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    Late-Breakthrough Submission

    Hemophilia and Rare Bleeding Disorders

    Late-Breakthrough Session I

    LB 01.2 - Emicizumab prophylaxis instead of immunosuppressive therapy in patients with acquired hemophilia A (AHA)

    Sunday, June 25, 2023
    11:00 – 11:15 ET
    Room: 517
    Session Available As: Live-Streamed and On-Demand

      Presenting Author(s)

    • Andreas Tiede, MD PhD (he/him/his)

      Professor of Hemostasis and Thrombosis
      Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany
      Hannover, Niedersachsen, Germany

      • Co-Author(s)

    • CH

      Christina Hart

      Senior physician
      Department of Hematology and Oncology, University Hospital Regensburg
      Regensburg, Bayern, Germany

    • Paul Knoebl, MD

      Professor
      Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna, Austria
      Vienna, Wien, Austria

    • RG

      Richard Greil

      Director
      IIIrd Medical Department, Paracelsus Medical University Salzburg
      Salzburg, Salzburg, Austria

    • Johannes Oldenburg, MD (he/him/his)

      Professor, Chairman and Director
      University Clinic Bonn AöR, Institute of Experimental Haematology and Transfusion Medicine, Bonn, Germany
      Bonn, Nordrhein-Westfalen, Germany

    • Ulrich J. J. Sachs, MD

      Senior physician
      Department of Thrombosis and Haemostasis, Giessen University Hospital
      Gießen, Hessen, Germany

    • Wolfgang Miesbach, MD

      Professor and Head of Coagulation Disorders and Comprehensive Care Centre
      University Hospital Frankfurt, Frankfurt, Germany
      Frankfurt, Hessen, Germany

    • CP

      Christian Pfrepper, MD

      Physician
      Division of Hemostaseology, University Hospital Leipzig, Germany
      Leipzig, Sachsen, Germany

    • KT

      Karolin Trautmann-Grill

      Senior physician
      Medical Clinic I, University Hospital Carl Gustav Carus
      Dresden, Sachsen, Germany

    • Katharina Holstein, MD (she/her/hers)

      physician
      University Hospital Hamburg Eppendorf
      Hamburg, Hamburg, Germany

    • Jan Pilch, MD

      Assistant Professor
      Institute of Clinical Hemostaseology and Transfusion Medicine, Saarland University School of Medicine and Saarland University Medical Center
      Homburg, Saarland, Germany

    • PM

      Patrick Möhnle

      Senior physician
      Department of Transfusion Medicine, Cellular Therapeutics and Hemostaseology, Department of Anesthesiology, Hospital of Ludwig Maximilian University
      Munich, Bayern, Germany

    • CS

      Christoph Schindler

      Director
      Center for Clinical Trials (ZKS), Hannover Medical School
      Hannover, Niedersachsen, Germany

    • CW

      Carmen Weigt

      Project lead
      GWT-TUD
      Dresden, Sachsen, Germany

    • DS

      Dorothea Schipp

      Senior statistician
      GWT-TUD
      Dresden, Sachsen, Germany

    • MM

      Marcus May

      Project manager
      Hematology, Hemostasis, Oncology, and Stem Cell Transplantation, Hannover Medical School
      Hannover, Niedersachsen, Germany

    • CD

      Christiane Dobbelstein

      Senior physician
      Hematology, Hemostasis, Oncology, and Stem Cell Transplantation, Hannover Medical School
      Hannover, Niedersachsen, Germany

    • SW

      Sonja Werwitzke

      Specialist in laboratory medicine
      Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany
      Hannover, Niedersachsen, Germany

    • Robert Klamroth, MD, PhD (he/him/his)

      Priv. Doz. Dr. med.
      Vivantes Klinikum im Friedrichshain, Clinic for Internal Medicine Angiology and Haemostaseology
      Berlin, Berlin, Germany

    Background: Immunosuppressive therapy (IST) is associated with adverse events and mortality in patients with acquired hemophilia A (AHA). This study aimed to assess whether patients can be treated with emicizumab prophylaxis instead of IST.

    Aims: To evaluate the efficacy and safety of emicizumab in AHA patients not treated with IST.

    Methods: This open-label, non-randomized clinical trial enrolled patients with AHA who had not received IST before enrollment. Patients received accelerated subcutaneous emicizumab loading on day 1 (6 mg/kg) and day 2 (3 mg/kg), followed by 1.5 mg/kg weekly until week 12. The primary endpoint was the mean rate of clinically relevant bleeds per patient-week until week 12. Clinical relevance was defined as bleeds requiring intervention by healthcare professionals or causing pain or any other kind of harm. A prophylactic effect of emicizumab was assumed if the mean bleeding rate was significantly below 0.15 bleeds per patient-week, the rate observed in the GTH-AH 01/2010 study in patients receiving IST but no emicizumab. Secondary endpoints included thromboembolic events and mortality until week 24.

    Results: Forty-seven patients were enrolled (24 males, 23 females; median age 76 years [interquartile range 66-80], FVIII 1.4 IU/dl [0.3-5.6], inhibitor 11.4 BU/ml [3.9-43]). The primary efficacy criterion was met with an observed rate of 0.04 bleeds per patient-week (p < 0.001). 70% of patients had zero breakthrough bleeds. Two thromboembolic events were observed. Four deaths occurred (two related to bleeding, one infection, one cardiac arrest, none related to emicizumab). The overall survival rate was 91% after 24 weeks.

    Conclusion(s): According to a predefined efficacy criterion, emicizumab was effective in preventing bleeds in AHA patients not treated with IST. The incidence of thromboembolic events was similar to historic registry studies and mortality from infections appeared low. These results suggest that patients with AHA can be managed with emicizumab prophylaxis.