Schimansky, Inga M. and Dobbelstein, Christiane and Klamroth, Robert and Hart, Christina and Sachs, Ulrich J. and Greil, Richard and Knobl, Paul and Oldenburg, Johannes and Miesbach, Wolfgang and Pfrepper, Christian and Trautmann-Grill, Karolin and Mohnle, Patrick and Holstein, Katharina and Eichler, Hermann and Werwitzke, Sonja and Tiede, Andreas (2025) Sustained survival benefit of emicizumab and postponed immunosuppression in acquired hemophilia A. BLOOD ADVANCES, 9 (22). pp. 5853-5860. ISSN 2473-9529, 2473-9537
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Acquired hemophilia A (AHA) is a severe bleeding disorder caused by neutralizing autoantibodies against coagulation factor VIII (FVIII). Standard treatment involves immunosuppressive therapy (IST), which carries a significant risk of serious infections, the leading cause of death in patients with AHA. The GTH-AHA-EMI study investigated the use of emicizumab to prevent bleeding during the first 12 weeks of management while postponing IST. We collected 2-year follow-up data from GTH-AHA-EMI patients (n = 47) and compared outcomes to a propensity score (PS)-matched cohort from the GTH-AH 01/2010 study (n = 101), in which patients received immediate IST. Outcome measures included overall survival (OS), infection-and bleed-related mortality, and time to complete remission (CR). The study cohorts were well-matched in age, sex, underlying conditions, baseline FVIII activity, inhibitor titer, and performance status. The PS-matched 2-year OS was 82% in the GTH-AHA-EMI cohort vs 63% in GTH-AH 01/2010 (hazard ratio, 0.39; 95% confidence interval, 0.19-0.80). Infection-related mortality was lower with emicizumab (4% vs 16%), whereas bleed-related mortality was similar (4% vs 3%). Spontaneous remission of AHA occurred in 15% of GTH-AHA-EMI patients. Time to CR estimated by the Kaplan-Meier method was longer with postponed IST in GTH-AHA-EMI (44 vs 16 weeks), but the estimated proportion of patients achieving CR was similar (76% vs 66%). In conclusion, emicizumab allowed for postponed IST initiation during early AHA management in the GTH-AHA-EMI study. Delayed IST was safe and effective. Compared to PS-matched historic controls receiving immediate IST but no emicizumab, GTH-AHA-EMI patients had fewer fatal infections and improved OS. This trial was registered at www.ClinicalTrials.gov as #NCT04188639.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | SURVEILLANCE; MANAGEMENT; RISK; |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Lehrstuhl für Innere Medizin III (Hämatologie und Internistische Onkologie) |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 01 Apr 2026 11:28 |
| Last Modified: | 01 Apr 2026 11:28 |
| URI: | https://pred.uni-regensburg.de/id/eprint/67731 |
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