Difference between revisions of "Aplastic anemia"

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m (Text replacement - "<span style="background:#eeee00; padding:3px 6px 3px 6px; border-color:black; border-width:2px; border-style:solid;">Phase II</span>" to "style="background-color:#EEEE00"|Phase II")
m
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|[[Levels_of_Evidence#Evidence|'''Evidence''']]
 
|[[Levels_of_Evidence#Evidence|'''Evidence''']]
 
|'''Comparator'''
 
|'''Comparator'''
 +
|[[Levels_of_Evidence#Efficacy|'''Efficacy''']]
 
|-
 
|-
 
|[http://www.bloodjournal.org/content/93/7/2191.long Marsh et al. 1999]
 
|[http://www.bloodjournal.org/content/93/7/2191.long Marsh et al. 1999]
 
|style="background-color:#00CD00"|Phase III
 
|style="background-color:#00CD00"|Phase III
 
|[[Aplastic_anemia#Cyclosporine|Cyclosporine]]
 
|[[Aplastic_anemia#Cyclosporine|Cyclosporine]]
 +
|style="background-color:#00CD00"|Seems to have superior ORR
 
|-
 
|-
 
|[http://www.bloodjournal.org/content/110/6/1756.long Teramura et al. 2007]
 
|[http://www.bloodjournal.org/content/110/6/1756.long Teramura et al. 2007]
 
|style="background-color:#00CD00"|Phase III
 
|style="background-color:#00CD00"|Phase III
 
|[[Aplastic_anemia#ATG_.28Horse.29.2C_Cyclosporine.2C_G-CSF|ATG (Horse), Cyclosporine, G-CSF]]
 
|[[Aplastic_anemia#ATG_.28Horse.29.2C_Cyclosporine.2C_G-CSF|ATG (Horse), Cyclosporine, G-CSF]]
 +
|style="background-color:#ff0000"|Seems to have inferior hematologic response rate at 6 months
 
|-
 
|-
 
|[http://www.haematologica.org/content/94/3/348.long Scheinberg et al. 2009]
 
|[http://www.haematologica.org/content/94/3/348.long Scheinberg et al. 2009]
 
|style="background-color:#00CD00"|Phase III
 
|style="background-color:#00CD00"|Phase III
 
|[[Aplastic_anemia#ATG_.28Horse.29.2C_Cyclosporine.2C_Sirolimus|ATG (Horse), Cyclosporine, Sirolimus]]
 
|[[Aplastic_anemia#ATG_.28Horse.29.2C_Cyclosporine.2C_Sirolimus|ATG (Horse), Cyclosporine, Sirolimus]]
 +
|style="background-color:#eeee00"|Seems not superior
 
|-
 
|-
 
|[http://www.bloodjournal.org/content/117/17/4434.long Tichelli et al. 2011]
 
|[http://www.bloodjournal.org/content/117/17/4434.long Tichelli et al. 2011]
 
|style="background-color:#00CD00"|Phase III
 
|style="background-color:#00CD00"|Phase III
 
|[[Aplastic_anemia#ATG_.28Horse.29.2C_Cyclosporine.2C_G-CSF|ATG (Horse), Cyclosporine, G-CSF]]
 
|[[Aplastic_anemia#ATG_.28Horse.29.2C_Cyclosporine.2C_G-CSF|ATG (Horse), Cyclosporine, G-CSF]]
 +
|style="background-color:#eeee00"|Seems not superior
 
|-
 
|-
 
|[http://www.nejm.org/doi/full/10.1056/NEJMoa1103975 Scheinberg et al. 2011]
 
|[http://www.nejm.org/doi/full/10.1056/NEJMoa1103975 Scheinberg et al. 2011]
 
|style="background-color:#00CD00"|Phase III
 
|style="background-color:#00CD00"|Phase III
 
|[[Aplastic_anemia#ATG_.28Rabbit.29_.26_Cyclosporine|ATG (Rabbit) & Cyclosporine]]
 
|[[Aplastic_anemia#ATG_.28Rabbit.29_.26_Cyclosporine|ATG (Rabbit) & Cyclosporine]]
 +
|style="background-color:#00CD00"|Superior OS
 
|-
 
|-
 
|}
 
|}
−
 
+
====Immunotherapy====
 
*[[Antithymocyte globulin, horse ATG (Atgam)]] 40 mg/kg IV once per day on days 1 to 4
 
*[[Antithymocyte globulin, horse ATG (Atgam)]] 40 mg/kg IV once per day on days 1 to 4
 
