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    Oral Communication

    Hemophilia and Rare Bleeding Disorders

    Hemophilia Clinical - Factor VIII Mimetics

    OC 43.1 - A survey on clinical praxis in initiating emicizumab prophylaxis in previously untreated patients in the PedNet centers

    Monday, June 26, 2023
    14:45 – 15:00 ET
    Room: 710B
    Session Available As: On-Demand

      Co-Author(s)

    • SR

      Susanna Ranta

      Physician
      Dep. of Pediatrics, Clinic of Coag. Disorders Karolinska Hospital
      Stockholm, Sodermanlands Lan, Sweden

    • JM

      Jayashree Motwani

      Hematologist
      Department of Paediatric Haematology, Birmingham Children's Hospital, United Kingdom
      Birmingham, England, United Kingdom

    • JB

      Jan Blatny

      Director of Haemophilia Comprehensive Care centre
      Children’s University Hospital Brno
      Brno, Moravskoslezsky kraj, Czech Republic

    • MB

      Martina Bührlen

      Physician
      Klinikum Bremen Mitte
      Bremen, Bremen, Germany

    • Manuel Carcao, MD, FRCPC

      Professor
      Division of Haematology/Oncology, Department of Paediatrics, Hospital for Sick Children, University of Toronto, Toronto, Canada
      Toronto, Ontario, Canada

    • HC

      Hervé Chambost

      Professor and Head of the Comprehensive Care Centre for Congenital Bleeding Disorders
      AP-HM, Department of Pediatric Hematology Oncology, Children Hospital La Timone & Aix Marseille University, INSERM, INRA, C2VN, Marseille, France
      Marseille, Provence-Alpes-Cote d'Azur, France

    • Carmen Escuriola-Ettingshausen, MD

      Physician
      Haemophilia Centre Rhine-Main, Mörfelden-Walldorf, Germany
      Mörfelden-Walldorf, Hessen, Germany

    • Kathelijn Fischer, MD, PhD (she/her/hers)

      Pediatric Hematologist
      Center for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, The Netherlands
      Utrecht, Utrecht, Netherlands

    • Mutlu Kartal-Kaess, MD (she/her/hers)

      Consultant
      Inselspital Bern, Bern, Schweiz
      Bern, Bern, Switzerland

    • GK

      Gili Kenet

      Director
      The PedNet Foundation
      Barn, Utrecht, Netherlands

    • Christoph Male, MD, MSc

      Head of the Pediatric Hemostasis & Thrombosis Unit
      Department of Paediatrics, Medical University of Vienna, Vienna, Austria.
      Vienna, Wien, Austria

    • BN

      Beatrice Nolan

      Pediatric hematologist
      Department of Haematology, Children's Health Ireland at Crumlin, Dublin, Ireland.
      Dublin, Dublin, Ireland

    • Roseline d'Oiron, MD

      Director
      Centre for Haemophilia and Rare Congenital Bleeding Disorders, Bicêtre Hospital AP-HP, University of Paris-Saclay and UMR_S1176 INSERM, Le Kremlin-Bicêtre, France
      Le Kremlin-Bicetre, France

    • Martin Olivieri (he/him/his)

      Head of the Paediatric Thrombosis and Haemostasis Unit
      Paediatric Thrombosis and Haemostasis Unit, Paediatric Haemophilia Center, Dr. von Hauner Children's Hospital, LMU Clinic, Munich, Germany
      Munich, Bayern, Germany

    • NG

      Nadine Gretenkort Andersson

      Physician
      Lund University
      Malmö, Skane Lan, Sweden

      • Presenting Author(s)

    • Christoph Koenigs, Dr. Dr.

      Professor
      University Hospital Frankfurt, Department of Paediatrics and Adolescent Medicine, Frankfurt, Germany
      Frankfurt, Hessen, Germany

    Background: Emicizumab has recently become a valid alternative for prophylaxis in hemophilia A (HA). However, data and guidance on starting prophylaxis in the youngest, previously untreated patients (PUPs) is scarce.

    Aims: We aimed to evaluate the clinical routine in initiating prophylaxis with emicizumab in PUPs among PedNet centers.

    Methods: The PedNet registry currently holds data on 2576 children. An electronic survey was sent to 33 PedNet centers in autumn 2022. The survey included questions on availability of emicizumab, the routine of initiating prophylaxis in PUPs, and concomitant use of FVIII.

    Results: 26/33 centers responded. Emicizumab was available as part of clinical care in 23/26 centers (88%) from 20 countries, in 1/26 only for patients with difficult venous access or on clinical trial, and on clinical trial only for 2/26. Most centers (n=15) used emicizumab for severe HA only. For treatment of PUPs or minimally treated patients (MTPs), 19/24 centers used emicizumab as first line prophylaxis, with varying age at the start. 26% started in the first 6 months ( < 3 months (n=2), 3-6 months (n=3)), 58% (n=11) started 6-12 months, while 16% started at 12-18 months (n=3). Loading according to drug label was performed for PUPs in 17/19 centers. Routine concomitant FVIII administration is used in 3/19 centers (16%) for all PUPs/MTPs and in one center in case of high inhibitor risk. For maintenance, 15/26 centers (58%) use entire vials only while 11/26 administer the exact recommended dose.

    Conclusion(s): Emicizumab is widely used in PUPs but the clinical practice in initiating emicizumab varies. The majority (84%) starts prophylaxis before 12 months, without routine concomitant FVIII treatment in 79%. In the absence of evidence-based guidelines for prophylaxis with emicizumab in PUPs, this survey highlights the need for collection of real-life data on the safety and efficacy in large international registries.

    Learning Objectives:

    • reflect on the use of emiczumab or FVIII in young children with hemophilia
    • recognize the current practise of the use ofemicizmab in PedNet centers in Israel, Canada and Europe.
    • summarize open question in safety, efficacy and immunology on the use of emicizumab or FVIII in young children with hemophilia A