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|Title:||Ahead international and german studies: 6-year interim effectiveness and safety outcomes in patients with hemophilia a receiving antihemophilic factor (recombinant) in a real-world setting|
Ozelo, M. C.
Tsakiris, D. A.
|Abstract:||The international (INT) and German (GER) Antihemophilic factor (recombinant) (rAHF) Hemophilia A (HA) outcome Database (AHEAD) studies were designed to assess long‐term, real‐world outcomes in patients with moderate/severe HA receiving rAHF (ADVATE®, Baxalta US Inc, a Takeda company, Lexington, MA, USA). These 2 non‐interventional, prospective, long‐term, multicenter studies (INT: NCT02078427; GER: DRKS 00000556) include patients receiving prophylaxis (PRO) or on‐demand (OD) rAHF treatment. Key outcomes include annualized bleeding rates (ABRs) by disease severity (moderate – INT study: factor VIII [FVIII] level ≥1% to ≤5%; GER study: FVIII level ≥1% to <5%; or severe – FVIII <1% in both studies), and adverse events (AEs). Results of the 6‐year interim analysis are reported here (data cut‐off 15 July 2019). ABRs are listed as median (min–max). The INT study safety analysis set (SAS) comprised 707 patients (208 with moderate HA, 497 with severe HA); 579 patients received rAHF PRO and 111 patients rAHF OD. The GER study SAS comprised 382 patients (72 with moderate HA, 310 with severe HA); 303 patients received rAHF PRO and 74 patients rAHF OD. In the INT study, ABRs at year 1 in moderate HA patients were: PRO 1.0 (0.0–28.7), OD 1.8 (0.0–61.1); and in severe HA patients: PRO 1.8 (0.0–21.3), OD 6.3 (0.0–61.0). At year 6, ABRs in moderate HA patients were: PRO 0.0 (0.0–15.9), OD 0.0 (0.0–13.2); and in severe HA patients: PRO 0.0 (0.0–9.2), OD 2.7 (2.1–37.1). In the GER study, ABRs at year 1 in moderate HA patients were: PRO 0.0 (0.0–37.0), OD 6.0 (0.0–16.0); and in severe HA patients: PRO 2.2 (0.0–45.0), OD 33.0 (33.0–33.0). At year 6, ABRs in moderate HA patients were: PRO 2.0 (0.0–17.0), OD 2.3 (1.0–9.0); and in severe HA patients: PRO 4.5 (0.0–23.0), OD 0.0 (0.0–3.0). Any AEs were reported in 406/701 (57.9%) INT and 308/382 (80.6%) GER patients, including de novo inhibitors in 10 INT and 9 GER patients.These data show long‐term effectiveness and tolerability of rAHF regardless of disease severity in INT and GER study populations in a real‐world setting|
|Appears in Collections:||FCM - Artigos e Outros Documentos|
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