Psychometrics of Patient-reported Outcomes Measurement Information System in Von Willebrand Disease, Inherited Platelet Function Disorders, and Rare Bleeding Disorders

Overview

Journal Res Pract Thromb Haemost

Publisher Elsevier

Date 2024 Jul 30

PMID 39076727

Authors

Evelien S van Hoorn

Sterre P E Willems

Wala Al Arashi

Annick S de Moor

Calvin B van Kwawegen

Lorynn Teela

Martijn A H Oude Voshaar

Idske C L Kremer Hovinga

Roger E G Schutgens

Saskia E M Schols

Frank W G Leebeek

Lotte Haverman

Marjon H Cnossen

Samantha C Gouw

Hester F Lingsma

Affiliations

Soon will be listed here.

Abstract

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.

Citing Articles

Patient-reported data on the severity of Von Willebrand disease.

van Kwawegen C, Fijnvandraat K, Kruip M, de Meris J, Schols S, Meijer K Haemophilia. 2024; 30(6):1348-1356.

PMID: 39403864 PMC: 11659499. DOI: 10.1111/hae.15103.

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