Inherited coagulopathy
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Section editors | |||
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Shruti Chaturvedi, MBBS, MSCI Johns Hopkins University Baltimore, MD |
Benjamin Tillman, MD Vanderbilt University Nashville, TN |
3 regimens on this page
3 variants on this page
|
Factor VIII deficiency (Hemophilia A), replacement products
To be completed
Factor VIII deficiency (Hemophilia A), without inhibitors, all lines of therapy
Emicizumab monotherapy
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Regimen
Study | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|
Mahlangu et al. 2018 (HAVEN 3) | Phase III (E) | No prophylaxis | Lower bleeding rate |
To be completed
Supportive therapy
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 PubMed
Factor VIII deficiency (Hemophilia A), with inhibitors, all lines of therapy
Emicizumab monotherapy
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Regimen
Study | Evidence | Comparator | Comparative Efficacy |
---|---|---|---|
Oldenburg et al. 2017 (HAVEN 1) | Phase III (E) | No prophylaxis | Lower bleeding rate |
To be completed
Supportive therapy
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 PubMed
Rituximab monotherapy
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Regimen
Study | Evidence |
---|---|
Leissinger et al. 2014 | Phase II |
Immunosuppressive therapy
- Rituximab (Rituxan) 375 mg/m2 IV once per day on days 1, 8, 15, 22
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 protocol 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. link to original article PubMed
Factor IX deficiency (Hemophilia B), all lines of therapy
To be completed