhou F, Liu GQ, Peng J, Hou M. High-dose dexamethasone vs prednisone for treatment of adult immune thrombocytopenia: a prospective multicenter randomized trial. Blood. 2016 Jan 21;127(3):296-302. Epub 2015 Oct 19. [http://www.bloodjournal.org/content/127/3/296.long link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/26480931 PubMed]
 
 
==RhIG monotherapy {{#subobject:3ab9b6|Regimen=1}}==
 
==RhIG monotherapy {{#subobject:3ab9b6|Regimen=1}}==
 
 
RhIG: '''<u>Rh</u>'''o(D) '''<u>I</u>'''mmune '''<u>G</u>'''lobulin
 
RhIG: '''<u>Rh</u>'''o(D) '''<u>I</u>'''mmune '''<u>G</u>'''lobulin
 +
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen variant #1, 25 mcg/kg {{#subobject:8335a6|Variant=1}}===
 
===Regimen variant #1, 25 mcg/kg {{#subobject:8335a6|Variant=1}}===
 
{| class="wikitable" style="width: 40%; text-align:center;"  
 
{| class="wikitable" style="width: 40%; text-align:center;"  
Line 300: Line 293:
 
|-
 
|-
 
|}
 
|}
''Eligibility: RhD-positive.''
+
<div class="toccolours" style="background-color:#fdcdac">
 +
====Eligibility criteria====
 +
*RhD-positive
 +
</div>
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Supportive therapy====
 
====Supportive therapy====
 
*[[Rho(D) immune globulin (RhoGAM)]] 25 mcg/kg IV once on day 1, repeated as needed on days 3 & 4
 
*[[Rho(D) immune globulin (RhoGAM)]] 25 mcg/kg IV once on day 1, repeated as needed on days 3 & 4
 
 
'''4-day course'''
 
'''4-day course'''
 
+
</div></div><br>
 +
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen variant #2, 50 mcg/kg {{#subobject:487067|Variant=1}}===
 
===Regimen variant #2, 50 mcg/kg {{#subobject:487067|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
Line 319: Line 316:
 
|-
 
|-
 
|}
 
|}
''Eligibility: RhD-positive, adult, HIV-negative, non-splenectomized, platelet count less than or equal to 30 x 10<sup>9</sup>/L.''
+
<div class="toccolours" style="background-color:#fdcdac">
 +
====Eligibility criteria====
 +
*RhD-positive, adult, HIV-negative, non-splenectomized, platelet count less than or equal to 30 x 10<sup>9</sup>/L
 +
</div>
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Supportive therapy====
 
====Supportive therapy====
 
*[[Rho(D) immune globulin (RhoGAM)]] 50 mcg/kg IV once on day 1
 
*[[Rho(D) immune globulin (RhoGAM)]] 50 mcg/kg IV once on day 1
 
 
====Supportive therapy====
 
====Supportive therapy====
 
*[[Acetaminophen (Tylenol)]] 650 mg PO once on day 1, prior to therapy
 
*[[Acetaminophen (Tylenol)]] 650 mg PO once on day 1, prior to therapy
 
*[[Prednisone (Sterapred)]] 20 mg PO once on day 1, prior to therapy
 
*[[Prednisone (Sterapred)]] 20 mg PO once on day 1, prior to therapy
 
 
'''One dose; can be repeated if required to increase platelet count'''
 
'''One dose; can be repeated if required to increase platelet count'''
 
+
</div></div><br>
 +
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen variant #3, 75 mcg/kg {{#subobject:b30788|Variant=1}}===
 
===Regimen variant #3, 75 mcg/kg {{#subobject:b30788|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
Line 342: Line 342:
 
|-
 
|-
 
|}
 
|}
''Eligibility: RhD-positive, adult, HIV-negative, non-splenectomized, platelet count less than or equal to 30 x 10<sup>9</sup>/L.''
+
<div class="toccolours" style="background-color:#fdcdac">
 +
====Eligibility criteria====
 +
*RhD-positive, adult, HIV-negative, non-splenectomized, platelet count less than or equal to 30 x 10<sup>9</sup>/L
 +
</div>
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Supportive therapy====
 
====Supportive therapy====
 
*[[Rho(D) immune globulin (RhoGAM)]] 75 mcg/kg IV once on day 1
 
*[[Rho(D) immune globulin (RhoGAM)]] 75 mcg/kg IV once on day 1
 
 
====Supportive therapy====
 
====Supportive therapy====
 
*[[Acetaminophen (Tylenol)]] 650 mg PO once on day 1, prior to therapy
 
*[[Acetaminophen (Tylenol)]] 650 mg PO once on day 1, prior to therapy
 
*[[Prednisone (Sterapred)]] 20 mg PO once on day 1, prior to therapy
 
*[[Prednisone (Sterapred)]] 20 mg PO once on day 1, prior to therapy
 
 
'''One dose; can be repeated if required to increase platelet count'''
 
'''One dose; can be repeated if required to increase platelet count'''
 
+
</div></div>
 
===References===
 
===References===
 
# Bussel JB, Graziano JN, Kimberly RP, Pahwa S, Aledort LM. Intravenous anti-D treatment of immune thrombocytopenic purpura: analysis of efficacy, toxicity, and mechanism of effect. Blood. 1991 May 1;77(9):1884-93. [http://www.bloodjournal.org/content/77/9/1884 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/1850307 PubMed]  
 
# Bussel JB, Graziano JN, Kimberly RP, Pahwa S, Aledort LM. Intravenous anti-D treatment of immune thrombocytopenic purpura: analysis of efficacy, toxicity, and mechanism of effect. Blood. 1991 May 1;77(9):1884-93. [http://www.bloodjournal.org/content/77/9/1884 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/1850307 PubMed]  
 
# Newman GC, Novoa MV, Fodero EM, Lesser ML, Woloski BM, Bussel JB. A dose of 75 microg/kg/day of iv anti-D increases the platelet count more rapidly and for a longer period of time than 50 microg/kg/day in adults with immune thrombocytopenic purpura. Br J Haematol. 2001 Mar;112(4):1076-8. [https://doi.org/10.1046/j.1365-2141.2001.02627.x link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/11298610 PubMed]
 
# Newman GC, Novoa MV, Fodero EM, Lesser ML, Woloski BM, Bussel JB. A dose of 75 microg/kg/day of iv anti-D increases the platelet count more rapidly and for a longer period of time than 50 microg/kg/day in adults with immune thrombocytopenic purpura. Br J Haematol. 2001 Mar;112(4):1076-8. [https://doi.org/10.1046/j.1365-2141.2001.02627.x link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/11298610 PubMed]
 
 
==TT4 {{#subobject:d5ad89|Regimen=1}}==
 
==TT4 {{#subobject:d5ad89|Regimen=1}}==
 
 
 
TT4: '''<u>T</u>'''riple '''<u>T</u>'''herapy (4?)
 
TT4: '''<u>T</u>'''riple '''<u>T</u>'''herapy (4?)
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen {{#subobject:be2a8d|Variant=1}}===
 
===Regimen {{#subobject:be2a8d|Variant=1}}===
 
{| class="wikitable" style="width: 40%; text-align:center;"  
 
{| class="wikitable" style="width: 40%; text-align:center;"  
Line 370: Line 369:
 
|-
 
|-
 
|}
 
|}
''The authors do not specify whether modified or non-modified cyclosporine was used in this protocol. Please contact them for clarifications.''
+
''Note: The authors do not specify whether modified or non-modified cyclosporine was used in this protocol. Please contact them for clarifications.''
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Dexamethasone (Decadron)]] 40 mg/day PO on days 1 to 4
 
*[[Dexamethasone (Decadron)]] 40 mg/day PO on days 1 to 4
Line 376: Line 376:
 
**Dose adjusted for target trough of 200 to 400 mcg/L
 
**Dose adjusted for target trough of 200 to 400 mcg/L
 
*[[Rituximab (Rituxan)]] 100 mg IV once per day on days 7, 14, 21, 28
 
*[[Rituximab (Rituxan)]] 100 mg IV once per day on days 7, 14, 21, 28
 
+
</div></div>
 
===References===
 
===References===
 
# Choi PY, Roncolato F, Badoux X, Ramanathan S, Ho SJ, Chong BH. A novel triple therapy for ITP using high-dose dexamethasone, low-dose rituximab, and cyclosporine (TT4). Blood. 2015 Jul 23;126(4):500-3. Epub 2015 May 13. [http://www.bloodjournal.org/content/126/4/500.long link to original article] '''contains dosing details in abstract''' [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4560338/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/25972158 PubMed]
 
# Choi PY, Roncolato F, Badoux X, Ramanathan S, Ho SJ, Chong BH. A novel triple therapy for ITP using high-dose dexamethasone, low-dose rituximab, and cyclosporine (TT4). Blood. 2015 Jul 23;126(4):500-3. Epub 2015 May 13. [http://www.bloodjournal.org/content/126/4/500.long link to original article] '''contains dosing details in abstract''' [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4560338/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/25972158 PubMed]
 
 
=Relapsed or refractory=
 
=Relapsed or refractory=
 
==ATRA & Danazol {{#subobject:43e110|Regimen=1}}==
 
==ATRA & Danazol {{#subobject:43e110|Regimen=1}}==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen {{#subobject:077574|Variant=1}}===
 
===Regimen {{#subobject:077574|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
Line 396: Line 395:
 
|-
 
|-
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Targeted therapy====
 
====Targeted therapy====
 
*[[All-trans retinoic acid (ATRA)]] 10 mg PO twice per day
 
*[[All-trans retinoic acid (ATRA)]] 10 mg PO twice per day
 
====Endocrine therapy====
 
====Endocrine therapy====
 
*[[Danazol (Danocrine)]] 200 mg PO twice per day
 
*[[Danazol (Danocrine)]] 200 mg PO twice per day
 
 
'''16-week course'''
 
'''16-week course'''
 
+
</div></div>
 
===References===
 
===References===
 
# '''U1111-1132-6877:''' Feng FE, Feng R, Wang M, Zhang JM, Jiang H, Jiang Q, Lu J, Liu H, Peng J, Hou M, Shen JL, Wang JW, Xu LP, Liu KY, Huang XJ, Zhang XH. Oral all-trans retinoic acid plus danazol versus danazol as second-line treatment in adults with primary immune thrombocytopenia: a multicentre, randomised, open-label, phase 2 trial. Lancet Haematol. 2017 Oct;4(10):e487-e496. Epub 2017 Sep 13. [https://doi.org/10.1016/S2352-3026(17)30170-9 link to original article] '''contains dosing details in abstract''' [https://pubmed.ncbi.nlm.nih.gov/28917657 PubMed] NCT01667263
 