*Cyclosporine A (type not specified) as follows:
 
*Cyclosporine A (type not specified) as follows:
Line 51: Line 57:
 
**Dose adjusted to keep trough blood level of 200 to 400 ng/ml  
 
**Dose adjusted to keep trough blood level of 200 to 400 ng/ml  
  
−
Supportive medications:
+
====Supportive medications====
 
*[[Pentamidine (Nebupent)]] (dose not specified) once per month
 
*[[Pentamidine (Nebupent)]] (dose not specified) once per month
 
*[[Valacyclovir (Valtrex)]] (dose not specified) once per day
 
*[[Valacyclovir (Valtrex)]] (dose not specified) once per day
Line 79: Line 85:
  
 
''In the abstract, the authors state that all patients received "standard" ATG (horse) and cyclosporine, without further specification. The doses below are from the published papers for [[#ATG_.28Horse.29_.26_Cyclosporine|ATG (horse) & cyclosporine]]. There were also 3 cohorts of eltrombopag, none were mentioned as preferred.''
 
''In the abstract, the authors state that all patients received "standard" ATG (horse) and cyclosporine, without further specification. The doses below are from the published papers for [[#ATG_.28Horse.29_.26_Cyclosporine|ATG (horse) & cyclosporine]]. There were also 3 cohorts of eltrombopag, none were mentioned as preferred.''
−
 
+
====Therapy====
 
*[[Antithymocyte globulin, horse ATG (Atgam)]] 40 mg/kg IV once per day on days 1 to 4
 
*[[Antithymocyte globulin, horse ATG (Atgam)]] 40 mg/kg IV once per day on days 1 to 4
 
*Cyclosporine A (type not specified) as follows:
 
*Cyclosporine A (type not specified) as follows:
Line 89: Line 95:
 
**Cohort 3: 150 mg PO once per day starting on day 1 and continuing for 6 months
 
**Cohort 3: 150 mg PO once per day starting on day 1 and continuing for 6 months
  
−
Supportive medications:
+
====Supportive medications====
 
*[[Pentamidine (Nebupent)]] (dose not specified) once per month
 
*[[Pentamidine (Nebupent)]] (dose not specified) once per month
 
*[[Valacyclovir (Valtrex)]] (dose not specified) once per day   
 
*[[Valacyclovir (Valtrex)]] (dose not specified) once per day   
Line 107: Line 113:
 
|[[Levels_of_Evidence#Evidence|'''Evidence''']]
 
|[[Levels_of_Evidence#Evidence|'''Evidence''']]
 
|'''Comparator'''
 
|'''Comparator'''
 +
|[[Levels_of_Evidence#Efficacy|'''Efficacy''']]
 
|-
 
|-
 
|[http://www.bloodjournal.org/content/110/6/1756.long Teramura et al. 2007]
 
|[http://www.bloodjournal.org/content/110/6/1756.long Teramura et al. 2007]
 
|style="background-color:#00CD00"|Phase III
 
|style="background-color:#00CD00"|Phase III
 
|[[Aplastic_anemia#ATG_.28Horse.29_.26_Cyclosporine|ATG (Horse) & Cyclosporine]]
 
|[[Aplastic_anemia#ATG_.28Horse.29_.26_Cyclosporine|ATG (Horse) & Cyclosporine]]
 +
|style="background-color:#00cd00"|Seems to have superior hematologic response rate at 6 months
 
|-
 
|-
 
|[http://www.bloodjournal.org/content/117/17/4434.long Tichelli et al. 2011]
 
|[http://www.bloodjournal.org/content/117/17/4434.long Tichelli et al. 2011]
 
|style="background-color:#00CD00"|Phase III
 
|style="background-color:#00CD00"|Phase III
 
|[[Aplastic_anemia#ATG_.28Horse.29_.26_Cyclosporine|ATG (Horse) & Cyclosporine]]
 
|[[Aplastic_anemia#ATG_.28Horse.29_.26_Cyclosporine|ATG (Horse) & Cyclosporine]]
 +
|style="background-color:#eeee00"|Seems not superior
 
|-
 
|-
 
|}
 
|}
Line 134: Line 143:
 
|[[Levels_of_Evidence#Evidence|'''Evidence''']]
 
|[[Levels_of_Evidence#Evidence|'''Evidence''']]
 
|'''Comparator'''
 
|'''Comparator'''
 +
|[[Levels_of_Evidence#Efficacy|'''Efficacy''']]
 
