Evidence map›Paper›PMID 40795229›Full record

Trial reportBlood advances2025

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

Inga M Schimansky, Christiane Dobbelstein, Robert Klamroth, Christina Hart, Ulrich J Sachs, Richard Greil, Paul Knöbl, Johannes Oldenburg, Wolfgang Miesbach, Christian Pfrepper and 6 more

Registry-linked trialAbstract readClinical Trial
In one paragraph

Trial report in Blood advances, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT04188639 (Emicizumab in Patients With Acquired Hemophilia A), which is not on this map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT04188639 phase2completednot on this map

Emicizumab in Patients With Acquired Hemophilia A: Multicenter, Single-arm, Open-label Clinical Trial

TypeinterventionalSponsorGWT-TUD GmbHRan2021 to 2023Enrolled47ConditionsHemophilia A, AcquiredArmsEmicizumab Injection
3 · Its place in the literature

Who cites it

4 citing papers in PubMed.

  1. Article
  2. Acquired hemophilia A: an illustrated review based on the French National Guidelines.Research and practice in thrombosis and haemostasis · 2026
    Review
  3. Article
  4. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

16 authors.

Inga M SchimanskyDepartment of Hematology, Hemostasis, Oncology, and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany.
Christiane DobbelsteinDepartment of Hematology, Hemostasis, Oncology, and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany.
Robert KlamrothDepartment of Internal Medicine, Vivantes Clinic Friedrichshain, Berlin, Germany.ORCID 0000-0003-4194-8183
Christina HartDepartment of Hematology and Oncology, University Hospital Regensburg, Regensburg, Germany.
Ulrich J SachsInstitute for Clinical Immunology and Transfusion Medicine, Justus Liebig University, Giessen, Germany.
Richard GreilInternal Medicine III, Paracelsus Medical University Salzburg, Salzburg Cancer Research Institute-Center for Clinical Cancer and Immunology Trials, Cancer Cluster Salzburg, Salzburg, Austria.
Paul KnöblDivision of Hematology and Hemostasis, Department of Medicine I, Medical University of Vienna, Vienna, Austria.ORCID 0000-0002-7909-7225
Johannes OldenburgInstitute of Experimental Hematology and Transfusion Medicine, University Clinic Bonn, Bonn, Germany.ORCID 0000-0002-1585-4100
Wolfgang MiesbachMedical Clinic II, Institute of Transfusion Medicine, Goethe University, Frankfurt, Germany.ORCID 0000-0002-4506-0061
Christian PfrepperDivision of Hemostaseology, Medical Department I, University Hospital Leipzig, Leipzig, Germany.ORCID 0000-0002-0485-7402
Karolin Trautmann-GrillMedical Clinic I, University Hospital Carl Gustav Carus, TU Dresden, Dresden, Germany.
Patrick MöhnleDepartment of Transfusion Medicine, Cellular Therapeutics and Hemostaseology, Department of Anesthesiology, Hospital of Ludwig Maximilian University, Munich, Germany.
Katharina HolsteinInstitute of Clinical Chemistry, University Medical Center Schleswig-Holstein, Kiel, Germany.
Hermann EichlerInstitute of Clinical Hemostaseology and Transfusion Medicine, Saarland University and Saarland University Hospital l, Saarbrücken, Germany.
Sonja WerwitzkeDepartment of Hematology, Hemostasis, Oncology, and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany.
Andreas TiedeDepartment of Hematology, Hemostasis, Oncology, and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany.ORCID 0000-0002-3600-8536

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

abstractAcquired 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.

Indexed as

Antibodies, BispecificAntibodies, Monoclonal, HumanizedHemophilia AImmunosuppression TherapyAdultAgedFactor VIIIFemaleHemorrhageHumansMaleMiddle AgedTreatment OutcomeAntibodies, BispecificAntibodies, Monoclonal, HumanizedemicizumabFactor VIII

Identifiers

PMID40795229
PMCPMC12663507

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.