Inherited coagulopathy

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Shruti Chaturvedi, MBBS, MSCI
Baltimore, MD

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Benjamin Tillman, MD
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 Efficacy
Mahlangu et al. 2018 (HAVEN 3) Phase III (E) No prophylaxis Lower bleeding rate

To be completed

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

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Regimen

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

To be completed

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

Rituximab monotherapy

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Regimen

Study Evidence
Leissinger et al. 2014 Phase II

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 protocol 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 PubMed

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

To be completed