Inherited coagulopathy - null regimens
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The purpose of this page is to provide references to "null therapy" such as placebo and observation. These references provide further insight into the historical development of the treatment landscape. See the main inherited coagulopathy page for regimens that include active treatment.
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 (C) | Emicizumab | Higher bleeding rate (primary endpoint) |
No active treatment.
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 NCT02847637