Hart, Christina and Klamroth, Robert and Sachs, Ulrich J. and Greil, Richard and Knoebl, Paul and Oldenburg, Johannes and Miesbach, Wolfgang and Pfrepper, Christian and Trautmann-Grill, Karolin and Pekrul, Isabell and Holstein, Katharina and Eichler, Hermann and Weigt, Carmen and Schipp, Dorothea and Werwitzke, Sonja and Tiede, Andreas (2024) Emicizumab versus immunosuppressive therapy for the management of acquired hemophilia A. JOURNAL OF THROMBOSIS AND HAEMOSTASIS, 22 (10). pp. 2692-2701. ISSN 1538-7933, 1538-7836
Full text not available from this repository. (Request a copy)Abstract
Background: Acquired hemophilia A (AHA) is an autoimmune bleeding disorder caused by neutralizing antibodies against coagulation factor VIII. Immunosuppressive therapy (IST) is standard of care to eradicate autoantibody production and protect from further bleeding but carries a risk of severe infection and mortality in frail patients with AHA. Recently, emicizumab has been studied for its potential to reduce the need for early and aggressive IST. Objectives: To compare outcomes of 2 studies that used either IST (GTH-AH 01/2010; N = 101) or prophylaxis with emicizumab (GTH-AHA-EMI; N = 47) early after diagnosis of AHA. Methods: Baseline characteristics were balanced by propensity score matching. Primary endpoint was the rate of clinically relevant new bleeds during the first 12 weeks; secondary endpoints were adverse events and overall survival. Results: The negative binominal model-based bleeding rate was 68% lower with emicizumab as compared with IST (incident rate ratio, 0.325; 95% CI, 0.182-0.581). No difference was apparent in the overall frequency of infections (emicizumab 21%, IST 29%) during the first 12 weeks, but infections were less often fatal in emicizumab-treated patients (0%) compared with IST-treated patients (11%). Thromboembolic events occurred less often with emicizumab (2%) than with IST (7%). Overall survival after 24 weeks was better with emicizumab (90% vs 76%; hazard ratio, 0.44; 95%, CI, 0.24-0.81). Conclusion: Using emicizumab instead of IST in the early phase after initial diagnosis of AHA reduced bleeding and fatal infections and improved overall survival.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | SURVEILLANCE; autoimmune hemophilia; bispecific antibody; bleeding; immunosuppression; inhibitor |
| 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: | 22 Jan 2026 11:20 |
| Last Modified: | 22 Jan 2026 11:20 |
| URI: | https://pred.uni-regensburg.de/id/eprint/65424 |
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