Difference between revisions of "Inherited coagulopathy"

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!colspan="2" align="center" style="color:white; font-size:125%; background-color:#31a354"|'''Section editor'''
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[[#top|Back to Top]]
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|style="background-color:#F0F0F0; width:15%"|[[File:Tillman_Benjamin-2.jpg|frameless|upright=0.3|center]]
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</div>
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|style="width:35%"|<big>[[User:Benjamintillman|Benjamin Tillman, MD]]<br>Vanderbilt University<br>Nashville, TN</big>
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{{#lst:Editorial board transclusions|heme}}
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*''We have moved [[How I Treat]] articles to a dedicated page.''
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{| class="wikitable" style="float:right; margin-right: 5px;"
 
{| class="wikitable" style="float:right; margin-right: 5px;"
 
|-
 
|-
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|<div style="background-color: #fee0d1; border: 1px solid #808000; padding: 5px; {{border-radius|16px}}" align="right"><font size="4"><b>{{#ask: [[-Has subobject::{{FULLPAGENAME}}]] |?Regimen |limit=10000|format=sum}} [[Tutorial#Regimens|regimens]] on this page</b></font></div>
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|<div style="background-color: #fee0d1; border: 1px solid #808000; padding: 5px; {{border-radius|16px}}" align="right"><font size="4"><b>{{#ask: [[-Has subobject::{{FULLPAGENAME}}]] |?Regimen |limit=10000|format=sum}} [[Tutorial#Regimens|regimens]] on this page</b></font></div>
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<div style="background-color: #deebf6; border: 1px solid #808000; padding: 5px; {{border-radius|16px}}"><font size="4"><b>{{#ask: [[-Has subobject::{{FULLPAGENAME}}]] |?Variant |limit=10000|format=sum}} [[Tutorial#Variants|variants]] on this page</b></font></div>
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<div style="background-color: #deebf6; border: 1px solid #808000; padding: 5px; {{border-radius|16px}}"><font size="4"><b>{{#ask: [[-Has subobject::{{FULLPAGENAME}}]] |?Variant |limit=10000|format=sum}} [[Tutorial#Variants|variants]] on this page</b></font></div>
 
|}
 
|}
 
{{TOC limit|limit=3}}
 
{{TOC limit|limit=3}}
 
=Guidelines=
 
=Guidelines=
 +
'''Given the rapid change in evidence in many areas of hematology/oncology, readers are encouraged to consider any guideline published 5+ years ago to be for historical purposes, only.'''
 
==ASH/ISTH/NHF/WFH==
 
==ASH/ISTH/NHF/WFH==
−
*'''2021:''' James et al. [https://doi.org/10.1182/bloodadvances.2020003265 ASH ISTH NHF WFH 2021 guidelines on the diagnosis of von Willebrand disease]
+
*'''2021:''' James et al. [https://doi.org/10.1182/bloodadvances.2020003265 ASH ISTH NHF WFH 2021 guidelines on the diagnosis of von Willebrand disease] [https://www.ncbi.nlm.nih.gov/pubmed/33570651 PubMed]
−
*'''2021:''' Connell et al. [https://doi.org/10.1182/bloodadvances.2020003264 ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease]
+
*'''2021:''' Connell et al. [https://doi.org/10.1182/bloodadvances.2020003264 ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease] [https://www.ncbi.nlm.nih.gov/pubmed/33570647 PubMed]
 +
 
 
==BSH==
 
==BSH==
−
*'''2020:''' Rayment et al. [https://onlinelibrary.wiley.com/doi/full/10.1111/bjh.16704 Guidelines on the use of prophylactic factor replacement for children and adults with Haemophilia A and B]
+
*'''2020:''' Rayment et al. [https://doi.org/10.1111/bjh.16704 Guidelines on the use of prophylactic factor replacement for children and adults with Haemophilia A and B] [https://www.ncbi.nlm.nih.gov/pubmed/32390158 PubMed]
−
=="How I treat"==
 
−
*'''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]
 
  
 
=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}}==
−
 
+
<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;"  
Line 35: Line 32:
 
!style="width: 25%"|[[Levels_of_Evidence#Comparative_efficacy|Comparative Efficacy]]
 
!style="width: 25%"|[[Levels_of_Evidence#Comparative_efficacy|Comparative Efficacy]]
 
|-
 
|-
−
|[https://www.nejm.org/doi/full/10.1056/NEJMoa1803550 Mahlangu et al. 2018 (HAVEN 3)]
+
|[https://doi.org/10.1056/NEJMoa1803550 Mahlangu et al. 2018 (HAVEN 3)]
 
|style="background-color:#1a9851"|Phase 3 (E-esc)
 
|style="background-color:#1a9851"|Phase 3 (E-esc)
−
|[[#Observation_88|No prophylaxis]]
+
|[[#Observation_888|No prophylaxis]]
−
|style="background-color:#1a9850"|Lower bleeding rate
+
|style="background-color:#1a9850"|Lower bleeding rate (primary endpoint)
 +
|-
 +
|[https://doi.org/10.1016/S2352-3026(22)00377-5 Negrier et al. 2023 (HAVEN 6)]
 +
| style="background-color:#91cf61" |Non-randomized phase 3
 +
| style="background-color:#d3d3d3" |
 +
| style="background-color:#d3d3d3" |
 
