Psychometrics of patient-reported outcomes measurement information system in von Willebrand disease, inherited platelet function disorders, and rare bleeding disorders.

Autor: van Hoorn ES; Department of Public Health, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands., Willems SPE; Department of Hematology, Radboud university medical center Nijmegen, Hemophilia Treatment Center Nijmegen-Eindhoven-Maastricht, Nijmegen, The Netherlands., Al Arashi W; Department of Pediatric Hematology, Erasmus MC Sophia Children's Hospital, University Medical Center Rotterdam, Rotterdam, The Netherlands., de Moor AS; Center for Benign Hematology, Thrombosis and Hemostasis, Van Creveldkliniek University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands., van Kwawegen CB; Department of Hematology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands., Teela L; Child and Adolescent Psychiatry & Psychosocial Care, Emma Children's Hospital, Amsterdam UMC location University of Amsterdam, Amsterdam, The Netherlands.; Public Health, Mental Health and Digital Health, Amsterdam UMC location University of Amsterdam, Amsterdam, The Netherlands.; Reproduction and Development, Child Development, Amsterdam UMC location University of Amsterdam, Amsterdam, The Netherlands., Oude Voshaar MAH; Department of Public Health, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands., Kremer Hovinga ICL; Center for Benign Hematology, Thrombosis and Hemostasis, Van Creveldkliniek University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands., Schutgens REG; Center for Benign Hematology, Thrombosis and Hemostasis, Van Creveldkliniek University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands., Schols SEM; Department of Hematology, Radboud university medical center Nijmegen, Hemophilia Treatment Center Nijmegen-Eindhoven-Maastricht, Nijmegen, The Netherlands., Leebeek FWG; Department of Hematology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands., Haverman L; Child and Adolescent Psychiatry & Psychosocial Care, Emma Children's Hospital, Amsterdam UMC location University of Amsterdam, Amsterdam, The Netherlands.; Public Health, Mental Health and Digital Health, Amsterdam UMC location University of Amsterdam, Amsterdam, The Netherlands.; Reproduction and Development, Child Development, Amsterdam UMC location University of Amsterdam, Amsterdam, The Netherlands., Cnossen MH; Department of Pediatric Hematology, Erasmus MC Sophia Children's Hospital, University Medical Center Rotterdam, Rotterdam, The Netherlands., Gouw SC; Pediatric Hematology, Emma Children's Hospital, Amsterdam UMC location University of Amsterdam, Amsterdam, The Netherlands., Lingsma HF; Department of Public Health, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands.
Jazyk: angličtina
Zdroj: Research and practice in thrombosis and haemostasis [Res Pract Thromb Haemost] 2024 Jun 13; Vol. 8 (4), pp. 102474. Date of Electronic Publication: 2024 Jun 13 (Print Publication: 2024).
DOI: 10.1016/j.rpth.2024.102474
Abstrakt: Background: Patient-reported outcomes measurement information system (PROMIS) measures can be used to measure patient-reported outcomes. PROMIS measures, including computer adaptive tests (CATs) and short forms, have demonstrated the ability to adequately assess outcomes in patients with hemophilia. It is, however, unclear if PROMIS measures are suitable for patients with von Willebrand disease (VWD), inherited platelet function disorders (IPFDs), and rare bleeding disorders (RBDs).
Objectives: To evaluate the feasibility, measurement properties, and relevance of PROMIS measures in adults with VWD, IPFDs, and RBDs.
Methods: In this cross-sectional multicenter study, adults with VWD, IPFDs, and RBDs completed 9 PROMIS measures and the Short Form-36 version 2 (SF-36v2) electronically. Feasibility was determined by the number of completed items and floor/ceiling effects. Measurement properties included construct validity based on a multitrait-multimethod analysis and reliability using the reliability coefficient and greatest lower bound. Relevance was evaluated based on comparison with the Dutch general population.
Results: In total, 111 patients (median age, 57 years [IQR, 44-67]; 60% VWD, 16% IPFD, 24% RBD) participated. Mean number of items answered varied from 5.3 to 8.7 (range, 4-12) per PROMIS CAT in patients with VWD. Construct validity was supported for all CATs and all instruments had a good reliability (≥0.70). The PROMIS measures had less ceiling effects than the SF-36v2.
Conclusion: The PROMIS measures are a feasible, valid, and reliable alternative for the SF-36v2 in patients with primarily nonsevere forms of VWD. The relevance of the selected measures was limited. Additional research is necessary to evaluate the PROMIS measures in adults with IPFDs and RBDs.
(© 2024 The Authors.)
Databáze: MEDLINE
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