|-
 
|-
 
|[http://www.haematologica.org/content/94/3/348.long Scheinberg et al. 2009]
 
|[http://www.haematologica.org/content/94/3/348.long Scheinberg et al. 2009]
 
|style="background-color:#00CD00"|Phase III
 
|style="background-color:#00CD00"|Phase III
 
|[[Aplastic_anemia#ATG_.28Horse.29_.26_Cyclosporine|ATG (Horse) & Cyclosporine]]
 
|[[Aplastic_anemia#ATG_.28Horse.29_.26_Cyclosporine|ATG (Horse) & Cyclosporine]]
 +
|style="background-color:#eeee00"|Seems not superior
 
|-
 
|-
 
|}
 
|}
 +
====Immunotherapy====
 
To be completed
 
To be completed
  
Line 156: Line 168:
 
|[[Levels_of_Evidence#Evidence|'''Evidence''']]
 
|[[Levels_of_Evidence#Evidence|'''Evidence''']]
 
|'''Comparator'''
 
|'''Comparator'''
 +
|[[Levels_of_Evidence#Efficacy|'''Efficacy''']]
 
|-
 
|-
 
|[http://www.nejm.org/doi/full/10.1056/NEJMoa1103975 Scheinberg et al. 2011]
 
|[http://www.nejm.org/doi/full/10.1056/NEJMoa1103975 Scheinberg et al. 2011]
 
|style="background-color:#00CD00"|Phase III
 
|style="background-color:#00CD00"|Phase III
 
|[[Aplastic_anemia#ATG_.28Horse.29_.26_Cyclosporine|ATG (Horse) & Cyclosporine]]
 
|[[Aplastic_anemia#ATG_.28Horse.29_.26_Cyclosporine|ATG (Horse) & Cyclosporine]]
 +
|style="background-color:#ff0000"|Inferior OS
 
|-
 
|-
 
|}
 
|}
−
 
+
====Immunotherapy====
 
*[[Antithymocyte globulin, rabbit ATG (Thymoglobulin)]] 3.5 mg/kg IV once per day on days 1 to 5
 
*[[Antithymocyte globulin, rabbit ATG (Thymoglobulin)]] 3.5 mg/kg IV once per day on days 1 to 5
 
*Cyclosporine A (type not specified) as follows:
 
*Cyclosporine A (type not specified) as follows:
Line 168: Line 182:
 
**Dose adjusted to keep trough blood level of 200 to 400 ng/ml  
 
**Dose adjusted to keep trough blood level of 200 to 400 ng/ml  
  
−
Supportive medications:
+
====Supportive medications====
 
*[[Pentamidine (Nebupent)]] (dose not specified) once per month
 
*[[Pentamidine (Nebupent)]] (dose not specified) once per month
 
*[[Valacyclovir (Valtrex)]] (dose not specified) once per day
 
*[[Valacyclovir (Valtrex)]] (dose not specified) once per day
Line 186: Line 200:
 
|[[Levels_of_Evidence#Evidence|'''Evidence''']]
 
|[[Levels_of_Evidence#Evidence|'''Evidence''']]
 
|'''Comparator'''
 
|'''Comparator'''
 +
|[[Levels_of_Evidence#Efficacy|'''Efficacy''']]
 
|-
 
|-
 
|[http://www.bloodjournal.org/content/93/7/2191.long Marsh et al. 1999]
 
|[http://www.bloodjournal.org/content/93/7/2191.long Marsh et al. 1999]
 
|style="background-color:#00CD00"|Phase III
 
|style="background-color:#00CD00"|Phase III
 
|[[Aplastic_anemia#ATG_.28Horse.29_.26_Cyclosporine|ATG (Horse) & Cyclosporine]]
 
|[[Aplastic_anemia#ATG_.28Horse.29_.26_Cyclosporine|ATG (Horse) & Cyclosporine]]
 +
|style="background-color:#00CD00"|Seems to have inferior ORR
 
|-
 
|-
 
|}
 
|}
 +
====Immunotherapy====
 
To be completed
 
To be completed
  
Line 217: Line 234:
 
|-
 
|-
 
|}
 
|}
 +
====Immunotherapy====
 
To be completed
 
To be completed
  
Line 238: Line 256:
 
|-
 
|-
 
|}
 
|}
−
 
+
====Growth factor therapy====
 
*[[Eltrombopag (Promacta)]] 50 mg PO once per day, increased by 25 mg every 2 weeks until platelet count > 20,000 per µL or maximum of 150 mg daily dose
 