# '''U1111-1132-6877:''' Feng FE, Feng R, Wang M, Zhang JM, Jiang H, Jiang Q, Lu J, Liu H, Peng J, Hou M, Shen JL, Wang JW, Xu LP, Liu KY, Huang XJ, Zhang XH. Oral all-trans retinoic acid plus danazol versus danazol as second-line treatment in adults with primary immune thrombocytopenia: a multicentre, randomised, open-label, phase 2 trial. Lancet Haematol. 2017 Oct;4(10):e487-e496. Epub 2017 Sep 13. [https://doi.org/10.1016/S2352-3026(17)30170-9 link to original article] '''contains dosing details in abstract''' [https://pubmed.ncbi.nlm.nih.gov/28917657 PubMed] NCT01667263
 
 
==Avatrombopag monotherapy {{#subobject:c8da29|Regimen=1}}==
 
==Avatrombopag monotherapy {{#subobject:c8da29|Regimen=1}}==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen {{#subobject:1aab54|Variant=1}}===
 
===Regimen {{#subobject:1aab54|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
Line 421: Line 419:
 
|-
 
|-
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Growth factor therapy====
 
====Growth factor therapy====
 
*[[Avatrombopag (Doptelet)]]
 
*[[Avatrombopag (Doptelet)]]
 
+
</div></div>
 
===References===
 
===References===
 
# Bussel JB, Kuter DJ, Aledort LM, Kessler CM, Cuker A, Pendergrass KB, Tang S, McIntosh J. A randomized trial of avatrombopag, an investigational thrombopoietin-receptor agonist, in persistent and chronic immune thrombocytopenia. Blood. 2014 Jun 19;123(25):3887-94. Epub 2014 May 6. [http://www.bloodjournal.org/content/123/25/3887.long link to original article] [https://pubmed.ncbi.nlm.nih.gov/24802775 PubMed]
 
# Bussel JB, Kuter DJ, Aledort LM, Kessler CM, Cuker A, Pendergrass KB, Tang S, McIntosh J. A randomized trial of avatrombopag, an investigational thrombopoietin-receptor agonist, in persistent and chronic immune thrombocytopenia. Blood. 2014 Jun 19;123(25):3887-94. Epub 2014 May 6. [http://www.bloodjournal.org/content/123/25/3887.long link to original article] [https://pubmed.ncbi.nlm.nih.gov/24802775 PubMed]
 
# '''E5501-G000-302:''' Jurczak W, Chojnowski K, Mayer J, Krawczyk K, Jamieson BD, Tian W, Allen LF. Phase 3 randomised study of avatrombopag, a novel thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia. Br J Haematol. 2018 Nov;183(3):479-490. Epub 2018 Sep 7. [https://doi.org/10.1111/bjh.15573 link to original article] [https://pubmed.ncbi.nlm.nih.gov/30191972 PubMed]
 
# '''E5501-G000-302:''' Jurczak W, Chojnowski K, Mayer J, Krawczyk K, Jamieson BD, Tian W, Allen LF. Phase 3 randomised study of avatrombopag, a novel thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia. Br J Haematol. 2018 Nov;183(3):479-490. Epub 2018 Sep 7. [https://doi.org/10.1111/bjh.15573 link to original article] [https://pubmed.ncbi.nlm.nih.gov/30191972 PubMed]
 
 
==Cyclosporine monotherapy {{#subobject:4d3847|Regimen=1}}==
 
==Cyclosporine monotherapy {{#subobject:4d3847|Regimen=1}}==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
 
 
===Regimen {{#subobject:df1b55|Variant=1}}===
 
===Regimen {{#subobject:df1b55|Variant=1}}===
 
{| class="wikitable" style="width: 40%; text-align:center;"  
 
{| class="wikitable" style="width: 40%; text-align:center;"  
Line 440: Line 437:
 
|-
 
|-
 
|}
 
|}
 
+
''Note: The authors do not specify whether modified or non-modified cyclosporine was used in this protocol. Please contact them for clarifications.''
''The authors do not specify whether modified or non-modified cyclosporine was used in this protocol. Please contact them for clarifications.''
+
<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Cyclosporine|Cyclosporine A]] as follows:
 
*[[Cyclosporine|Cyclosporine A]] as follows:
 
**Days 1 to 6: 5 mg/kg/day PO split into twice per day dosing
 
**Days 1 to 6: 5 mg/kg/day PO split into twice per day dosing
 
**Day 7 onwards: 2.5 to 3 mg/kg/day PO with dose adjusted for target trough of 200 to 400 mcg/L
 
**Day 7 onwards: 2.5 to 3 mg/kg/day PO with dose adjusted for target trough of 200 to 400 mcg/L
 
+
</div></div>
 
===References===
 
===References===
 
# Emilia G, Morselli M, Luppi M, Longo G, Marasca R, Gandini G, Ferrara L, D'Apollo N, Potenza L, Bertesi M, Torelli G. Long-term salvage therapy with cyclosporin A in refractory idiopathic thrombocytopenic purpura. Blood. 2002 Feb 15;99(4):1482-5. [http://www.bloodjournal.org/content/99/4/1482 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/11830504 PubMed]
 
# Emilia G, Morselli M, Luppi M, Longo G, Marasca R, Gandini G, Ferrara L, D'Apollo N, Potenza L, Bertesi M, Torelli G. Long-term salvage therapy with cyclosporin A in refractory idiopathic thrombocytopenic purpura. Blood. 2002 Feb 15;99(4):1482-5. [http://www.bloodjournal.org/content/99/4/1482 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/11830504 PubMed]
 
 
==Danazol monotherapy {{#subobject:e7bf80|Regimen=1}}==
 
==Danazol monotherapy {{#subobject:e7bf80|Regimen=1}}==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen {{#subobject:9a63e6|Variant=1}}===
 
===Regimen {{#subobject:9a63e6|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
Line 471: Line 467:
 
|}
 
|}
 
''Note: Ahn et al. 1983 gave a range of 200 mg PO from two to four times per day; the dose below is from U1111-1132-6877''
 
''Note: Ahn et al. 1983 gave a range of 200 mg PO from two to four times per day; the dose below is from U1111-1132-6877''
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Endocrine therapy====
 
====Endocrine therapy====
 
*[[Danazol (Danocrine)]] 200 mg PO twice per day
 
*[[Danazol (Danocrine)]] 200 mg PO twice per day
 
 
'''16-week course'''
 
'''16-week course'''
 
+
</div></div>
 
===References===
 
===References===
 
# Ahn YS, Harrington WJ, Simon SR, Mylvaganam R, Pall LM, So AG. Danazol for the treatment of idiopathic thrombocytopenic purpura. N Engl J Med. 1983 Jun 9;308(23):1396-9. [https://doi.org/10.1056/NEJM198306093082306 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/6682484 PubMed]
 
# Ahn YS, Harrington WJ, Simon SR, Mylvaganam R, Pall LM, So AG. Danazol for the treatment of idiopathic thrombocytopenic purpura. N Engl J Med. 1983 Jun 9;308(23):1396-9. [https://doi.org/10.1056/NEJM198306093082306 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/6682484 PubMed]
 
# '''U1111-1132-6877:''' Feng FE, Feng R, Wang M, Zhang JM, Jiang H, Jiang Q, Lu J, Liu H, Peng J, Hou M, Shen JL, Wang JW, Xu LP, Liu KY, Huang XJ, Zhang XH. Oral all-trans retinoic acid plus danazol versus danazol as second-line treatment in adults with primary immune thrombocytopenia: a multicentre, randomised, open-label, phase 2 trial. Lancet Haematol. 2017 Oct;4(10):e487-e496. Epub 2017 Sep 13. [https://doi.org/10.1016/S2352-3026(17)30170-9 link to original article] '''contains dosing details in abstract''' [https://pubmed.ncbi.nlm.nih.gov/28917657 PubMed] NCT01667263
 
# '''U1111-1132-6877:''' Feng FE, Feng R, Wang M, Zhang JM, Jiang H, Jiang Q, Lu J, Liu H, Peng J, Hou M, Shen JL, Wang JW, Xu LP, Liu KY, Huang XJ, Zhang XH. Oral all-trans retinoic acid plus danazol versus danazol as second-line treatment in adults with primary immune thrombocytopenia: a multicentre, randomised, open-label, phase 2 trial. Lancet Haematol. 2017 Oct;4(10):e487-e496. Epub 2017 Sep 13. [https://doi.org/10.1016/S2352-3026(17)30170-9 link to original article] '''contains dosing details in abstract''' [https://pubmed.ncbi.nlm.nih.gov/28917657 PubMed] NCT01667263
 
 
==Dexamethasone monotherapy {{#subobject:a6cd06|Regimen=1}}==
 
==Dexamethasone monotherapy {{#subobject:a6cd06|Regimen=1}}==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen {{#subobject:8c260f|Variant=1}}===
 
===Regimen {{#subobject:8c260f|Variant=1}}===
 
{| class="wikitable" style="width: 40%; text-align:center;"  
 
{| class="wikitable" style="width: 40%; text-align:center;"  
Line 491: Line 486:
 
|-
 
|-
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Dexamethasone (Decadron)]] 40 mg PO once per day on days 1 to 4
 
*[[Dexamethasone (Decadron)]] 40 mg PO once per day on days 1 to 4
 
 
'''28-day cycles for 6 cycles'''
 
'''28-day cycles for 6 cycles'''
 
+
</div></div>
 
===References===
 
===References===
 
# Andersen JC. Response of resistant idiopathic thrombocytopenic purpura to pulsed high-dose dexamethasone therapy. N Engl J Med. 1994 Jun 2;330(22):1560-4. [https://doi.org/10.1056/NEJM199406023302203 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/8177245 PubMed]
 
# Andersen JC. Response of resistant idiopathic thrombocytopenic purpura to pulsed high-dose dexamethasone therapy. N Engl J Med. 1994 Jun 2;330(22):1560-4. [https://doi.org/10.1056/NEJM199406023302203 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/8177245 PubMed]
 
 
==Dexamethasone & Rituximab {{#subobject:52f32|Regimen=1}}==
 
==Dexamethasone & Rituximab {{#subobject:52f32|Regimen=1}}==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
 
