Difference between revisions of "Inherited coagulopathy"

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*'''2020:''' Leebeek et al. [https://doi.org/10.1182/blood.2019000964 How I manage pregnancy in carriers of hemophilia and patients with von Willebrand disease]
 
*'''2020:''' Leebeek et al. [https://doi.org/10.1182/blood.2019000964 How I manage pregnancy in carriers of hemophilia and patients with von Willebrand disease]
 
*'''2019:''' Young. [https://doi.org/10.1111/bjh.15942 How I treat children with haemophilia and inhibitors]
 
*'''2019:''' Young. [https://doi.org/10.1111/bjh.15942 How I treat children with haemophilia and inhibitors]
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=Factor VIII deficiency (Hemophilia A), replacement products=
 
=Factor VIII deficiency (Hemophilia A), replacement products=
 
To be completed
 
To be completed
−
 
 
=Factor VIII deficiency (Hemophilia A), without inhibitors, all lines of therapy=
 
=Factor VIII deficiency (Hemophilia A), without inhibitors, all lines of therapy=
 
==Emicizumab monotherapy {{#subobject:c1a40e|Regimen=1}}==
 
==Emicizumab monotherapy {{#subobject:c1a40e|Regimen=1}}==
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<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen {{#subobject:cfdeed|Variant=1}}===
 
===Regimen {{#subobject:cfdeed|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
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|-
 
|-
 
|}
 
|}
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To be completed
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<div class="toccolours" style="background-color:#b3e2cd">
 
====Supportive therapy====
 
====Supportive therapy====
 
*[[Emicizumab (Hemlibra)]]
 
*[[Emicizumab (Hemlibra)]]
−
 
+
</div></div>
 
===References===
 
===References===
 
# '''HAVEN 3:''' Mahlangu J, Oldenburg J, Paz-Priel I, Negrier C, Niggli M, Mancuso ME, Schmitt C, Jiménez-Yuste V, Kempton C, Dhalluin C, Callaghan MU, Bujan W, Shima M, Adamkewicz JI, Asikanius E, Levy GG, Kruse-Jarres R. Emicizumab prophylaxis in patients who have hemophilia A without inhibitors. N Engl J Med. 2018 Aug 30;379(9):811-822. [https://doi.org/10.1056/NEJMoa1803550 link to original article] [https://pubmed.ncbi.nlm.nih.gov/30157389 PubMed]
 
# '''HAVEN 3:''' Mahlangu J, Oldenburg J, Paz-Priel I, Negrier C, Niggli M, Mancuso ME, Schmitt C, Jiménez-Yuste V, Kempton C, Dhalluin C, Callaghan MU, Bujan W, Shima M, Adamkewicz JI, Asikanius E, Levy GG, Kruse-Jarres R. Emicizumab prophylaxis in patients who have hemophilia A without inhibitors. N Engl J Med. 2018 Aug 30;379(9):811-822. [https://doi.org/10.1056/NEJMoa1803550 link to original article] [https://pubmed.ncbi.nlm.nih.gov/30157389 PubMed]
−
 
 
=Factor VIII deficiency (Hemophilia A), with inhibitors, all lines of therapy=
 
=Factor VIII deficiency (Hemophilia A), with inhibitors, all lines of therapy=
 
==Emicizumab monotherapy {{#subobject:f198c0|Regimen=1}}==
 
==Emicizumab monotherapy {{#subobject:f198c0|Regimen=1}}==
−
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen {{#subobject:cbc686|Variant=1}}===
 
===Regimen {{#subobject:cbc686|Variant=1}}===
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
 
{| class="wikitable sortable" style="width: 100%; text-align:center;"  
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|-
 
|-
 
|}
 
|}
−
To be completed
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<div class="toccolours" style="background-color:#b3e2cd">
 
====Supportive therapy====
 
====Supportive therapy====
 
*[[Emicizumab (Hemlibra)]]
 
*[[Emicizumab (Hemlibra)]]
−
 
+
</div></div>
 
===References===
 
===References===
 
# '''HAVEN 1:''' Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017 Aug 31;377(9):809-818. Epub 2017 Jul 10. [https://doi.org/10.1056/NEJMoa1703068 link to original article] [https://pubmed.ncbi.nlm.nih.gov/28691557 PubMed]
 