*[[Eltrombopag (Promacta)]] 50 mg PO once per day, increased by 25 mg every 2 weeks until platelet count > 20,000 per µL or maximum of 150 mg daily dose
  
Line 244: Line 262:
 
# Olnes MJ, Scheinberg P, Calvo KR, Desmond R, Tang Y, Dumitriu B, Parikh AR, Soto S, Biancotto A, Feng X, Lozier J, Wu CO, Young NS, Dunbar CE. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012 Jul 5;367(1):11-9. Erratum in: N Engl J Med. 2012 Jul 19;367(3):284. [http://www.nejm.org/doi/full/10.1056/NEJMoa1200931 link to original article] '''contains verified protocol''' [https://www.ncbi.nlm.nih.gov/pubmed/22762314 PubMed]
 
# Olnes MJ, Scheinberg P, Calvo KR, Desmond R, Tang Y, Dumitriu B, Parikh AR, Soto S, Biancotto A, Feng X, Lozier J, Wu CO, Young NS, Dunbar CE. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012 Jul 5;367(1):11-9. Erratum in: N Engl J Med. 2012 Jul 19;367(3):284. [http://www.nejm.org/doi/full/10.1056/NEJMoa1200931 link to original article] '''contains verified protocol''' [https://www.ncbi.nlm.nih.gov/pubmed/22762314 PubMed]
 
## '''Update:''' Desmond R, Townsley DM, Dumitriu B, Olnes MJ, Scheinberg P, Bevans M, Parikh AR, Broder K, Calvo KR, Wu CO, Young NS, Dunbar CE. Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug. Blood. 2014 Mar 20;123(12):1818-25. Epub 2013 Dec 17. [http://bloodjournal.hematologylibrary.org/content/123/12/1818.full link to original article] '''contains verified protocol''' [https://www.ncbi.nlm.nih.gov/pubmed/24345753 PubMed]
 
## '''Update:''' Desmond R, Townsley DM, Dumitriu B, Olnes MJ, Scheinberg P, Bevans M, Parikh AR, Broder K, Calvo KR, Wu CO, Young NS, Dunbar CE. Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug. Blood. 2014 Mar 20;123(12):1818-25. Epub 2013 Dec 17. [http://bloodjournal.hematologylibrary.org/content/123/12/1818.full link to original article] '''contains verified protocol''' [https://www.ncbi.nlm.nih.gov/pubmed/24345753 PubMed]
 +
 +
[[Category:Benign hematology regimens]]

Revision as of 03:05, 25 December 2016

Use of this site is subject to you reading and agreeing with the terms set forth in the disclaimer.

Is there a regimen missing from this list? Would you like to share a different dosage/schedule or an additional reference for a regimen? Have you noticed an error? Do you have an idea that will help the site grow to better meet your needs and the needs of many others? You are invited to contribute to the site.

9 regimens on this page
12 variants on this page


Initial therapy

ATG (Horse) & Cyclosporine

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Regimen

Study Evidence Comparator Efficacy
Marsh et al. 1999 Phase III Cyclosporine Seems to have superior ORR
Teramura et al. 2007 Phase III ATG (Horse), Cyclosporine, G-CSF Seems to have inferior hematologic response rate at 6 months
Scheinberg et al. 2009 Phase III ATG (Horse), Cyclosporine, Sirolimus Seems not superior
Tichelli et al. 2011 Phase III ATG (Horse), Cyclosporine, G-CSF Seems not superior
Scheinberg et al. 2011 Phase III ATG (Rabbit) & Cyclosporine Superior OS

Immunotherapy

  • Antithymocyte globulin, horse ATG (Atgam) 40 mg/kg IV once per day on days 1 to 4
  • Cyclosporine A (type not specified) as follows:
    • Starting dose: 10 mg/kg in divided doses q12h (15 mg/kg for children younger than 12)
    • Dose adjusted to keep trough blood level of 200 to 400 ng/ml

