Inherited coagulopathy

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For placebo or observational studies in this condition, please visit this page.
*We have moved How I Treat articles to a dedicated page.
Last updated on 2024-12-02:4 regimens on this page
4 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==
*2021: James et al. ASH ISTH NHF WFH 2021 guidelines on the diagnosis of von Willebrand disease link to PMC article EuroPMC
*2021: Connell et al. ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease link to PMC article EuroPMC
==BSH==
*2020: Rayment et al. Guidelines on the use of prophylactic factor replacement for children and adults with Haemophilia A and B EuroPMC
=Factor VIII deficiency (Hemophilia A), replacement products=
==Efanesoctocog alfa monotherapy==
===Regimen===
Study 	Dates of enrollment	Evidence
von Drygalski et al. 2023 (XTEND-1)	Not reported 	Non-randomized Phase 3 (RT)
Malec et al. 2024 (XTEND-Kids) 	Not reported 	Non-randomized Phase 3 (RT)
====Supportive therapy====
*Efanesoctocog alfa (Altuviiio) 50 IU/kg IV once on day 1
7-day cycle for 52 cycles (1 year)
===References===
#XTEND-1: von Drygalski A, Chowdary P, Kulkarni R, Susen S, Konkle BA, Oldenburg J, Matino D, Klamroth R, Weyand AC, Jimenez-Yuste V, Nogami K, Poloskey S, Winding B, Willemze A, Knobe K; XTEND-1 Trial Group. Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A. N Engl J Med. 2023 Jan 26;388(4):310-318. link to original article dosing details in manuscript have been reviewed by our editors EuroPMC NCT04161495
#XTEND-Kids: Malec L, Peyvandi F, Chan AKC, Königs C, Zulfikar B, Yuan H, Simpson M, Álvarez Román MT, Carcao M, Staber JM, Dunn AL, Chou SC, d'Oiron R, Albisetti M, Demissie M, Santagostino E, Yarramaneni A, Wong N, Abad-Franch L, Gunawardena S, Fijnvandraat K; XTEND-Kids Trial Group. Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A. N Engl J Med. 2024 Jul 18;391(3):235-246. link to original article dosing details in manuscript have been reviewed by our editors EuroPMC NCT04759131
=Factor VIII deficiency (Hemophilia A), without inhibitors, all lines of therapy=
==Emicizumab monotherapy==
===Regimen===
Study 	Dates of enrollment 	Evidence 	Comparator 	Comparative Efficacy
Mahlangu et al. 2018 (HAVEN 3)	2016-09 to not reported 	Phase 3 (E-esc) 	No prophylaxis	Lower bleeding rate (primary endpoint)
Negrier et al. 2023 (HAVEN 6) 	2020-02-10 to 2021-08-31	Non-randomized phase 3	 	
====Supportive therapy====
*Emicizumab (Hemlibra)
===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. link to original article EuroPMC 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. link to original article EuroPMC NCT04158648
=Factor VIII deficiency (Hemophilia A), with inhibitors, all lines of therapy=
==Emicizumab monotherapy==
===Regimen===
Study 	Dates of enrollment 	Evidence 	Comparator 	Comparative Efficacy
Oldenburg et al. 2017 (HAVEN 1)	2015-11-17 to not reported	Phase 3 (E-esc)	No prophylaxis	Lower bleeding rate (primary endpoint)
====Supportive therapy====
*Emicizumab (Hemlibra)
===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. link to original article EuroPMC
# 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 link to PMC article EuroPMC
==Rituximab monotherapy==
===Regimen===
Study 	Dates of enrollment	Evidence
Leissinger et al. 2014 (RICH)	2006-08 to 2010-11 	Phase 2
====Immunosuppressive therapy====
*Rituximab (Rituxan) 375 mg/m2 IV once on day 1
7-day cycle for 4 cycles (28-day course)
===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. link to PMC article does not contain dosing details EuroPMC
# 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 dosing details in manuscript have been reviewed by our editors EuroPMC NCT00331006
=Factor IX deficiency (Hemophilia B), all lines of therapy=
To be completed
Category:Inherited coagulopathy regimens
Category:Disease-specific pages
Category:Bleeding disorders