Inherited coagulopathy

Section editor transclusions

3 regimens on this page 3 variants on this page

Guidelines

ASH/ISTH/NHF/WFH

• 2021: James et al. ASH ISTH NHF WFH 2021 guidelines on the diagnosis of von Willebrand disease
• 2021: Connell et al. ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease

BSH

• 2020: Rayment et al. Guidelines on the use of prophylactic factor replacement for children and adults with Haemophilia A and B

"How I treat"

• 2020: Leebeek et al. How I manage pregnancy in carriers of hemophilia and patients with von Willebrand disease
• 2019: Young. How I treat children with haemophilia and inhibitors
• 2019: Lavin & O'Donnell How I treat low von Willebrand factor levels

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

• Emicizumab (Hemlibra)

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

• Emicizumab (Hemlibra)

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

• Rituximab (Rituxan) 375 mg/m² IV once per day on days 1, 8, 15, 22

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