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Poster Networking

Inherited Bleeding Disorders and Inhibitors

PB0279 - Feasibility, measurement properties and relevance of generic PROMIS item banks in adults with von Willebrand disease, platelet function disorders, and rare bleeding disorders

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

    Co-Author(s)

  • Ev

    Evelien S. van Hoorn

    PhD Candidate
    Department of Public Health, Erasmus Medical Center, University Medical Center Rotterdam, Rotterdam, the Netherlands
    Woerden, Utrecht, Netherlands

  • SW

    Sterre P.E. Willems

    PhD Candidate
    Radboud university medical centre Nijmegen, Department of Hematology and Hemophilia Treatment Centre Nijmegen-Eindhoven-Maastricht, Nijmegen, The Netherlands
    Nijmegen, Gelderland, Netherlands

  • WA

    Wala Al Arashi

    MD
    Department of Pediatric Hematology and Oncology, Erasmus MC Sophia Children’s Hospital, University Medical Center Rotterdam, Rotterdam, The Netherlands
    Rotterdam, Zuid-Holland, Netherlands

  • Ad

    Annick S. de Moor

    Resident
    Center for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek University Medical Centre Utrecht, Utrecht University, Utrecht, The Netherlands
    Utrecht, Utrecht, Netherlands

  • Calvin B. van Kwawegen, MSc

    PhD candidate
    Erasmus MC, University Medical Centre Rotterdam, Department of Haematology, Rotterdam, The Netherlands
    Rotterdam, Zuid-Holland, Netherlands

  • LT

    Lorynn Teela

    Researcher
    Amsterdam UMC location University of Amsterdam, Emma Children’s Hospital, Child and Adolescent Psychiatry & Psychosocial Care, Meibergdreef 9, Amsterdam, The Netherlands
    Amsterdam, Noord-Holland, Netherlands

  • MO

    Martijn A.H. Oude Voshaar

    Assistent professor
    Erasmus MC, University Medical Centre Rotterdam, Department of Public Health, Rotterdam, The Netherlands
    Rotterdam, Zuid-Holland, Netherlands

  • IK

    Idske C.L. Kremer Hovinga

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

  • Roger E.G. Schutgens

    Internist-hematologist
    Department of Benign Hematology, van Creveldkliniek, University Medical Center Utrecht and University Utrecht, Utrecht, the Netherlands
    Utrecht, Utrecht, Netherlands

    • Presenting Author(s)

  • SS

    Saskia E.M. Schols

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

    • Co-Author(s)

  • Frank W.G Leebeek, MD PhD

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

  • LH

    Lotte Haverman

    Medical psychologist
    Amsterdam UMC location University of Amsterdam, Emma Children’s Hospital, Child and Adolescent Psychiatry & Psychosocial Care, Meibergdreef 9, Amsterdam, The Netherlands
    Amsterdam, Noord-Holland, Netherlands

  • MC

    Marjon H. Cnossen

    MD, PhD, professor
    Department of Pediatric Hematology and Oncology, Erasmus MC Sophia Children’s Hospital, University Medical Center Rotterdam, Rotterdam, The Netherlands
    Rotterdam, 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

  • HL

    Hester F. Lingsma

    Professor
    Erasmus MC, University Medical Centre Rotterdam, Department of Public Health, Rotterdam, The Netherlands
    Rotterdam, Zuid-Holland, Netherlands

Background: Inherited bleeding disorders are chronic diseases that can have a great impact on quality of life (QoL). Patient Reported Outcomes Measurement Information System (PROMIS) item banks, including Computer Adaptive Tests (CATs) and short forms, have the advantage of being shorter and more patient friendly compared to classical PROMs. These item banks have been successfully validated in patients with hemophilia. It is however unclear if PROMIS item banks are suitable to measure the QoL of patients with other inherited bleeding disorders.

Aims: To evaluate the feasibility, measurement properties and relevance of nine PROMIS measures in adults with von Willebrand disease (VWD), inherited platelet function disorders (IPFDs) and rare bleeding disorders (RBDs) compared to a legacy instrument.

Methods: In this cross-sectional study, adults with VWD, IPFDs, and RBDs completed nine PROMIS measures and the legacy instrument SF-36 version 2 electronically. Feasibility was determined by the number of completed questions. Construct validity was assessed based on a multitrait-multimethod analysis. Reliability was determined using the reliability coefficient and greatest lower bound. Relevance was evaluated based on floor/ceiling effects and comparisons with the Dutch general population.

Results: In total, 110 patients (median age 57 years [IQR 43-67], 61% VWD, 15% IPFD, 25% RBD) participated. Per PROMIS CAT, mean number of items answered varied from 5.5 to 8.8 (range 4-12). Construct validity was supported for all CATs and all instruments had a good reliability (≥0.70). Floor and ceiling effects were observed in both the PROMIS measures (0-35.5%) and SF-36v2 (0-92.5%). The study population reported lower scores on CAT physical function than the general population. A synthesis of the results is presented in Table 1.

Conclusion(s): The PROMIS measures are a reliable alternative for measuring QoL compared to the SF-36v2 for patients with VWD, IPDFs, and RBDs.