 
===Regimen ("R+3Dex") {{#subobject:abae3b|Variant=1}}===
 
===Regimen ("R+3Dex") {{#subobject:abae3b|Variant=1}}===
 
{| class="wikitable" style="width: 40%; text-align:center;"  
 
{| class="wikitable" style="width: 40%; text-align:center;"  
Line 511: Line 504:
 
|-
 
|-
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Dexamethasone (Decadron)]] 28 mg/m<sup>2</sup>/day (maximum dose of 40 mg) on days 1 to 4
 
*[[Dexamethasone (Decadron)]] 28 mg/m<sup>2</sup>/day (maximum dose of 40 mg) on days 1 to 4
 
*[[Rituximab (Rituxan)]] as follows:
 
*[[Rituximab (Rituxan)]] as follows:
 
**Cycles 1 & 2: 375 mg/m<sup>2</sup> IV once per day on days 1 & 8
 
**Cycles 1 & 2: 375 mg/m<sup>2</sup> IV once per day on days 1 & 8
 
 
'''14-day cycle for 3 cycles'''
 
'''14-day cycle for 3 cycles'''
 
+
</div></div>
 
===References===
 
===References===
 
# '''Abstract:''' Allison Imahiyerobo, Micha Thompson, Marina Izak Karaev, Waleed Ghanima, James B Bussel. Rituximab Combined With Three Cycles Of High Dose Dexamethasone Provides a Long Term Response Rate Similar To That Of Splenectomy In Patients With Immune Thrombocytopenia (ITP) Of Duration Less Than 2 Years. Blood Nov 2013,122(21)2310. [http://www.bloodjournal.org/content/122/21/2310 link to abstract]
 
# '''Abstract:''' Allison Imahiyerobo, Micha Thompson, Marina Izak Karaev, Waleed Ghanima, James B Bussel. Rituximab Combined With Three Cycles Of High Dose Dexamethasone Provides a Long Term Response Rate Similar To That Of Splenectomy In Patients With Immune Thrombocytopenia (ITP) Of Duration Less Than 2 Years. Blood Nov 2013,122(21)2310. [http://www.bloodjournal.org/content/122/21/2310 link to abstract]
 
 
==Eltrombopag monotherapy {{#subobject:170bd|Regimen=1}}==
 
==Eltrombopag monotherapy {{#subobject:170bd|Regimen=1}}==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
 
 
===Regimen variant #1 {{#subobject:90336c|Variant=1}}===
 
===Regimen variant #1 {{#subobject:90336c|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
Line 537: Line 528:
 
|-
 
|-
 
|}
 
|}
''This regimen was intended for pediatric patients.''
+
''Note: This regimen was intended for pediatric patients.''
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Growth factor therapy====
 
====Growth factor therapy====
 
*[[Eltrombopag (Promacta)]] with starting dose by the following criteria:
 
*[[Eltrombopag (Promacta)]] with starting dose by the following criteria:
Line 546: Line 538:
 
**Age 1 to 5, non-east Asian: 1.2 mg/kg/day oral suspension
 
**Age 1 to 5, non-east Asian: 1.2 mg/kg/day oral suspension
 
**Age 1 to 5, east Asian: 0.8 mg/kg/day oral suspension
 
**Age 1 to 5, east Asian: 0.8 mg/kg/day oral suspension
 
 
'''Dose adjusted to a maximum of 75 mg/day, with temporary discontinuation for platelet count greater than 400 x 10<sup>9</sup>/L'''
 
'''Dose adjusted to a maximum of 75 mg/day, with temporary discontinuation for platelet count greater than 400 x 10<sup>9</sup>/L'''
 
+
</div></div><br>
 +
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen variant #2 {{#subobject:5912e7|Variant=1}}===
 
===Regimen variant #2 {{#subobject:5912e7|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
Line 562: Line 554:
 
|-
 
|-
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Growth factor therapy====
 
====Growth factor therapy====
 
*[[Eltrombopag (Promacta)]] 50 mg (starting dose) PO once per day
 
*[[Eltrombopag (Promacta)]] 50 mg (starting dose) PO once per day
 
 
'''The dose could be increased from 50 mg to 75 mg after 3 weeks in patients whose platelet counts were less than 50 x 10<sup>9</sup>/L. Treatment was discontinued in patients who attained a platelet count greater than 200 x 10<sup>9</sup>/L'''
 
'''The dose could be increased from 50 mg to 75 mg after 3 weeks in patients whose platelet counts were less than 50 x 10<sup>9</sup>/L. Treatment was discontinued in patients who attained a platelet count greater than 200 x 10<sup>9</sup>/L'''
 
+
</div></div><br>
 +
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen variant #3 {{#subobject:9ac7d2|Variant=1}}===
 
===Regimen variant #3 {{#subobject:9ac7d2|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
Line 581: Line 574:
 
|}
 
|}
 
''Note: this trial should not be confused with the trial by the same name in colorectal cancer.''
 
''Note: this trial should not be confused with the trial by the same name in colorectal cancer.''
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Growth factor therapy====
 
====Growth factor therapy====
 
*[[Eltrombopag (Promacta)]] 50 mg PO once per day, with dose modifications:
 
*[[Eltrombopag (Promacta)]] 50 mg PO once per day, with dose modifications:
Line 586: Line 580:
 
**Decrease to 25 mg PO once per day required for platelets counts between 200 and 400 x 10<sup>9</sup>/L
 
**Decrease to 25 mg PO once per day required for platelets counts between 200 and 400 x 10<sup>9</sup>/L
 
**Drug was held for platelet count greater than 400 x 10<sup>9</sup>/L, until platelet count dropped below 150 x 10<sup>9</sup>/L
 
**Drug was held for platelet count greater than 400 x 10<sup>9</sup>/L, until platelet count dropped below 150 x 10<sup>9</sup>/L
 
 
'''6-month course'''
 
'''6-month course'''
 
+
</div></div><br>
 +
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen variant #4 {{#subobject:b1f517|Variant=1}}===
 
===Regimen variant #4 {{#subobject:b1f517|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
Line 602: Line 596:
 
|-
 
|-
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Growth factor therapy====
 
====Growth factor therapy====
 
*[[Eltrombopag (Promacta)]] 50 mg PO once per day
 
*[[Eltrombopag (Promacta)]] 50 mg PO once per day
 
 
'''Up to 6-week course'''
 
'''Up to 6-week course'''
 
+
</div></div><br>
 +
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen variant #5 {{#subobject:55f69d|Variant=1}}===
 
===Regimen variant #5 {{#subobject:55f69d|Variant=1}}===
 
{| class="wikitable" style="width: 40%; text-align:center;"  
 
{| class="wikitable" style="width: 40%; text-align:center;"  
Line 616: Line 611:
 
|-
 
|-
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Growth factor therapy====
 
====Growth factor therapy====
 
*[[Eltrombopag (Promacta)]] 50 mg PO once per day, with adjustments:
 
*[[Eltrombopag (Promacta)]] 50 mg PO once per day, with adjustments:
 
 
''Dose and schedule were individualized with the goal of achieving and maintaining platelets between 50 and 200 x 10<sup>9</sup>/L.''
 
''Dose and schedule were individualized with the goal of achieving and maintaining platelets between 50 and 200 x 10<sup>9</sup>/L.''
 
+
</div></div>
 
===References===
 
===References===
 
# '''TRA100773:''' Bussel JB, Cheng G, Saleh MN, Psaila B, Kovaleva L, Meddeb B, Kloczko J, Hassani H, Mayer B, Stone NL, Arning M, Provan D, Jenkins JM. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med. 2007 Nov 29;357(22):2237-47. [https://doi.org/10.1056/NEJMoa073275 link to original article] [https://pubmed.ncbi.nlm.nih.gov/18046028 PubMed] NCT00102739
 
# '''TRA100773:''' Bussel JB, Cheng G, Saleh MN, Psaila B, Kovaleva L, Meddeb B, Kloczko J, Hassani H, Mayer B, Stone NL, Arning M, Provan D, Jenkins JM. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med. 2007 Nov 29;357(22):2237-47. [https://doi.org/10.1056/NEJMoa073275 link to original article] [https://pubmed.ncbi.nlm.nih.gov/18046028 PubMed] NCT00102739
Line 628: Line 623:
 
# '''PETIT2:''' Grainger JD, Locatelli F, Chotsampancharoen T, Donyush E, Pongtanakul B, Komvilaisak P, Sosothikul D, Drelichman G, Sirachainan N, Holzhauer S, Lebedev V, Lemons R, Pospisilova D, Ramenghi U, Bussel JB, Bakshi KK, Iyengar M, Chan GW, Chagin KD, Theodore D, Marcello LM, Bailey CK. Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomised, multicentre, placebo-controlled trial. Lancet. 2015 Oct 24;386(10004):1649-58. Epub 2015 Jul 28. [https://doi.org/10.1016/S0140-6736(15)61107-2 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/26231455 PubMed] NCT01520909
 
# '''PETIT2:''' Grainger JD, Locatelli F, Chotsampancharoen T, Donyush E, Pongtanakul B, Komvilaisak P, Sosothikul D, Drelichman G, Sirachainan N, Holzhauer S, Lebedev V, Lemons R, Pospisilova D, Ramenghi U, Bussel JB, Bakshi KK, Iyengar M, Chan GW, Chagin KD, Theodore D, Marcello LM, Bailey CK. Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomised, multicentre, placebo-controlled trial. Lancet. 2015 Oct 24;386(10004):1649-58. Epub 2015 Jul 28. [https://doi.org/10.1016/S0140-6736(15)61107-2 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/26231455 PubMed] NCT01520909
 
# Yang R, Li J, Jin J, Huang M, Yu Z, Xu X, Zhang X, Hou M. Multicentre, randomised phase III study of the efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia. Br J Haematol. 2017 Jan;176(1):101-110. [https://doi.org/10.1111/bjh.14380 link to original article] [https://pubmed.ncbi.nlm.nih.gov/27734464 PubMed]
 
# Yang R, Li J, Jin J, Huang M, Yu Z, Xu X, Zhang X, Hou M. Multicentre, randomised phase III study of the efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia. Br J Haematol. 2017 Jan;176(1):101-110. [https://doi.org/10.1111/bjh.14380 link to original article] [https://pubmed.ncbi.nlm.nih.gov/27734464 PubMed]
 
 
==Fostamatinib monotherapy {{#subobject:ddb228|Regimen=1}}==
 
==Fostamatinib monotherapy {{#subobject:ddb228|Regimen=1}}==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen {{#subobject:85221f|Variant=1}}===
 