Supportive medications

References

  1. Marsh J, Schrezenmeier H, Marin P, Ilhan O, Ljungman P, McCann S, Socie G, Tichelli A, Passweg J, Hows J, Raghavachar A, Locasciulli A, Bacigalupo A. Prospective randomized multicenter study comparing cyclosporin alone versus the combination of antithymocyte globulin and cyclosporin for treatment of patients with nonsevere aplastic anemia: a report from the European Blood and Marrow Transplant (EBMT) Severe Aplastic Anaemia Working Party. Blood. 1999 Apr 1;93(7):2191-5. link to original article PubMed
  2. Teramura M, Kimura A, Iwase S, Yonemura Y, Nakao S, Urabe A, Omine M, Mizoguchi H. Treatment of severe aplastic anemia with antithymocyte globulin and cyclosporin A with or without G-CSF in adults: a multicenter randomized study in Japan. Blood. 2007 Sep 15;110(6):1756-61. Epub 2007 May 25. link to original article PubMed
  3. Scheinberg P, Wu CO, Nunez O, Scheinberg P, Boss C, Sloand EM, Young NS. Treatment of severe aplastic anemia with a combination of horse antithymocyte globulin and cyclosporine, with or without sirolimus: a prospective randomized study. Haematologica. 2009 Mar;94(3):348-54. Epub 2009 Jan 30. link to original article PubMed
  4. Tichelli A, Schrezenmeier H, Socié G, Marsh J, Bacigalupo A, Dührsen U, Franzke A, Hallek M, Thiel E, Wilhelm M, Höchsmann B, Barrois A, Champion K, Passweg JR. A randomized controlled study in patients with newly diagnosed severe aplastic anemia receiving antithymocyte globulin (ATG), cyclosporine, with or without G-CSF: a study of the SAA Working Party of the European Group for Blood and Marrow Transplantation. Blood. 2011 Apr 28;117(17):4434-41. Epub 2011 Jan 13. link to original article PubMed
  5. Scheinberg P, Nunez O, Weinstein B, Scheinberg P, Biancotto A, Wu CO, Young NS. Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. N Engl J Med. 2011 Aug 4;365(5):430-8. link to original article PubMed

ATG (Horse), Cyclosporine, Eltrombopag

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Regimen

Study Evidence
Townsley et al. 2015 Phase II

In the abstract, the authors state that all patients received "standard" ATG (horse) and cyclosporine, without further specification. The doses below are from the published papers for ATG (horse) & cyclosporine. There were also 3 cohorts of eltrombopag, none were mentioned as preferred.

Therapy

  • Antithymocyte globulin, horse ATG (Atgam) 40 mg/kg IV once per day on days 1 to 4
  • Cyclosporine A (type not specified) as follows:
    • Starting dose: 10 mg/kg in divided doses q12h (15 mg/kg for children younger than 12)
    • Dose adjusted to keep trough blood level of 200 to 400 ng/ml
  • Eltrombopag (Promacta) on one of the following 3 schedules:
    • Cohort 1: 150 mg PO once per day starting on day 14 and continuing for 6 months
    • Cohort 2: 150 mg PO once per day starting on day 14 and continuing for 3 months
    • Cohort 3: 150 mg PO once per day starting on day 1 and continuing for 6 months

Supportive medications

References

  1. Abstract: Townsley et al. Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia Accelerates Count Recovery and Increases Response Rates. ASH 2015 Annual Meeting LBA-2 link to abstract

ATG (Horse), Cyclosporine, G-CSF

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Regimen

Study Evidence Comparator Efficacy
Teramura et al. 2007 Phase III ATG (Horse) & Cyclosporine Seems to have superior hematologic response rate at 6 months
Tichelli et al. 2011 Phase III ATG (Horse) & Cyclosporine Seems not superior

To be completed

References

  1. Teramura M, Kimura A, Iwase S, Yonemura Y, Nakao S, Urabe A, Omine M, Mizoguchi H. Treatment of severe aplastic anemia with antithymocyte globulin and cyclosporin A with or without G-CSF in adults: a multicenter randomized study in Japan. Blood. 2007 Sep 15;110(6):1756-61. Epub 2007 May 25. link to original article PubMed
  2. Tichelli A, Schrezenmeier H, Socié G, Marsh J, Bacigalupo A, Dührsen U, Franzke A, Hallek M, Thiel E, Wilhelm M, Höchsmann B, Barrois A, Champion K, Passweg JR. A randomized controlled study in patients with newly diagnosed severe aplastic anemia receiving antithymocyte globulin (ATG), cyclosporine, with or without G-CSF: a study of the SAA Working Party of the European Group for Blood and Marrow Transplantation. Blood. 2011 Apr 28;117(17):4434-41. Epub 2011 Jan 13. link to original article PubMed

ATG (Horse), Cyclosporine, Sirolimus

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Regimen

Study Evidence Comparator Efficacy
Scheinberg et al. 2009 Phase III ATG (Horse) & Cyclosporine Seems not superior

