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hemophilia-a
Hemophilia A
Hemophilia A is X-linked FVIII deficiency (F8 gene; Xq28); severe <1 IU/dL → joint/muscle hemorrhage. Emicizumab (bispecific FIXa/FX mAb; HAVEN-3: ABR 0.3 vs 22.9; FDA 2017) replaced prophylactic FVIII as standard of care in inhibitor and non-inhibitor severe HA.
Entry Metadata
| Field | Value |
|---|---|
| ID | hemophilia-a |
| Name | Hemophilia A |
| Status | draft |
| Last reviewed | 2026-06-07 |
| Atlas | 01-human |
| Scale | 07-system |
Cross-Atlas Connections
Sources
- Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017;377(9):809-818. · PubMed 28691557
- Mahlangu J, Oldenburg J, Paz-Priel I, et al. Emicizumab prophylaxis in patients who have hemophilia A without inhibitors. N Engl J Med. 2018;379(9):811-822. · PubMed 30157389
- Pipe SW, Leebeek FW, Recht M, et al. Once-monthly subcutaneous fitusiran versus on-demand bypassing agent for haemophilia A or B with inhibitors (ATLAS-INH): a multicentre, open-label, randomised phase 3 trial. Lancet. 2023;401(10386):1427-1439. · PubMed 37003297