===Regimen {{#subobject:85221f|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
Line 652: Line 646:
 
|-
 
|-
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Targeted therapy====
 
====Targeted therapy====
 
*[[Fostamatinib (Tavalisse)]] 100 mg PO twice per day  
 
*[[Fostamatinib (Tavalisse)]] 100 mg PO twice per day  
 
**Dose may be increased to 150 mg PO twice per day after 4 weeks if needed to achieve platelet count of at least 50
 
**Dose may be increased to 150 mg PO twice per day after 4 weeks if needed to achieve platelet count of at least 50
 
 
'''24-week course'''
 
'''24-week course'''
 
+
</div></div>
 
===References===
 
===References===
 
# '''FIT1:''' Bussel J, Arnold DM, Grossbard E, Mayer J, Treliński J, Homenda W, Hellmann A, Windyga J, Sivcheva L, Khalafallah AA, Zaja F, Cooper N, Markovtsov V, Zayed H, Duliege AM. Fostamatinib for the treatment of adult persistent and chronic immune thrombocytopenia: results of two phase 3, randomized, placebo-controlled trials. Am J Hematol. 2018 Jul;93(7):921-930. Epub 2018 May 15. [https://doi.org/10.1002/ajh.25125 link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6055608/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/29696684 PubMed] NCT02076399
 
# '''FIT1:''' Bussel J, Arnold DM, Grossbard E, Mayer J, Treliński J, Homenda W, Hellmann A, Windyga J, Sivcheva L, Khalafallah AA, Zaja F, Cooper N, Markovtsov V, Zayed H, Duliege AM. Fostamatinib for the treatment of adult persistent and chronic immune thrombocytopenia: results of two phase 3, randomized, placebo-controlled trials. Am J Hematol. 2018 Jul;93(7):921-930. Epub 2018 May 15. [https://doi.org/10.1002/ajh.25125 link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6055608/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/29696684 PubMed] NCT02076399
 
# '''FIT2:''' Bussel J, Arnold DM, Grossbard E, Mayer J, Treliński J, Homenda W, Hellmann A, Windyga J, Sivcheva L, Khalafallah AA, Zaja F, Cooper N, Markovtsov V, Zayed H, Duliege AM. Fostamatinib for the treatment of adult persistent and chronic immune thrombocytopenia: results of two phase 3, randomized, placebo-controlled trials. Am J Hematol. 2018 Jul;93(7):921-930. Epub 2018 May 15. [https://doi.org/10.1002/ajh.25125 link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6055608/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/29696684 PubMed] NCT02076412
 
# '''FIT2:''' Bussel J, Arnold DM, Grossbard E, Mayer J, Treliński J, Homenda W, Hellmann A, Windyga J, Sivcheva L, Khalafallah AA, Zaja F, Cooper N, Markovtsov V, Zayed H, Duliege AM. Fostamatinib for the treatment of adult persistent and chronic immune thrombocytopenia: results of two phase 3, randomized, placebo-controlled trials. Am J Hematol. 2018 Jul;93(7):921-930. Epub 2018 May 15. [https://doi.org/10.1002/ajh.25125 link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6055608/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/29696684 PubMed] NCT02076412
 
# '''Review:''' Newland A, Lee EJ, McDonald V, Bussel JB. Fostamatinib for persistent/chronic adult immune thrombocytopenia. Immunotherapy. 2018 Jan;10(1):9-25. [https://www.futuremedicine.com/doi/10.2217/imt-2017-0097?url_ver=Z39.88-2003 link to original article] [https://pubmed.ncbi.nlm.nih.gov/28967793 PubMed]
 
# '''Review:''' Newland A, Lee EJ, McDonald V, Bussel JB. Fostamatinib for persistent/chronic adult immune thrombocytopenia. Immunotherapy. 2018 Jan;10(1):9-25. [https://www.futuremedicine.com/doi/10.2217/imt-2017-0097?url_ver=Z39.88-2003 link to original article] [https://pubmed.ncbi.nlm.nih.gov/28967793 PubMed]
 
 
==Mycophenolate mofetil monotherapy {{#subobject:ddb338|Regimen=1}}==
 
==Mycophenolate mofetil monotherapy {{#subobject:ddb338|Regimen=1}}==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
 
 
===Regimen {{#subobject:850f1f|Variant=1}}===
 
===Regimen {{#subobject:850f1f|Variant=1}}===
 
{| class="wikitable" style="width: 40%; text-align:center;"  
 
{| class="wikitable" style="width: 40%; text-align:center;"  
Line 675: Line 667:
 
|-
 
|-
 
|}
 
|}
 +
<div class="toccolours" style="background-color:#b3e2cd">
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Mycophenolate mofetil (CellCept)]] 1 gm/day PO
 
*[[Mycophenolate mofetil (CellCept)]] 1 gm/day PO
 
 
'''Continued indefinitely'''
 
'''Continued indefinitely'''
 
+
</div></div>
 
===References===
 
===References===
 
# '''Retrospective:''' Taylor A, Neave L, Solanki S, Westwood JP, Terrinonive I, McGuckin S, Kothari J, Cooper N, Stasi R, Scully M. Mycophenolate mofetil therapy for severe immune thrombocytopenia. Br J Haematol. 2015 Nov;171(4):625-30. Epub 2015 Aug 6. [https://doi.org/10.1111/bjh.13622 link to original article] [https://pubmed.ncbi.nlm.nih.gov/26250874 PubMed]
 
# '''Retrospective:''' Taylor A, Neave L, Solanki S, Westwood JP, Terrinonive I, McGuckin S, Kothari J, Cooper N, Stasi R, Scully M. Mycophenolate mofetil therapy for severe immune thrombocytopenia. Br J Haematol. 2015 Nov;171(4):625-30. Epub 2015 Aug 6. [https://doi.org/10.1111/bjh.13622 link to original article] [https://pubmed.ncbi.nlm.nih.gov/26250874 PubMed]
 
 
==Placebo==  
 
==Placebo==  
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
 
 
===Regimen===
 
===Regimen===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
Line 730: Line 720:
 
|}
 
|}
 
''No active treatment; used as a comparator arm and here for reference purposes only. Note that RAISE should not be confused with the trial by the same name in colorectal cancer.''
 
''No active treatment; used as a comparator arm and here for reference purposes only. Note that RAISE should not be confused with the trial by the same name in colorectal cancer.''
 
+
</div></div>
 
===References===
 
===References===
 
# '''Amgen 20030105:''' Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrère F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. [https://doi.org/10.1016/S0140-6736(08)60203-2 link to original article] [https://pubmed.ncbi.nlm.nih.gov/18242413 PubMed] NCT00102323
 
# '''Amgen 20030105:''' Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrère F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. [https://doi.org/10.1016/S0140-6736(08)60203-2 link to original article] [https://pubmed.ncbi.nlm.nih.gov/18242413 PubMed] NCT00102323
Line 740: Line 730:
 
# '''Amgen 20080279:''' Tarantino MD, Bussel JB, Blanchette VS, Despotovic J, Bennett C, Raj A, Williams B, Beam D, Morales J, Rose MJ, Carpenter N, Nie K, Eisen M. Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study. Lancet. 2016 Jul 2;388(10039):45-54. Epub 2016 Apr 18. [https://doi.org/10.1016/S0140-6736(16)00279-8 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/27103127 PubMed] NCT01444417
 
# '''Amgen 20080279:''' Tarantino MD, Bussel JB, Blanchette VS, Despotovic J, Bennett C, Raj A, Williams B, Beam D, Morales J, Rose MJ, Carpenter N, Nie K, Eisen M. Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study. Lancet. 2016 Jul 2;388(10039):45-54. Epub 2016 Apr 18. [https://doi.org/10.1016/S0140-6736(16)00279-8 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/27103127 PubMed] NCT01444417
 
# Yang R, Li J, Jin J, Huang M, Yu Z, Xu X, Zhang X, Hou M. Multicentre, randomised phase III study of the efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia. Br J Haematol. 2017 Jan;176(1):101-110. [https://doi.org/10.1111/bjh.14380 link to original article] [https://pubmed.ncbi.nlm.nih.gov/27734464 PubMed]
 
# Yang R, Li J, Jin J, Huang M, Yu Z, Xu X, Zhang X, Hou M. Multicentre, randomised phase III study of the efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia. Br J Haematol. 2017 Jan;176(1):101-110. [https://doi.org/10.1111/bjh.14380 link to original article] [https://pubmed.ncbi.nlm.nih.gov/27734464 PubMed]
 
 
==Rituximab monotherapy {{#subobject:d7d211|Regimen=1}}==
 
==Rituximab monotherapy {{#subobject:d7d211|Regimen=1}}==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
 
 
===Regimen {{#subobject:128a52|Variant=1}}===
 
===Regimen {{#subobject:128a52|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
Line 765: Line 753:
 
====Immunosuppressive therapy====
 
====Immunosuppressive therapy====
 
*[[Rituximab (Rituxan)]] 375 mg/m<sup>2</sup> IV once per day on days 1, 8, 15, 22
 
*[[Rituximab (Rituxan)]] 375 mg/m<sup>2</sup> IV once per day on days 1, 8, 15, 22
 
 
====Supportive therapy====
 
====Supportive therapy====
 
*(per Godeau et al. 2008):
 
*(per Godeau et al. 2008):
Line 771: Line 758:
 
*[[Acetaminophen (Tylenol)]] 1000 mg once per day on days 1, 8, 15, 22, prior to [[Rituximab (Rituxan)]]
 
*[[Acetaminophen (Tylenol)]] 1000 mg once per day on days 1, 8, 15, 22, prior to [[Rituximab (Rituxan)]]
 
*[[Methylprednisolone (Solumedrol)]] 60 mg IV once per day on days 1, 8, 15, 22, prior to [[Rituximab (Rituxan)]]
 
*[[Methylprednisolone (Solumedrol)]] 60 mg IV once per day on days 1, 8, 15, 22, prior to [[Rituximab (Rituxan)]]
 
 
'''4-week course'''
 
'''4-week course'''
 
+
</div></div>
 
===References===
 
===References===
 
# Godeau B, Porcher R, Fain O, Lefrère F, Fenaux P, Cheze S, Vekhoff A, Chauveheid MP, Stirnemann J, Galicier L, Bourgeois E, Haiat S, Varet B, Leporrier M, Papo T, Khellaf M, Michel M, Bierling P. Rituximab efficacy and safety in adult splenectomy candidates with chronic immune thrombocytopenic purpura: results of a prospective multicenter phase 2 study. Blood. 2008 Aug 15;112(4):999-1004. [http://www.bloodjournal.org/content/112/4/999.long link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/18463354 PubMed]
 