Immunotherapy

To be completed

References

  1. Scheinberg P, Wu CO, Nunez O, Scheinberg P, Boss C, Sloand EM, Young NS. Treatment of severe aplastic anemia with a combination of horse antithymocyte globulin and cyclosporine, with or without sirolimus: a prospective randomized study. Haematologica. 2009 Mar;94(3):348-54. Epub 2009 Jan 30. link to original article PubMed

ATG (Rabbit) & Cyclosporine

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Regimen

Study Evidence Comparator Efficacy
Scheinberg et al. 2011 Phase III ATG (Horse) & Cyclosporine Inferior OS

Immunotherapy

  • Antithymocyte globulin, rabbit ATG (Thymoglobulin) 3.5 mg/kg IV once per day on days 1 to 5
  • Cyclosporine A (type not specified) as follows:
    • Starting dose: 10 mg/kg in divided doses q12h (15 mg/kg for children younger than 12)
    • Dose adjusted to keep trough blood level of 200 to 400 ng/ml

Supportive medications

References

  1. Scheinberg P, Nunez O, Weinstein B, Scheinberg P, Biancotto A, Wu CO, Young NS. Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. N Engl J Med. 2011 Aug 4;365(5):430-8. link to original article PubMed

Cyclosporine

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Regimen

Study Evidence Comparator Efficacy
Marsh et al. 1999 Phase III ATG (Horse) & Cyclosporine Seems to have inferior ORR

Immunotherapy

To be completed

References

  1. Marsh J, Schrezenmeier H, Marin P, Ilhan O, Ljungman P, McCann S, Socie G, Tichelli A, Passweg J, Hows J, Raghavachar A, Locasciulli A, Bacigalupo A. Prospective randomized multicenter study comparing cyclosporin alone versus the combination of antithymocyte globulin and cyclosporin for treatment of patients with nonsevere aplastic anemia: a report from the European Blood and Marrow Transplant (EBMT) Severe Aplastic Anaemia Working Party. Blood. 1999 Apr 1;93(7):2191-5. link to original article PubMed

Relapsed/Refractory

ATG (Rabbit) & Cyclosporine

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Regimen

Study Evidence
Di Bona et al. 1999 Phase II
Scheinberg et al. 2006 Phase II

Immunotherapy

To be completed

References

  1. Di Bona E, Rodeghiero F, Bruno B, Gabbas A, Foa P, Locasciulli A, Rosanelli C, Camba L, Saracco P, Lippi A, Iori AP, Porta F, De Rossi G, Comotti B, Iacopino P, Dufour C, Bacigalupo A. Rabbit antithymocyte globulin (r-ATG) plus cyclosporine and granulocyte colony stimulating factor is an effective treatment for aplastic anaemia patients unresponsive to a first course of intensive immunosuppressive therapy. Gruppo Italiano Trapianto di Midollo Osseo (GITMO). Br J Haematol. 1999 Nov;107(2):330-4. Erratum in: Br J Haematol 2000 Feb;108(2):461. De Rossi V [corrected to De Rossi G]. link to original article PubMed
  2. Scheinberg P, Nunez O, Young NS. Retreatment with rabbit anti-thymocyte globulin and ciclosporin for patients with relapsed or refractory severe aplastic anaemia. Br J Haematol. 2006 Jun;133(6):622-7. link to original article PubMed

Eltrombopag (Promacta)

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Regimen

Study Evidence
Olnes et al. 2012 Phase II

Growth factor therapy

  • Eltrombopag (Promacta) 50 mg PO once per day, increased by 25 mg every 2 weeks until platelet count > 20,000 per µL or maximum of 150 mg daily dose

References

  1. Olnes MJ, Scheinberg P, Calvo KR, Desmond R, Tang Y, Dumitriu B, Parikh AR, Soto S, Biancotto A, Feng X, Lozier J, Wu CO, Young NS, Dunbar CE. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012 Jul 5;367(1):11-9. Erratum in: N Engl J Med. 2012 Jul 19;367(3):284. link to original article contains verified protocol PubMed
    1. Update: Desmond R, Townsley DM, Dumitriu B, Olnes MJ, Scheinberg P, Bevans M, Parikh AR, Broder K, Calvo KR, Wu CO, Young NS, Dunbar CE. Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug. Blood. 2014 Mar 20;123(12):1818-25. Epub 2013 Dec 17. link to original article contains verified protocol PubMed
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