|-
 
|-
 
|}
 
|}
−
To be completed
+
<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://www.nejm.org/doi/full/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] [https://clinicaltrials.gov/study/NCT02847637 NCT02847637]
 +
#'''HAVEN 6:''' Negrier C, Mahlangu J, Lehle M, Chowdary P, Catalani O, Bernardi RJ, Jimenez-Yuste V, Beckermann BM, Schmitt C, Ventriglia G, Windyga J, d'Oiron R, Moorehead P, Koparkar S, Teodoro V, Shapiro AD, Oldenburg J, Hermans C. Emicizumab in people with moderate or mild haemophilia A (HAVEN 6): a multicentre, open-label, single-arm, phase 3 study. Lancet Haematol. 2023 Mar;10(3):e168-e177. Epub 2023 Jan 27. [https://doi.org/10.1016/S2352-3026(22)00377-5 link to original article] [https://pubmed.ncbi.nlm.nih.gov/36716761/ PubMed] [https://clinicaltrials.gov/study/NCT04158648 NCT04158648]
  
 
=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;"  
Line 58: Line 61:
 
!style="width: 25%"|[[Levels_of_Evidence#Comparative_efficacy|Comparative Efficacy]]
 
!style="width: 25%"|[[Levels_of_Evidence#Comparative_efficacy|Comparative Efficacy]]
 
|-
 
|-
−
|[https://www.nejm.org/doi/full/10.1056/NEJMoa1703068 Oldenburg et al. 2017 (HAVEN 1)]
+
|[https://doi.org/10.1056/NEJMoa1703068 Oldenburg et al. 2017 (HAVEN 1)]
 
|style="background-color:#1a9851"|Phase 3 (E-esc)
 
|style="background-color:#1a9851"|Phase 3 (E-esc)
−
|[[#Observation_88|No prophylaxis]]
+
|[[#Observation_888|No prophylaxis]]
−
|style="background-color:#1a9850"|Lower bleeding rate
+
|style="background-color:#1a9850"|Lower bleeding rate (primary endpoint)
 
|-
 
|-
 
|}
 
|}
−
To be completed
+
<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://www.nejm.org/doi/full/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://ashpublications.org/blood/article-lookup/doi/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 sortable" style="width: 60%; text-align:center;"  
−
!style="width: 25%"|Study
+
!style="width: 33%"|Study
−
!style="width: 25%"|[[Levels_of_Evidence#Evidence|Evidence]]
+
!style="width: 33%"|Dates of enrollment
 +
!style="width: 33%"|[[Levels_of_Evidence#Evidence|Evidence]]
 
|-
 
|-
−
|[http://th.schattauer.de/en/contents/archive/issue/1975/manuscript/21293.html Leissinger et al. 2014]
+
|[https://www.ncbi.nlm.nih.gov/pmc/articles/pmc5726888/ Leissinger et al. 2014 (RICH)]
 +
|2006-08 to 2010-11
 
|style="background-color:#91cf61"|Phase 2
 
|style="background-color:#91cf61"|Phase 2
 
|-
 
|-
 
|}
 
|}
 +
<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 on day 1
−
 
+
'''7-day cycle for 4 cycles (28-day course)'''
−
'''28-day course'''
+
</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 protocol''' [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. [http://th.schattauer.de/en/contents/archive/issue/1975/manuscript/21293.html link to original article] [https://pubmed.ncbi.nlm.nih.gov/24919980 PubMed]
+
# '''RICH:''' 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. Epub 2014 Jun 12. [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] '''contains dosing details in manuscript''' [https://pubmed.ncbi.nlm.nih.gov/24919980/ PubMed] [https://clinicaltrials.gov/study/NCT00331006 NCT00331006]
  
 
=Factor IX deficiency (Hemophilia B), all lines of therapy=
 
=Factor IX deficiency (Hemophilia B), all lines of therapy=
 
To be completed
 
To be completed
−
 
 
[[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]]
 

Latest revision as of 11:34, 15 May 2024

Back to Top

Section editor
Tillman Benjamin-2.jpg
 Benjamin Tillman, MD
Vanderbilt University
Nashville, TN, USA
LinkedIn
  • We have moved How I Treat articles to a dedicated page.
3 regimens on this page
3 variants on this page


Guidelines

Given the rapid change in evidence in many areas of hematology/oncology, readers are encouraged to consider any guideline published 5+ years ago to be for historical purposes, only.

ASH/ISTH/NHF/WFH

BSH

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 (primary endpoint)
Negrier et al. 2023 (HAVEN 6) Non-randomized phase 3

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 NCT02847637
  2. HAVEN 6: Negrier C, Mahlangu J, Lehle M, Chowdary P, Catalani O, Bernardi RJ, Jimenez-Yuste V, Beckermann BM, Schmitt C, Ventriglia G, Windyga J, d'Oiron R, Moorehead P, Koparkar S, Teodoro V, Shapiro AD, Oldenburg J, Hermans C. Emicizumab in people with moderate or mild haemophilia A (HAVEN 6): a multicentre, open-label, single-arm, phase 3 study. Lancet Haematol. 2023 Mar;10(3):e168-e177. Epub 2023 Jan 27. link to original article PubMed NCT04158648

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 (primary endpoint)

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 Dates of enrollment Evidence
Leissinger et al. 2014 (RICH) 2006-08 to 2010-11 Phase 2

Immunosuppressive therapy

7-day cycle for 4 cycles (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. RICH: 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. Epub 2014 Jun 12. link to original article link to PMC article contains dosing details in manuscript PubMed NCT00331006

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

To be completed

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