# Godeau B, Porcher R, Fain O, Lefrère F, Fenaux P, Cheze S, Vekhoff A, Chauveheid MP, Stirnemann J, Galicier L, Bourgeois E, Haiat S, Varet B, Leporrier M, Papo T, Khellaf M, Michel M, Bierling P. Rituximab efficacy and safety in adult splenectomy candidates with chronic immune thrombocytopenic purpura: results of a prospective multicenter phase 2 study. Blood. 2008 Aug 15;112(4):999-1004. [http://www.bloodjournal.org/content/112/4/999.long link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/18463354 PubMed]
 
# '''Prospective cohort:''' Patel VL, Mahévas M, Lee SY, Stasi R, Cunningham-Rundles S, Godeau B, Kanter J, Neufeld E, Taube T, Ramenghi U, Shenoy S, Ward MJ, Mihatov N, Patel VL, Bierling P, Lesser M, Cooper N, Bussel JB. Outcomes 5 years after response to rituximab therapy in children and adults with immune thrombocytopenia. Blood. 2012 Jun 21;119(25):5989-95. [http://www.bloodjournal.org/content/119/25/5989.long link to original article] '''contains dosing details in manuscript''' [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3383014/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/22566601 PubMed]
 
# '''Prospective cohort:''' Patel VL, Mahévas M, Lee SY, Stasi R, Cunningham-Rundles S, Godeau B, Kanter J, Neufeld E, Taube T, Ramenghi U, Shenoy S, Ward MJ, Mihatov N, Patel VL, Bierling P, Lesser M, Cooper N, Bussel JB. Outcomes 5 years after response to rituximab therapy in children and adults with immune thrombocytopenia. Blood. 2012 Jun 21;119(25):5989-95. [http://www.bloodjournal.org/content/119/25/5989.long link to original article] '''contains dosing details in manuscript''' [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3383014/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/22566601 PubMed]
 
# '''RITP:''' Ghanima W, Khelif A, Waage A, Michel M, Tjønnfjord GE, Romdhan NB, Kahrs J, Darne B, Holme PA; RITP study group. Rituximab as second-line treatment for adult immune thrombocytopenia (the RITP trial): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet. 2015 Apr 25;385(9978):1653-61. Epub 2015 Feb 4.[https://doi.org/10.1016/S0140-6736(14)61495-1 link to original article] '''contains dosing details in abstract''' [https://pubmed.ncbi.nlm.nih.gov/25662413 PubMed] NCT00344149
 
# '''RITP:''' Ghanima W, Khelif A, Waage A, Michel M, Tjønnfjord GE, Romdhan NB, Kahrs J, Darne B, Holme PA; RITP study group. Rituximab as second-line treatment for adult immune thrombocytopenia (the RITP trial): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet. 2015 Apr 25;385(9978):1653-61. Epub 2015 Feb 4.[https://doi.org/10.1016/S0140-6736(14)61495-1 link to original article] '''contains dosing details in abstract''' [https://pubmed.ncbi.nlm.nih.gov/25662413 PubMed] NCT00344149
 
 
==Romiplostim monotherapy {{#subobject:a6df46|Regimen=1}}==  
 
==Romiplostim monotherapy {{#subobject:a6df46|Regimen=1}}==  
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
 
 
===Regimen {{#subobject:8b8d3b|Variant=1}}===
 
===Regimen {{#subobject:8b8d3b|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
Line 808: Line 792:
 
====Growth factor therapy====
 
====Growth factor therapy====
 
*[[Romiplostim (Nplate)]] 1 mcg/kg SC once per day on days 1, 8, 15, 22
 
*[[Romiplostim (Nplate)]] 1 mcg/kg SC once per day on days 1, 8, 15, 22
 
 
'''28-day cycles'''
 
'''28-day cycles'''
 
+
</div></div>
 
===References===
 
===References===
 
# '''Amgen 20030105:''' Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrère F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. [https://doi.org/10.1016/S0140-6736(08)60203-2 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/18242413 PubMed] NCT00102323
 
# '''Amgen 20030105:''' Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrère F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. [https://doi.org/10.1016/S0140-6736(08)60203-2 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/18242413 PubMed] NCT00102323
 
# '''Amgen 20030212:''' Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrère F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. [https://doi.org/10.1016/S0140-6736(08)60203-2 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/18242413 PubMed] NCT00102336
 
# '''Amgen 20030212:''' Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrère F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. [https://doi.org/10.1016/S0140-6736(08)60203-2 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/18242413 PubMed] NCT00102336
 
# '''Amgen 20080279:''' Tarantino MD, Bussel JB, Blanchette VS, Despotovic J, Bennett C, Raj A, Williams B, Beam D, Morales J, Rose MJ, Carpenter N, Nie K, Eisen M. Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study. Lancet. 2016 Jul 2;388(10039):45-54. Epub 2016 Apr 18. [https://doi.org/10.1016/S0140-6736(16)00279-8 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/27103127 PubMed] NCT01444417
 
# '''Amgen 20080279:''' Tarantino MD, Bussel JB, Blanchette VS, Despotovic J, Bennett C, Raj A, Williams B, Beam D, Morales J, Rose MJ, Carpenter N, Nie K, Eisen M. Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study. Lancet. 2016 Jul 2;388(10039):45-54. Epub 2016 Apr 18. [https://doi.org/10.1016/S0140-6736(16)00279-8 link to original article] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/27103127 PubMed] NCT01444417
 
 
== Vinblastine-loaded platelets ==
 
== Vinblastine-loaded platelets ==
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen ===
 
===Regimen ===
 
{| class="wikitable" style="width: 60%; text-align:center;"  
 
{| class="wikitable" style="width: 60%; text-align:center;"  
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|CR in 6 of 11 patients
 
|CR in 6 of 11 patients
 
|}
 
|}
 
 
=== Reference ===
 
=== Reference ===
 
# '''Phase 1:''' Ahn YS, Byrnes JJ, Harrington WJ, Cayer ML, Smith DS, Brunskill DE, Pall LM. New England Journal of Medicine. 1978; 298:1101-1107. [https://doi.org/10.1056/NEJM197805182982001 link to original article] [https://pubmed.ncbi.nlm.nih.gov/565464 PubMed]
 
# '''Phase 1:''' Ahn YS, Byrnes JJ, Harrington WJ, Cayer ML, Smith DS, Brunskill DE, Pall LM. New England Journal of Medicine. 1978; 298:1101-1107. [https://doi.org/10.1056/NEJM197805182982001 link to original article] [https://pubmed.ncbi.nlm.nih.gov/565464 PubMed]
 
 
[[Category:Immune thrombocytopenia regimens]]
 
[[Category:Immune thrombocytopenia regimens]]
 
[[Category:Disease-specific pages]]
 
[[Category:Disease-specific pages]]

Revision as of 12:14, 17 October 2022

Section editor transclusions

20 regimens on this page
30 variants on this page


Guidelines

ASH

Older

BSH

"How I Treat"

Initial therapy

Dexamethasone monotherapy

Regimen variant #1

Study Evidence Comparator Efficacy
Wei et al. 2015 Phase 3 (E-esc) Prednisone Superior CR rate

Immunosuppressive therapy

4-day course

Subsequent treatment

  • If platelets remained below 30 x 109/L or bleeding by day 10, course is repeated once


Regimen variant #2

Study Evidence Comparator Efficacy
Matschke et al. 2013 Phase 3 (E-esc) Prednisone Seems to have superior responding patients maintaining remission for at least 6 mo

Immunosuppressive therapy, part 1

Immunosuppressive therapy, part 2

21-day cycle for 6 cycles


Regimen variant #3

Study Evidence
Cheng et al. 2003 Phase 2

Immunosuppressive therapy

4-day course Patients who had an initial response, but whose platelets dropped below 30 x 109/L within 6 months received:


Regimen variant #4, monthly dexamethasone

Study Evidence
Mazzucconi et al. 2007 Phase 2

Immunosuppressive therapy

28-day cycle for 6 cycles


Regimen variant #5, bi-weekly dexamethasone

Study Evidence
Mazzucconi et al. 2007 Phase 2

Immunosuppressive therapy

  • Dexamethasone (Decadron) by the following criteria:
    • Adults: 40 mg IV or PO once per day on days 1 to 4
    • Patients less than 15 years old: 20 mg/m2 (maximum dose of 40 mg) IV or PO once per day on days 1 to 4
    • Patients who had platelet counts of less than or equal to 30 x 109/L between cycles and/or who had bleeding related to thrombocytopenia received 0.035 mg/kg PO once per day "between courses"

14-day cycle for 4 cycles

References

  1. Cheng Y, Wong RS, Soo YO, Chui CH, Lau FY, Chan NP, Wong WS, Cheng G. Initial treatment of immune thrombocytopenic purpura with high-dose dexamethasone. N Engl J Med. 2003 Aug 28;349(9):831-6. link to original article contains dosing details in manuscript PubMed content property of HemOnc.org
  2. Mazzucconi MG, Fazi P, Bernasconi S, De Rossi G, Leone G, Gugliotta L, Vianelli N, Avvisati G, Rodeghiero F, Amendola A, Baronci C, Carbone C, Quattrin S, Fioritoni G, D'Alfonso G, Mandelli F; Gruppo Italiano Malattie EMatologiche dell'Adulto (GIMEMA) Thrombocytopenia Working Party. Therapy with high-dose dexamethasone (HD-DXM) in previously untreated patients affected by idiopathic thrombocytopenic purpura: a GIMEMA experience. Blood. 2007 Feb 15;109(4):1401-7. Epub 2006 Oct 31. link to original article contains dosing details in manuscript PubMed
  3. Abstract: Johannes Matschke, Hannes Müller-Beißenhirtz, Ilona Vester, Bernd Hertenstein, Lewin Eisele, Hildegard Lax, Claudia Ose, Ulrich Dührsen. A Randomized Trial Of Daily Prednisone Versus Pulsed Dexamethasone In Treatment Naïve Patients With Idiopathic Thrombocytopenic Purpura. Blood Nov 2013,122(21)325. link to abstract
  4. Wei Y, Ji XB, Wang YW, Wang JX, Yang EQ, Wang ZC, Sang YQ, Bi ZM, Ren CA, Zhou F, Liu GQ, Peng J, Hou M. High-dose dexamethasone vs prednisone for treatment of adult immune thrombocytopenia: a prospective multicenter randomized trial. Blood. 2016 Jan 21;127(3):296-302. Epub 2015 Oct 19. link to original article contains dosing details in manuscript PubMed