# '''HAVEN 1:''' Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017 Aug 31;377(9):809-818. Epub 2017 Jul 10. [https://doi.org/10.1056/NEJMoa1703068 link to original article] [https://pubmed.ncbi.nlm.nih.gov/28691557 PubMed]
 
# '''HAVEN 2:''' Young G, Liesner R, Chang T, Sidonio R, Oldenburg J, Jiménez-Yuste V, Mahlangu J, Kruse-Jarres R, Wang M, Uguen M, Doral MY, Wright LY, Schmitt C, Levy GG, Shima M, Mancuso ME. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019 Dec 12;134(24):2127-2138. [https://doi.org/10.1182/blood.2019001869 link to original article] [https://pubmed.ncbi.nlm.nih.gov/31697801 PubMed]
 
# '''HAVEN 2:''' Young G, Liesner R, Chang T, Sidonio R, Oldenburg J, Jiménez-Yuste V, Mahlangu J, Kruse-Jarres R, Wang M, Uguen M, Doral MY, Wright LY, Schmitt C, Levy GG, Shima M, Mancuso ME. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019 Dec 12;134(24):2127-2138. [https://doi.org/10.1182/blood.2019001869 link to original article] [https://pubmed.ncbi.nlm.nih.gov/31697801 PubMed]
−
 
 
==Rituximab monotherapy {{#subobject:6d79c2|Regimen=1}}==
 
==Rituximab monotherapy {{#subobject:6d79c2|Regimen=1}}==
−
 
+
<div class="toccolours" style="background-color:#eeeeee">
 
===Regimen {{#subobject:3f9925|Variant=1}}===
 
===Regimen {{#subobject:3f9925|Variant=1}}===
 
{| class="wikitable" style="width: 40%; text-align:center;"  
 
{| class="wikitable" style="width: 40%; text-align:center;"  
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!style="width: 25%"|[[Levels_of_Evidence#Evidence|Evidence]]
 
!style="width: 25%"|[[Levels_of_Evidence#Evidence|Evidence]]
 
|-
 
|-
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|[https://doi.org/10.1160/TH14-01-0078 Leissinger et al. 2014]
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|[https://www.ncbi.nlm.nih.gov/pmc/articles/pmc5726888/ Leissinger et al. 2014]
 
|style="background-color:#91cf61"|Phase 2
 
|style="background-color:#91cf61"|Phase 2
 
|-
 
|-
 
|}
 
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<div class="toccolours" style="background-color:#b3e2cd">
 
====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
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'''28-day course'''
 
'''28-day course'''
−
 
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</div></div>
 
===References===
 
===References===
 
# Kempton CL, Allen G, Hord J, Kruse-Jarres R, Pruthi RK, Walsh C, Young G, Soucie JM. Eradication of factor VIII inhibitors in patients with mild and moderate hemophilia A. Am J Hematol. 2012 Sep;87(9):933-6. [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3645919/ link to PMC article] '''does not contain dosing details'''  [https://pubmed.ncbi.nlm.nih.gov/22733686 PubMed]
 
# Kempton CL, Allen G, Hord J, Kruse-Jarres R, Pruthi RK, Walsh C, Young G, Soucie JM. Eradication of factor VIII inhibitors in patients with mild and moderate hemophilia A. Am J Hematol. 2012 Sep;87(9):933-6. [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3645919/ link to PMC article] '''does not contain dosing details'''  [https://pubmed.ncbi.nlm.nih.gov/22733686 PubMed]
−
# Leissinger C, Josephson CD, Granger S, Konkle BA, Kruse-Jarres R, Ragni MV, Journeycake JM, Valentino L, Key NS, Gill JC, McCrae KR, Neufeld EJ, Manno C, Raffini L, Saxena K, Torres M, Marder V, Bennett CM, Assmann SF. Rituximab for treatment of inhibitors in haemophilia A: a phase II study. Thromb Haemost. 2014 Sep 2;112(3):445-58. [https://doi.org/10.1160/TH14-01-0078 link to original article] [https://pubmed.ncbi.nlm.nih.gov/24919980 PubMed]
+
# Leissinger C, Josephson CD, Granger S, Konkle BA, Kruse-Jarres R, Ragni MV, Journeycake JM, Valentino L, Key NS, Gill JC, McCrae KR, Neufeld EJ, Manno C, Raffini L, Saxena K, Torres M, Marder V, Bennett CM, Assmann SF. Rituximab for treatment of inhibitors in haemophilia A: a phase II study. Thromb Haemost. 2014 Sep 2;112(3):445-58. [https://doi.org/10.1160/TH14-01-0078 link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/pmc5726888/ link to PMC article] [https://pubmed.ncbi.nlm.nih.gov/24919980 PubMed]
−
 
