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CME Evaluation

Oral Communication Session

Inherited Bleeding Disorders and Inhibitors

Session: Hemophilia - Joint health clinical

OC 01.5 - People with mild hemophilia are at risk of joint disease: the nationwide cohort HiN-6 Study

Saturday, June 22, 2024
14:00 – 14:15 ICT
Room: Ballroom B1

    Presenting Author(s)

  • Ricardo Mesquita Camelo, MD, MBA, MSc, PhD, PostD

    Professor
    Department of Internal Medicine, Faculty of Medicine, Universidade Federal de Minas Gerais (UFMG), Belo Horizonte, Brazil
    Belo Horizonte, Minas Gerais, Brazil

    • Co-Author(s)

  • CS

    Cees Smit

    Patient representative
    Department of Clinical Epidemiology, Leiden University Medical Center (LUMC)
    Leinden, Zuid-Holland, Netherlands

  • Lv

    Lize F. D. van Vulpen

    Hematologist
    Van Creveldkliniek, University Medical Center Utrecht, Utrecht University
    Utrecht, Utrecht, Netherlands

  • Jeroen Eikenboom

    MD, PhD, prof
    Department of Thrombosis and Hemostasis, Leiden University Medical Center, Leiden, The Netherlands
    Leiden, Zuid-Holland, Netherlands

  • EB

    Erik Beckers

    Professor Hematology
    Department of Internal medicing - Hematology, Maastricht University Medical Center +, Maastricht, the Netherlands
    Maastricht, Limburg, Netherlands

  • LH

    Louise Hooimeijer

    Pediatrician
    Department of Hematology and Oncology, UMC Groningen Beatrix Children’s Hospital, University Medical Center Groningen, Groningen, The Netherlands
    Groningen, Groningen, Netherlands

  • Michiel Coppens

    MD, PhD
    Department of Vascular Medicine, Amsterdam UMC location University of Amsterdam
    Amsterdam, Noord-Holland, Netherlands

  • SS

    Saskia E.M. Schols

    Internist-hematologist
    Department of Hematology, Radboud university medical center, Nijmegen, The Netherlands
    Nijmegen, Gelderland, Netherlands

  • Frank W.G Leebeek, MD PhD

    Internist-hematologist, Professor
    Department of Hematology, Erasmus University Medical Center, Rotterdam, The Netherlands
    Rotterdam, Zuid-Holland, Netherlands

  • MD

    Mariëtte H.E. Driessens

    Patient representative
    Netherlands Hemophilia Patient Society (NVHP), Nijkerk, the Netherlands
    Nijkerk, Gelderland, Netherlands

  • FR

    Frits R Rosendaal

    Professor at Leiden University Medical Center and Chairman of the Clinical Epidemiology Department
    Department of Clinical Epidemiology, Leiden University Medical Center, Leiden, The Netherlands.
    Leiden, Zuid-Holland, Netherlands

  • Suely M. Rezende, MD, PhD

    Professor of Internal Medicine
    Universidade Federal de Minas Gerais
    Belo Horizonte, Minas Gerais, Brazil

  • Jv

    Johanna van der bom

    Professor of Clinical Epidemiology
    Leiden University Medical Center Leiden, Leiden, Netherlands
    Leiden, Zuid-Holland, Netherlands

  • SG

    Samantha C. Gouw, MD PhD

    Pediatric hematologist
    Department of Pediatric Hematology, Emma Children’s Hospital, Amsterdam UMC location University of Amsterdam, Amsterdam, the Netherlands
    Amsterdam, Noord-Holland, Netherlands

Background: Clinical data on people with mild hemophilia (mPwH) is important to guide their treatment but remains scarce worldwide. In addition, since modern hemophilia therapies can turn severe phenotypes into mild or normal ones, knowing the clinical, therapeutical, and outcomes data of mPwH becomes essential as a proxy of these treatments.

Aims: We described mPwH who participated in the 6th version of the Hemophilia in the Netherlands (HiN-6) cross-sectional nationwide study.

Methods: Male mPwH (lowest plasmatic factor activity 5.1%-40.0%) answered a questionnaire about their disease, treatment, and outcomes, in 2019. Questionnaires were elaborated according to age: younger than 12 years (children and responded by their parents/caregivers), between 12 and 17.9 years (adolescents), and 18 years or older (adults).

Results: A total of 387 mPwH responded to the questionnaires. Their median (interquartile range; IQR) age was 49.0 years (26.0-62.0), 340 (87.8%) were adults, 29 (7.5%) had past or current inhibitors, 197 (50.9%) had ever been treated with any clotting product, and 12 (3.1%) were currently on prophylaxis. Approximately 74% reported they perceived their disease was not serious or not serious at all, and the different activity groups responded similarly. Recent bleed at any site was reported by 24/47 (51.1%) non-adults, related to the previous quarter, and 96/340 (28.2%) adults, related to the previous year. Joint impairment was reported for 180 joints (22 perceived as severe) by 50/387 (12.9%) mPwH. Age at first hemarthrosis was 10.0 years [5.0-16.0] among 97 (25%) mPwH. Lifetime hospitalization due to joint surgery was reported by 31/358 (8.7%) mPwH. The analyses of PROMIS29 and RAND-26 (quality of life), and HAL (functional abilities) indicated good quality of life and functional abilities.

Conclusion(s): mPwH who participated in the HiN-6 Study reported good social functioning and a non-impactant disease in their lives, although bleeds, joint impairment, and need for joint surgeries still occur.