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CME Evaluation

Oral Communication Session

Inherited Bleeding Disorders and Inhibitors

Session: Clinical hemophilia - Real world data

OC 71.2 - Intracranial hemorrhage before the initiation of prophylaxis in early age in hemophilia A – data from the PedNet registry

Wednesday, June 26, 2024
10:30 – 10:45 ICT
Room: Ballroom B2

    Presenting Author(s)

  • Ester Zapotocka, MD

    Senior haematologist
    Medical faculty, Masaryk University, Brno, Czech Republic
    Prague, Hlavni mesto Praha, Czech Republic

    • Co-Author(s)

  • Md

    Marloes de Kovel

    statistician
    PedNet Haemophilia Research Foundation, Baarn, The Netherlands
    Barn, Utrecht, Netherlands

  • Giancarlo Castaman

    Medical Director Hematologist
    Department of Oncology Careggi University Hospital, Florence, Italy
    Florence, Toscana, Italy

  • Roseline d'Oiron

    Associate Director
    Centre de Référence de l'Hémophilie et des Maladies Hémorragiques Rares, and HITh UMR_S1176 INSERM, Hopital Bicêtre, APHP Université Paris Saclay, Le Kremlin Bicêtre, France
    Le Kremlin-Bicêtre, Ile-de-France, France

  • Kathelijn Fischer

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

  • GK

    Gili Kenet

    CONSULTANT PAEDIATRIC HAEMATOLOGIST
    National Hemophilia Center Sheba Medical center, Tel Hashomer & Amalia Biron Research Institute of Thrombosis & Hemostasis, Tel Aviv University, Israel,
    Tel Aviv, Tel Aviv, Israel

  • Christoph Male

    Consultant, Associate professor
    Department of Paediatrics, Medical University Hospital of Vienna, Vienna, Austria
    Wien, Wien, Austria

  • BN

    Beatrice Nolan

    Clinical Senior Lecturer
    Children's Health Ireland at Crumlin, Dublin, Republic of Ireland
    Dublin, Dublin, Ireland

  • Martin Olivieri

    Head of Pediatric Thrombosis and Hemostasis Unit, Pediatric Hemophilia Center
    Paediatric Thrombosis and Haemostasis Unit, Dr. von Hauner Children's Hospital LMU Munich, Germany
    munich, Bayern, Germany

  • FP

    Fernando Pinto

    Hematologist
    Department of Haematology, Royal Hospital for Sick Children, Yorkhill, Glasgow, UK
    Glasgow, Scotland, United Kingdom

  • SS

    Sirgidur Sigurgisladottir

    physician
    Department of Pediatrics, Skåne University Hospital, Malmö, Sweden
    Malmo, Uppsala Lan, Sweden

  • NA

    Nadine Gretenkort Andersson

    Senior consultant
    Department of Clinical Sciences, Lund University, Lund; Department of Pediatrics and Malmö Centre for Thrombosis and Haemostasis, Skåne University Hospital, Malmö, Sweden
    Malmo, Skane Lan, Sweden

Background: Children with hemophilia A (HA) have a significantly higher risk of intracranial hemorrhage (ICH) than the general population. Prophylaxis reduces the risk of ICH and earlier initiation of prophylaxis may now be feasible, with subcutaneous injections of non-replacement therapy. However, the ideal timing of prophylaxis initiation required to prevent ICH is unclear.

Aims: To evaluate the incidence and timing of ICH in children with HA after the neonatal period until 3 years of age, before initiation of prophylaxis.

Methods: Data from the PedNet Registry were used. The study was approved by the institutional review boards of each participating center, and informed consent was obtained from all guardians.

Results: 2,275 children with HA (1,466, 292, and 517 with severe, moderate, and mild HA, respectively) enrolled in the Registry by January 1st, 2023 were included. The median age at diagnosis of HA was 4.5 (0.0-12.0) months. A total of 57 ICH was observed, 24 traumatic and 33 spontaneous/unknown. In 39.6% of cases, the diagnosis of HA was unknown and was made on the day of ICH or later. The incidence of ICH before age 3 years/prophylaxis was 3.5% in severe, 1.0% in moderate and 0.58% in mild HA (Table 1), with a statistically significant difference between severe and non-severe patients (51/1466 versus 6/809; p< 0.0001). Figure 1 shows the timing of ICH in severe and non-severe HA: the incidence increases at 3 months of age in severe HA, with 37 % of ICH (19/51) reported before 6 months of age; however, 8 of 19 children were not yet diagnosed with HA.

Conclusion(s): The early incidence of ICH in children beyond the neonatal period should be taken into account when considering the initiation of prophylaxis in patients with severe HA, as ICH may be potentially preventable with early prophylaxis in children with a known diagnosis.