 
 
=Factor IX deficiency (Hemophilia B), all lines of therapy=
 
=Factor IX deficiency (Hemophilia B), all lines of therapy=
 
To be completed
 
To be completed
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[[Category:Inherited coagulopathy regimens]]
 
[[Category:Inherited coagulopathy regimens]]
 
[[Category:Disease-specific pages]]
 
[[Category:Disease-specific pages]]
 
[[Category:Bleeding disorders]]
 
[[Category:Bleeding disorders]]
 
[[Category:Clinical pharmacology]]
 
[[Category:Clinical pharmacology]]

Revision as of 14:57, 26 February 2023

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Section editor transclusions

3 regimens on this page
3 variants on this page


Guidelines

ASH/ISTH/NHF/WFH

BSH

"How I treat"

Factor VIII deficiency (Hemophilia A), replacement products

To be completed

Factor VIII deficiency (Hemophilia A), without inhibitors, all lines of therapy

Emicizumab monotherapy

Regimen

Study Evidence Comparator Comparative Efficacy
Mahlangu et al. 2018 (HAVEN 3) Phase 3 (E-esc) No prophylaxis Lower bleeding rate

Supportive therapy

References

  1. HAVEN 3: Mahlangu J, Oldenburg J, Paz-Priel I, Negrier C, Niggli M, Mancuso ME, Schmitt C, Jiménez-Yuste V, Kempton C, Dhalluin C, Callaghan MU, Bujan W, Shima M, Adamkewicz JI, Asikanius E, Levy GG, Kruse-Jarres R. Emicizumab prophylaxis in patients who have hemophilia A without inhibitors. N Engl J Med. 2018 Aug 30;379(9):811-822. link to original article PubMed

Factor VIII deficiency (Hemophilia A), with inhibitors, all lines of therapy

Emicizumab monotherapy

Regimen

Study Evidence Comparator Comparative Efficacy
Oldenburg et al. 2017 (HAVEN 1) Phase 3 (E-esc) No prophylaxis Lower bleeding rate

Supportive therapy

References

  1. HAVEN 1: Oldenburg J, Mahlangu JN, Kim B, Schmitt C, Callaghan MU, Young G, Santagostino E, Kruse-Jarres R, Negrier C, Kessler C, Valente N, Asikanius E, Levy GG, Windyga J, Shima M. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017 Aug 31;377(9):809-818. Epub 2017 Jul 10. link to original article PubMed
  2. HAVEN 2: Young G, Liesner R, Chang T, Sidonio R, Oldenburg J, Jiménez-Yuste V, Mahlangu J, Kruse-Jarres R, Wang M, Uguen M, Doral MY, Wright LY, Schmitt C, Levy GG, Shima M, Mancuso ME. A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019 Dec 12;134(24):2127-2138. link to original article PubMed

Rituximab monotherapy

Regimen

Study Evidence
Leissinger et al. 2014 Phase 2

Immunosuppressive therapy

28-day course

References

  1. Kempton CL, Allen G, Hord J, Kruse-Jarres R, Pruthi RK, Walsh C, Young G, Soucie JM. Eradication of factor VIII inhibitors in patients with mild and moderate hemophilia A. Am J Hematol. 2012 Sep;87(9):933-6. link to PMC article does not contain dosing details PubMed
  2. Leissinger C, Josephson CD, Granger S, Konkle BA, Kruse-Jarres R, Ragni MV, Journeycake JM, Valentino L, Key NS, Gill JC, McCrae KR, Neufeld EJ, Manno C, Raffini L, Saxena K, Torres M, Marder V, Bennett CM, Assmann SF. Rituximab for treatment of inhibitors in haemophilia A: a phase II study. Thromb Haemost. 2014 Sep 2;112(3):445-58. link to original article link to PMC article PubMed

Factor IX deficiency (Hemophilia B), all lines of therapy

To be completed

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