Dexamethasone & Eltrombopag

Regimen

Study Evidence
Gómez-Almaguer et al. 2014 Pilot, <20 pts reported

Immunosuppressive therapy

Growth factor therapy

5-week course

References

  1. Gómez-Almaguer D, Herrera-Rojas MA, Jaime-Pérez JC, Gómez-De León A, Cantú-Rodríguez OG, Gutiérrez-Aguirre CH, Tarín-Arzaga L, Hernández-Reyes J, Ruiz-Arguelles GJ. Eltrombopag and high-dose dexamethasone as frontline treatment of newly diagnosed immune thrombocytopenia in adults. Blood. 2014 Jun 19;123(25):3906-8. Epub 2014 May 6. link to original article contains dosing details in manuscript PubMed

Dexamethasone & Mycophenolate mofetil

Regimen

Study Evidence Comparator Efficacy
Bradbury et al. 2021 (FLIGHT) Phase 3 (E-esc) 1. Dexamethasone
2. Prednisolone
Superior treatment failure

References

  1. FLIGHT: Bradbury CA, Pell J, Hill Q, Bagot C, Cooper N, Ingram J, Breheny K, Kandiyali R, Rayment R, Evans G, Talks K, Thomas I, Greenwood R. Mycophenolate Mofetil for First-Line Treatment of Immune Thrombocytopenia. N Engl J Med. 2021 Sep 2;385(10):885-895. link to original article PubMed NCT03156452

Dexamethasone & Rituximab

Regimen ("R+3Dex")

Study Evidence
Imahiyerobo et al. 2013 Retrospective

Immunosuppressive therapy

14-day cycle for 3 cycles

References

  1. Abstract: Retrospective: Allison Imahiyerobo, Micha Thompson, Marina Izak Karaev, Waleed Ghanima, James B Bussel. Rituximab Combined With Three Cycles Of High Dose Dexamethasone Provides a Long Term Response Rate Similar To That Of Splenectomy In Patients With Immune Thrombocytopenia (ITP) Of Duration Less Than 2 Years. Blood Nov 2013,122(21)2310. link to abstract

Intravenous immunoglobulin monotherapy

IVIG: IntraVenous ImmunoGlobulin

Regimen

Study Evidence Comparator Efficacy
Godeau et al. 1999 Phase 3 (E-esc) IVIG; 0.5 g/kg Superior response rate

Supportive therapy

One dose

References

  1. Imbach P, Wagner HP, Berchtold W, Gaedicke G, Hirt A, Joller P, Mueller-Eckhardt C, Müller B, Rossi E, Barandun S. Intravenous immunoglobulin versus oral corticosteroids in acute immune thrombocytopenic purpura in childhood. Lancet. 1985 Aug 31;2(8453):464-8. link to original article PubMed
  2. Blanchette VS, Luke B, Andrew M, Sommerville-Nielsen S, Barnard D, de Veber B, Gent M. A prospective, randomized trial of high-dose intravenous immune globulin G therapy, oral prednisone therapy, and no therapy in childhood acute immune thrombocytopenic purpura. J Pediatr. 1993 Dec;123(6):989-95. link to original article PubMed
  3. Blanchette V, Imbach P, Andrew M, Adams M, McMillan J, Wang E, Milner R, Ali K, Barnard D, Bernstein M, Esseltine D, Chan KW, de Veber B, Israels S, Kobrinsky N, Luke B. Randomised trial of intravenous immunoglobulin G, intravenous anti-D, and oral prednisone in childhood acute immune thrombocytopenic purpura. Lancet. 1994 Sep 10;344(8924):703-7. link to original article PubMed
  4. Godeau B, Caulier MT, Decuypere L, Rose C, Schaeffer A, Bierling P. Intravenous immunoglobulin for adults with autoimmune thrombocytopenic purpura: results of a randomized trial comparing 05 and 1 g/kg bw. Br J Haematol. 1999 Dec;107(4):716-9. link to original article contains dosing details in abstract PubMed
  5. Godeau B, Chevret S, Varet B, Lefrère F, Zini JM, Bassompierre F, Chèze S, Legouffe E, Hulin C, Grange MJ, Fain O, Bierling P; French ATIP Study Group. Intravenous immunoglobulin or high-dose methylprednisolone, with or without oral prednisone, for adults with untreated severe autoimmune thrombocytopenic purpura: a randomised, multicentre trial. Lancet. 2002 Jan 5;359(9300):23-9. link to original article PubMed
  6. TIKI: Heitink-Pollé KMJ, Uiterwaal CSPM, Porcelijn L, Tamminga RYJ, Smiers FJ, van Woerden NL, Wesseling J, Vidarsson G, Laarhoven AG, de Haas M, Bruin MCA; TIKI Investigators. Intravenous immunoglobulin vs observation in childhood immune thrombocytopenia: a randomized controlled trial. Blood. 2018 Aug 30;132(9):883-891. Epub 2018 Jun 26. link to original article PubMed

Mycophenolate mofetil & Prednisolone

Regimen

Study Evidence Comparator Efficacy
Bradbury et al. 2021 (FLIGHT) Phase 3 (E-esc) 1. Dexamethasone
2. Prednisolone
Superior treatment failure

References

  1. FLIGHT: Bradbury CA, Pell J, Hill Q, Bagot C, Cooper N, Ingram J, Breheny K, Kandiyali R, Rayment R, Evans G, Talks K, Thomas I, Greenwood R. Mycophenolate Mofetil for First-Line Treatment of Immune Thrombocytopenia. N Engl J Med. 2021 Sep 2;385(10):885-895. link to original article PubMed NCT03156452

Prednisone monotherapy

Regimen variant #1

Study Evidence Comparator Efficacy
Wei et al. 2015 Phase 3 (C) Dexamethasone Inferior CR rate

Immunosuppressive therapy

  • Prednisone (Sterapred) as follows:
    • Days 1 to 28: 1 mg/kg/day PO
    • Day 29 onwards: tapered over 4 to 6 weeks with a goal of maintenance dosing less than 15 mg/day or "complete termination"
      • Taper schedule determined by treating physician


Regimen variant #2

Study Evidence Comparator Efficacy
Matschke et al. 2013 Phase 3 (C) Dexamethasone Seems to have inferior responding patients maintaining remission for at least 6 mo

Immunosuppressive therapy

  • Prednisone (Sterapred) as follows:
    • Weeks 1 to 2: 1 mg/kg/day PO
    • Weeks 3 to 19: Tapered over 19 weeks with a goal of maintenance dosing less than 7.5 mg/day at the end of week 19

References

  1. Blanchette VS, Luke B, Andrew M, Sommerville-Nielsen S, Barnard D, de Veber B, Gent M. A prospective, randomized trial of high-dose intravenous immune globulin G therapy, oral prednisone therapy, and no therapy in childhood acute immune thrombocytopenic purpura. J Pediatr. 1993 Dec;123(6):989-95. link to original article PubMed
  2. Blanchette V, Imbach P, Andrew M, Adams M, McMillan J, Wang E, Milner R, Ali K, Barnard D, Bernstein M, Esseltine D, Chan KW, de Veber B, Israels S, Kobrinsky N, Luke B. Randomised trial of intravenous immunoglobulin G, intravenous anti-D, and oral prednisone in childhood acute immune thrombocytopenic purpura. Lancet. 1994 Sep 10;344(8924):703-7. link to original article PubMed
  3. Abstract: Johannes Matschke, Hannes Müller-Beißenhirtz, Ilona Vester, Bernd Hertenstein, Lewin Eisele, Hildegard Lax, Claudia Ose, Ulrich Dührsen. A Randomized Trial Of Daily Prednisone Versus Pulsed Dexamethasone In Treatment Naïve Patients With Idiopathic Thrombocytopenic Purpura. Blood Nov 2013,122(21)325. link to abstract
  4. Wei Y, Ji XB, Wang YW, Wang JX, Yang EQ, Wang ZC, Sang YQ, Bi ZM, Ren CA, Zhou F, Liu GQ, Peng J, Hou M. High-dose dexamethasone vs prednisone for treatment of adult immune thrombocytopenia: a prospective multicenter randomized trial. Blood. 2016 Jan 21;127(3):296-302. Epub 2015 Oct 19. link to original article contains dosing details in manuscript PubMed

RhIG monotherapy

RhIG: Rho(D) Immune Globulin

Regimen variant #1, 25 mcg/kg

Study Evidence
Bussel et al. 1991 Phase 2

Eligibility criteria

  • RhD-positive

Supportive therapy

4-day course


Regimen variant #2, 50 mcg/kg

Study Evidence Comparator Efficacy
Newman et al. 2001 Randomized Phase 2, <20 pts (C) Rho(D); 75 mcg/kg Seems to have inferior platelet effect

Eligibility criteria

  • RhD-positive, adult, HIV-negative, non-splenectomized, platelet count less than or equal to 30 x 109/L

Supportive therapy

Supportive therapy

One dose; can be repeated if required to increase platelet count


Regimen variant #3, 75 mcg/kg

Study Evidence Comparator Efficacy
Newman et al. 2001 Randomized Phase 2, <20 pts (E-esc) Rho(D); 50 mcg/kg Seems to have superior platelet effect

Eligibility criteria

  • RhD-positive, adult, HIV-negative, non-splenectomized, platelet count less than or equal to 30 x 109/L

Supportive therapy

Supportive therapy

One dose; can be repeated if required to increase platelet count

References

  1. Bussel JB, Graziano JN, Kimberly RP, Pahwa S, Aledort LM. Intravenous anti-D treatment of immune thrombocytopenic purpura: analysis of efficacy, toxicity, and mechanism of effect. Blood. 1991 May 1;77(9):1884-93. link to original article contains dosing details in manuscript PubMed
  2. Newman GC, Novoa MV, Fodero EM, Lesser ML, Woloski BM, Bussel JB. A dose of 75 microg/kg/day of iv anti-D increases the platelet count more rapidly and for a longer period of time than 50 microg/kg/day in adults with immune thrombocytopenic purpura. Br J Haematol. 2001 Mar;112(4):1076-8. link to original article contains dosing details in manuscript PubMed

TT4

TT4: Triple Therapy (4?)

