Sustained survival benefit of emicizumab and postponed immunosuppression in acquired hemophilia A

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.

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Publication Details

Journal
Blood Advances
Published
2025-08-12
DOI
https://doi.org/10.1182/bloodadvances.2025017144
Citations
1
Primary Topic
Hemophilia Treatment and Research
Type
article
Field-Weighted Citation Impact
1.77

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Sustained survival benefit of emicizumab and postponed immunosuppression in acquired hemophilia A

Ulrich J. Sachs, Karolin Trautmann‐Grill, Robert Klamroth, Hermann Eichler et al.
1 citations
Blood Advances
Hemophilia Treatment and Research
1.77
article

Sustained survival benefit of emicizumab and postponed immunosuppression in acquired hemophilia A

Ulrich J. Sachs, Karolin Trautmann‐Grill, Robert Klamroth, Hermann Eichler, Richard Greil, Christian Pfrepper, Sonja Werwitzke, Inga Schimansky, Patrick Möhnle, Katharina Holstein, Paul Knoebl, Johannes Oldenburg, Andreas Tiede, Christiane Dobbelstein, Wolfgang Miesbach, Christina Hart
article en
1 citations

Abstract

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.

Blood AdvancesVol. 9(22)
Goethe University Frankfurt (DE), University of Bonn (DE), MODUL University Vienna (AT), Justus-Liebig-Universität Gießen (DE), Paracelsus Medical University (AT), University Hospital Bonn (DE), University Hospital Regensburg (DE), Medizinische Hochschule Hannover (DE), Institute for Transfusion Medicine (US), Universitätsklinikum des Saarlandes (DE), University Hospital Schleswig-Holstein (DE), University Hospital Leipzig (DE), University Hospital Frankfurt (DE), Klinikum im Friedrichshain (DE), University Hospital Carl Gustav Carus (DE), Klinik und Poliklinik für Psychotherapie und Psychosomatik (DE), Medical University of Vienna (AT), Ludwig-Maximilians-Universität München (DE), Saarland University (DE), University of Lübeck (DE)
Amgen, Bristol-Myers Squibb, Pfizer, AstraZeneca, Sanofi, Gilead Sciences, Biogen, Alexion Pharmaceuticals, Celgene, F. Hoffmann-La Roche, CSL Behring, Regeneron Pharmaceuticals, Ablynx, Novo Nordisk, Swedish Orphan Biovitrum, Grifols, Chugai Pharmaceutical, Bayer Vital, Daiichi Sankyo Europe
Openalex Percentile: Top 14%
Hemophilia Treatment and Research
1.77
DOIOpen Access PDFOpenAlex
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