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Poster Networking

Inherited Bleeding Disorders and Inhibitors

PB0262 - Effectiveness of emicizumab under real-world conditions in patients of all ages with hemophilia A with and without FVIII inhibitors: Third interim analysis of the non-interventional study EMIIL

Sunday, June 23, 2024
13:45 – 14:45 ICT
Room: Exhibition Hall

    Presenting Author(s)

  • Johannes Oldenburg, MD

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

    • Co-Author(s)

  • Hermann Eichler

    Physician
    Institute of Clinical Hemostaseology and Transfusion Medicine, Saarland University and University Hospital, Homburg, Germany
    Homburg/Saar, Saarland, Germany

  • Carmen Escuriola Ettingshausen, MD

    Director
    HZRM Hämophilie Zentrum Rhein Main
    Mörfelden-Walldorf, Hessen, Germany

  • Pierre Fontana

    Professor
    Division of Angiology and Hemostasis, Geneva University Hospitals
    Genève, Geneve, Switzerland

  • BH

    Bianca Heidersdorf

    Medical Manager Hemophilia
    Roche Pharma AG, Grenzach-Wyhlen, Germany
    Grenzach-Wyhlen, Baden-Wurttemberg, Germany

  • SK

    Sonja Kramer

    Physician
    Universitätsklinikum Regensburg, Regensburg, Germany
    Regensburg, Bayern, Germany

  • JK

    Johanna A. Kremer Hovinga

    Physician
    Bern University Hospital, University of Bern, Bern, Switzerland
    Bern, Bern, Switzerland

  • Wolfgang Miesbach, MD

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

  • CP

    Christian Pfrepper

    Physician
    Universitätsklinikum Leipzig, Leipzig, Germany
    Leipzig, Sachsen, Germany

  • SR

    Susanne Reimering

    Statistician
    Roche Pharma AG, Grenzach-Wyhlen, Germany
    Grenzach-Wyhlen, Baden-Wurttemberg, Germany

  • Mattia Rizzi, MD, PhD

    Staff Physician
    Pediatric Hematology-Oncology Unit - Pediatric Institute of Southern Switzerland, Ente Ospedaliero Cantonale, Bellinzona (TI), Switzerland - Lausanne University Hospital (CHUV) and University of Lausanne (UNIL), Lausanna (VD), Switzerland
    Le Mont sur Lausanne, Vaud, Switzerland

  • US

    Ulrich Sachs

    Physician
    Universitätsklinikum Giessen, Giessen, Germany
    Giessen, Hessen, Germany

  • US

    Ute Scholz

    Physician
    MVZ Labor, Leipzig, Germany
    Leipzig, Sachsen, Germany

  • PU

    Patrick Thomas Udvardi

    Medical Science Partner (MSP) Medical Affairs
    Roche Pharma (Schweiz) AG, Basel, Switzerland
    Basel, Basel-Stadt, Switzerland

  • Nv

    Nicolas von der Weid

    Physician
    Universitäts-Kinderspital beider Basel, Basel, Switzerland
    Basel, Basel-Stadt, Switzerland

  • SW

    Swee Wenning

    Physician
    SRH Kurpfalzkrankenhaus Heidelberg GmbH, Heidelberg, Germany
    Heidelberg, Baden-Wurttemberg, Germany

  • IW

    Ivonne Wieland

    Physician
    Medizinische Hochschule Hannover, Hannover, Germany
    Hannover, Niedersachsen, Germany

  • MA

    Manuela Albisetti

    Professor
    University Children's Hospital Zurich, Zurich, Switzerland
    Zurich, Zurich, Switzerland

Background: Emicizumab is a monoclonal, humanized bispecific antibody bridging coagulation factors IXa and X and thereby replacing the coagulation function of activated factor VIII (FVIII) even in the presence of FVIII inhibitors. Emicizumab is approved for routine prophylaxis in adult and pediatric patients with hemophilia A (PwHA) with or without FVIII inhibitors. Subcutaneous administration of emicizumab has demonstrated a positive benefit/risk profile in clinical trial settings. The non-interventional study (NIS) EMIIL was initiated to generate additional evidence for long-term effectiveness under real-world conditions.

Aims: To better understand the long-term effectiveness of emicizumab prophylaxis in PwHA with and without FVIII inhibitors under real-world conditions.

Methods: EMIIL (ISRCTN58752772) is an ongoing single-arm, two cohorts, prospective, multicenter NIS in Germany and Switzerland collecting safety and effectiveness data in PwHA newly treated with emicizumab. The primary endpoint is the annualized bleeding rate (ABR) of treated bleeds, estimated using a negative binomial regression model. Here we report results from the third interim analysis of cohort A.

Results: At data cut-off (May 15, 2023), a total of 112 patients aged 0-75 years were available for evaluation of cohort A (Table 1). After a median treatment duration of 629 days (range 21-1244), the model-based ABR was 0.64 (95% CI 0.49-0.85). Zero treated spontaneous, joint, and target joint bleeds were recorded in 78.6%, 70.5%, and 95.5% of patients, respectively. Recorded in 12-week time windows, the majority of patients experienced zero treated bleeds across the study period (range 83.0-100.0%). At data cut-off, 66 (58.9%) patients experienced 218 adverse events, none of them leading to treatment discontinuation (Table 2).

Conclusion(s): The results from the third interim analysis on the effectiveness and safety of emicizumab in a real-world setting, particularly data on ABR and the proportion of patients with zero bleeds appear to be consistent with results from previous clinical trials.