Regimen

Study Evidence
Choi et al. 2015 Phase 2

Note: The authors do not specify whether modified or non-modified cyclosporine was used in this protocol. Please contact them for clarifications.

Immunosuppressive therapy

References

  1. Choi PY, Roncolato F, Badoux X, Ramanathan S, Ho SJ, Chong BH. A novel triple therapy for ITP using high-dose dexamethasone, low-dose rituximab, and cyclosporine (TT4). Blood. 2015 Jul 23;126(4):500-3. Epub 2015 May 13. link to original article contains dosing details in abstract link to PMC article PubMed

Relapsed or refractory

ATRA & Danazol

Regimen

Study Evidence Comparator Efficacy
Feng et al. 2017 (U1111-1132-6877) Randomized Phase 2 (E-esc) Danazol Superior 12-month sustained response

Targeted therapy

Endocrine therapy

16-week course

References

  1. U1111-1132-6877: Feng FE, Feng R, Wang M, Zhang JM, Jiang H, Jiang Q, Lu J, Liu H, Peng J, Hou M, Shen JL, Wang JW, Xu LP, Liu KY, Huang XJ, Zhang XH. Oral all-trans retinoic acid plus danazol versus danazol as second-line treatment in adults with primary immune thrombocytopenia: a multicentre, randomised, open-label, phase 2 trial. Lancet Haematol. 2017 Oct;4(10):e487-e496. Epub 2017 Sep 13. link to original article contains dosing details in abstract PubMed NCT01667263

Avatrombopag monotherapy

Regimen

Study Evidence Comparator Efficacy
Jurczak et al. 2018 (E5501-G000-302) Phase 3 (E-esc) Placebo Superior cumulative weeks of platelet response

Growth factor therapy

References

  1. Bussel JB, Kuter DJ, Aledort LM, Kessler CM, Cuker A, Pendergrass KB, Tang S, McIntosh J. A randomized trial of avatrombopag, an investigational thrombopoietin-receptor agonist, in persistent and chronic immune thrombocytopenia. Blood. 2014 Jun 19;123(25):3887-94. Epub 2014 May 6. link to original article PubMed
  2. E5501-G000-302: Jurczak W, Chojnowski K, Mayer J, Krawczyk K, Jamieson BD, Tian W, Allen LF. Phase 3 randomised study of avatrombopag, a novel thrombopoietin receptor agonist for the treatment of chronic immune thrombocytopenia. Br J Haematol. 2018 Nov;183(3):479-490. Epub 2018 Sep 7. link to original article PubMed

Cyclosporine monotherapy

Regimen

Study Evidence
Emilia et al. 2002 Pilot, <20 pts

Note: The authors do not specify whether modified or non-modified cyclosporine was used in this protocol. Please contact them for clarifications.

Immunosuppressive therapy

  • Cyclosporine A as follows:
    • Days 1 to 6: 5 mg/kg/day PO split into twice per day dosing
    • Day 7 onwards: 2.5 to 3 mg/kg/day PO with dose adjusted for target trough of 200 to 400 mcg/L

References

  1. Emilia G, Morselli M, Luppi M, Longo G, Marasca R, Gandini G, Ferrara L, D'Apollo N, Potenza L, Bertesi M, Torelli G. Long-term salvage therapy with cyclosporin A in refractory idiopathic thrombocytopenic purpura. Blood. 2002 Feb 15;99(4):1482-5. link to original article contains dosing details in manuscript PubMed

Danazol monotherapy

Regimen

Study Evidence Comparator Efficacy
Ahn et al. 1983 Pilot, >20 pts
Feng et al. 2017 (U1111-1132-6877) Randomized Phase 2 (C) ATRA & Danazol Inferior 12-month sustained response

Note: Ahn et al. 1983 gave a range of 200 mg PO from two to four times per day; the dose below is from U1111-1132-6877

Endocrine therapy

16-week course

References

  1. Ahn YS, Harrington WJ, Simon SR, Mylvaganam R, Pall LM, So AG. Danazol for the treatment of idiopathic thrombocytopenic purpura. N Engl J Med. 1983 Jun 9;308(23):1396-9. link to original article contains dosing details in manuscript PubMed
  2. U1111-1132-6877: Feng FE, Feng R, Wang M, Zhang JM, Jiang H, Jiang Q, Lu J, Liu H, Peng J, Hou M, Shen JL, Wang JW, Xu LP, Liu KY, Huang XJ, Zhang XH. Oral all-trans retinoic acid plus danazol versus danazol as second-line treatment in adults with primary immune thrombocytopenia: a multicentre, randomised, open-label, phase 2 trial. Lancet Haematol. 2017 Oct;4(10):e487-e496. Epub 2017 Sep 13. link to original article contains dosing details in abstract PubMed NCT01667263

Dexamethasone monotherapy

Regimen

Study Evidence
Andersen 1994 Pilot, <20 pts

Immunosuppressive therapy

28-day cycles for 6 cycles

References

  1. Andersen JC. Response of resistant idiopathic thrombocytopenic purpura to pulsed high-dose dexamethasone therapy. N Engl J Med. 1994 Jun 2;330(22):1560-4. link to original article contains dosing details in manuscript PubMed

Dexamethasone & Rituximab

Regimen ("R+3Dex")

Study Evidence
Imahiyerobo et al. 2013 Retrospective

Immunosuppressive therapy

14-day cycle for 3 cycles

References

  1. Abstract: Allison Imahiyerobo, Micha Thompson, Marina Izak Karaev, Waleed Ghanima, James B Bussel. Rituximab Combined With Three Cycles Of High Dose Dexamethasone Provides a Long Term Response Rate Similar To That Of Splenectomy In Patients With Immune Thrombocytopenia (ITP) Of Duration Less Than 2 Years. Blood Nov 2013,122(21)2310. link to abstract

Eltrombopag monotherapy

Regimen variant #1

Study Evidence Comparator Efficacy
Grainger et al. 2015 (PETIT2) Phase 3 (E-esc) Placebo Superior durable platelet response

Note: This regimen was intended for pediatric patients.

Growth factor therapy

  • Eltrombopag (Promacta) with starting dose by the following criteria:
    • Age 6 to 17, weighing at least 27kg, non-east Asian: 50 mg PO once per day
    • Age 6 to 17, weighing at least 27kg, east Asian: 25 mg PO once per day
    • Age 6 to 17, weighing less than 27kg, non-east Asian: 37.5 mg PO once per day
    • Age 6 to 17, weighing less than 27kg, east Asian: 25 mg PO once per day
    • Age 1 to 5, non-east Asian: 1.2 mg/kg/day oral suspension
    • Age 1 to 5, east Asian: 0.8 mg/kg/day oral suspension

Dose adjusted to a maximum of 75 mg/day, with temporary discontinuation for platelet count greater than 400 x 109/L


Regimen variant #2

Study Evidence Comparator Efficacy
Bussel et al. 2009 (TRA100773) Phase 3 (E-esc) Placebo Superior plt count of greater than or equal to 50 x 109/L on day 43

Growth factor therapy

The dose could be increased from 50 mg to 75 mg after 3 weeks in patients whose platelet counts were less than 50 x 109/L. Treatment was discontinued in patients who attained a platelet count greater than 200 x 109/L


Regimen variant #3

Study Evidence Comparator Efficacy
Cheng et al. 2010 (RAISE-ITP) Phase 3 (E-esc) Placebo Superior RR

Note: this trial should not be confused with the trial by the same name in colorectal cancer.

Growth factor therapy

  • Eltrombopag (Promacta) 50 mg PO once per day, with dose modifications:
    • Increase to 75 mg PO once per day allowed after day 22 for patients with platelet counts lower than 50 x 109/L
    • Decrease to 25 mg PO once per day required for platelets counts between 200 and 400 x 109/L
    • Drug was held for platelet count greater than 400 x 109/L, until platelet count dropped below 150 x 109/L

6-month course


Regimen variant #4

Study Evidence Comparator Efficacy
Bussel et al. 2007 (TRA100773) Phase 3 (E-esc) Placebo Superior RR

Growth factor therapy

Up to 6-week course


Regimen variant #5

Study Evidence
Saleh et al. 2012 (EXTEND) Phase 2

Growth factor therapy

Dose and schedule were individualized with the goal of achieving and maintaining platelets between 50 and 200 x 109/L.

References

  1. TRA100773: Bussel JB, Cheng G, Saleh MN, Psaila B, Kovaleva L, Meddeb B, Kloczko J, Hassani H, Mayer B, Stone NL, Arning M, Provan D, Jenkins JM. Eltrombopag for the treatment of chronic idiopathic thrombocytopenic purpura. N Engl J Med. 2007 Nov 29;357(22):2237-47. link to original article PubMed NCT00102739
  2. TRA100773: Bussel JB, Provan D, Shamsi T, Cheng G, Psaila B, Kovaleva L, Salama A, Jenkins JM, Roychowdhury D, Mayer B, Stone N, Arning M. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet. 2009 Feb 21;373(9664):641-8. link to original article contains dosing details in manuscript PubMed NCT00102739
  3. RAISE: Cheng G, Saleh MN, Marcher C, Vasey S, Mayer B, Aivado M, Arning M, Stone NL, Bussel JB. Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomised, phase 3 study. Lancet. 2011 Jan 29;377(9763):393-402. Epub 2010 Aug 23. Erratum in: Lancet. 2011 Jan 29;377(9763):382. link to original article contains dosing details in manuscript PubMed NCT00370331
  4. EXTEND: Saleh MN, Bussel JB, Cheng G, Meyer O, Bailey CK, Arning M, Brainsky A; EXTEND Study Group. Safety and efficacy of eltrombopag for treatment of chronic immune thrombocytopenia: results of the long-term, open-label EXTEND study. Blood. 2013 Jan 17;121(3):537-45. Epub 2012 Nov 20. link to original article contains dosing details in manuscript PubMed
  5. PETIT2: Grainger JD, Locatelli F, Chotsampancharoen T, Donyush E, Pongtanakul B, Komvilaisak P, Sosothikul D, Drelichman G, Sirachainan N, Holzhauer S, Lebedev V, Lemons R, Pospisilova D, Ramenghi U, Bussel JB, Bakshi KK, Iyengar M, Chan GW, Chagin KD, Theodore D, Marcello LM, Bailey CK. Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomised, multicentre, placebo-controlled trial. Lancet. 2015 Oct 24;386(10004):1649-58. Epub 2015 Jul 28. link to original article contains dosing details in manuscript PubMed NCT01520909
  6. Yang R, Li J, Jin J, Huang M, Yu Z, Xu X, Zhang X, Hou M. Multicentre, randomised phase III study of the efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia. Br J Haematol. 2017 Jan;176(1):101-110. link to original article PubMed

Fostamatinib monotherapy

Regimen

Study Years of enrollment Evidence Comparator Comparative Efficacy
Bussel et al. 2018 (FIT1) 2014-2016 Phase 3 (E-esc) Placebo Superior stable response
Bussel et al. 2018 (FIT2) 2015-2016 Phase 3 (E-esc) Placebo Superior stable response

Targeted therapy

  • Fostamatinib (Tavalisse) 100 mg PO twice per day
    • Dose may be increased to 150 mg PO twice per day after 4 weeks if needed to achieve platelet count of at least 50

24-week course

References

  1. FIT1: Bussel J, Arnold DM, Grossbard E, Mayer J, Treliński J, Homenda W, Hellmann A, Windyga J, Sivcheva L, Khalafallah AA, Zaja F, Cooper N, Markovtsov V, Zayed H, Duliege AM. Fostamatinib for the treatment of adult persistent and chronic immune thrombocytopenia: results of two phase 3, randomized, placebo-controlled trials. Am J Hematol. 2018 Jul;93(7):921-930. Epub 2018 May 15. link to original article link to PMC article PubMed NCT02076399
  2. FIT2: Bussel J, Arnold DM, Grossbard E, Mayer J, Treliński J, Homenda W, Hellmann A, Windyga J, Sivcheva L, Khalafallah AA, Zaja F, Cooper N, Markovtsov V, Zayed H, Duliege AM. Fostamatinib for the treatment of adult persistent and chronic immune thrombocytopenia: results of two phase 3, randomized, placebo-controlled trials. Am J Hematol. 2018 Jul;93(7):921-930. Epub 2018 May 15. link to original article link to PMC article PubMed NCT02076412
  3. Review: Newland A, Lee EJ, McDonald V, Bussel JB. Fostamatinib for persistent/chronic adult immune thrombocytopenia. Immunotherapy. 2018 Jan;10(1):9-25. link to original article PubMed

Mycophenolate mofetil monotherapy

Regimen

Study Evidence
Taylor et al. 2015 Retrospective

Immunosuppressive therapy

Continued indefinitely

References

  1. Retrospective: Taylor A, Neave L, Solanki S, Westwood JP, Terrinonive I, McGuckin S, Kothari J, Cooper N, Stasi R, Scully M. Mycophenolate mofetil therapy for severe immune thrombocytopenia. Br J Haematol. 2015 Nov;171(4):625-30. Epub 2015 Aug 6. link to original article PubMed

Placebo

Regimen

Study Evidence Comparator Efficacy
Kuter et al. 2008 (Amgen 20030105) Phase 3 (C) Romiplostim Inferior durable platelet response
Kuter et al. 2008 (Amgen 20030212) Phase 3 (C) Romiplostim Inferior durable platelet response
Bussel et al. 2009 (TRA100773) Phase 3 (C) Eltrombopag Inferior RR
Cheng et al. 2010 (RAISE-ITP) Phase 3 (C) Eltrombopag Inferior RR
Ghanima et al. 2015 (RITP) Phase 3 (C) Rituximab Did not meet primary endpoint of rate of treatment failure within 78 weeks
Grainger et al. 2015 (PETIT2) Phase 3 (C) Eltrombopag Inferior durable platelet response
Tarantino et al. 2016 (Amgen 20080279) Phase 3 (C) Romiplostim Inferior durable platelet response

No active treatment; used as a comparator arm and here for reference purposes only. Note that RAISE should not be confused with the trial by the same name in colorectal cancer.

References

  1. Amgen 20030105: Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrère F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. link to original article PubMed NCT00102323
  2. Amgen 20030212: Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrère F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. link to original article contains dosing details in manuscript PubMed NCT00102336
  3. TRA100773: Bussel JB, Provan D, Shamsi T, Cheng G, Psaila B, Kovaleva L, Salama A, Jenkins JM, Roychowdhury D, Mayer B, Stone N, Arning M. Effect of eltrombopag on platelet counts and bleeding during treatment of chronic idiopathic thrombocytopenic purpura: a randomised, double-blind, placebo-controlled trial. Lancet. 2009 Feb 21;373(9664):641-8. link to original article contains dosing details in manuscript PubMed NCT00102739
  4. RAISE: Cheng G, Saleh MN, Marcher C, Vasey S, Mayer B, Aivado M, Arning M, Stone NL, Bussel JB. Eltrombopag for management of chronic immune thrombocytopenia (RAISE): a 6-month, randomised, phase 3 study. Lancet. 2011 Jan 29;377(9763):393-402. Epub 2010 Aug 23. Erratum in: Lancet. 2011 Jan 29;377(9763):382. link to original article contains dosing details in manuscript PubMed NCT00370331
  5. RITP: Ghanima W, Khelif A, Waage A, Michel M, Tjønnfjord GE, Romdhan NB, Kahrs J, Darne B, Holme PA; RITP study group. Rituximab as second-line treatment for adult immune thrombocytopenia (the RITP trial): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet. 2015 Apr 25;385(9978):1653-61. Epub 2015 Feb 4. link to original article contains dosing details in abstract PubMed NCT00344149
  6. PETIT2: Grainger JD, Locatelli F, Chotsampancharoen T, Donyush E, Pongtanakul B, Komvilaisak P, Sosothikul D, Drelichman G, Sirachainan N, Holzhauer S, Lebedev V, Lemons R, Pospisilova D, Ramenghi U, Bussel JB, Bakshi KK, Iyengar M, Chan GW, Chagin KD, Theodore D, Marcello LM, Bailey CK. Eltrombopag for children with chronic immune thrombocytopenia (PETIT2): a randomised, multicentre, placebo-controlled trial. Lancet. 2015 Oct 24;386(10004):1649-58. Epub 2015 Jul 28. link to original article contains dosing details in manuscript PubMed NCT01520909
  7. Amgen 20080279: Tarantino MD, Bussel JB, Blanchette VS, Despotovic J, Bennett C, Raj A, Williams B, Beam D, Morales J, Rose MJ, Carpenter N, Nie K, Eisen M. Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study. Lancet. 2016 Jul 2;388(10039):45-54. Epub 2016 Apr 18. link to original article contains dosing details in manuscript PubMed NCT01444417
  8. Yang R, Li J, Jin J, Huang M, Yu Z, Xu X, Zhang X, Hou M. Multicentre, randomised phase III study of the efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia. Br J Haematol. 2017 Jan;176(1):101-110. link to original article PubMed

Rituximab monotherapy

Regimen

Study Evidence Comparator Efficacy
Godeau et al. 2008 Phase 2
Ghanima et al. 2015 (RITP) Phase 3 (E-esc) Placebo Did not meet primary endpoint of rate of treatment failure within 78 weeks

Patients in Godeau et al. 2008 had "ITP lasting 6 or more months before inclusion; at least 1 previous treatment for ITP; and platelet count less than 30 × 109/L at inclusion" and were candidates for splenectomy."

Immunosuppressive therapy

Supportive therapy

4-week course

References

  1. Godeau B, Porcher R, Fain O, Lefrère F, Fenaux P, Cheze S, Vekhoff A, Chauveheid MP, Stirnemann J, Galicier L, Bourgeois E, Haiat S, Varet B, Leporrier M, Papo T, Khellaf M, Michel M, Bierling P. Rituximab efficacy and safety in adult splenectomy candidates with chronic immune thrombocytopenic purpura: results of a prospective multicenter phase 2 study. Blood. 2008 Aug 15;112(4):999-1004. link to original article contains dosing details in manuscript PubMed
  2. Prospective cohort: Patel VL, Mahévas M, Lee SY, Stasi R, Cunningham-Rundles S, Godeau B, Kanter J, Neufeld E, Taube T, Ramenghi U, Shenoy S, Ward MJ, Mihatov N, Patel VL, Bierling P, Lesser M, Cooper N, Bussel JB. Outcomes 5 years after response to rituximab therapy in children and adults with immune thrombocytopenia. Blood. 2012 Jun 21;119(25):5989-95. link to original article contains dosing details in manuscript link to PMC article PubMed
  3. RITP: Ghanima W, Khelif A, Waage A, Michel M, Tjønnfjord GE, Romdhan NB, Kahrs J, Darne B, Holme PA; RITP study group. Rituximab as second-line treatment for adult immune thrombocytopenia (the RITP trial): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet. 2015 Apr 25;385(9978):1653-61. Epub 2015 Feb 4.link to original article contains dosing details in abstract PubMed NCT00344149

Romiplostim monotherapy

Regimen

Study Evidence Comparator Efficacy
Kuter et al. 2008 (Amgen 20030105) Phase 3 (E-esc) Placebo Superior durable platelet response
Kuter et al. 2008 (Amgen 20030212) Phase 3 (E-esc) Placebo Superior durable platelet response
Tarantino et al. 2016 (Amgen 20080279) Phase 3 (E-esc) Placebo Superior durable platelet response

Patients in Amgen 20030105 had undergone splenectomy. Patients in Amgen 20080279 were younger than 18 years old at time of study entry; see paper for details on dose titration.

Growth factor therapy

28-day cycles

References

  1. Amgen 20030105: Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrère F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. link to original article contains dosing details in manuscript PubMed NCT00102323
  2. Amgen 20030212: Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrère F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. link to original article contains dosing details in manuscript PubMed NCT00102336
  3. Amgen 20080279: Tarantino MD, Bussel JB, Blanchette VS, Despotovic J, Bennett C, Raj A, Williams B, Beam D, Morales J, Rose MJ, Carpenter N, Nie K, Eisen M. Romiplostim in children with immune thrombocytopenia: a phase 3, randomised, double-blind, placebo-controlled study. Lancet. 2016 Jul 2;388(10039):45-54. Epub 2016 Apr 18. link to original article contains dosing details in manuscript PubMed NCT01444417

Vinblastine-loaded platelets

Regimen

Study Evidence Efficacy
Ahn et al. 1978 Phase 1 CR in 6 of 11 patients

Reference

  1. Phase 1: Ahn YS, Byrnes JJ, Harrington WJ, Cayer ML, Smith DS, Brunskill DE, Pall LM. New England Journal of Medicine. 1978; 298:1101-1107. link to original article PubMed