Generic PROMIS item banks in adults with hemophilia for patient-reported outcome assessment: Feasibility, measurement properties, and relevance

Isolde A.R. Kuijlaars*, Lorynn Teela, Lize F.D. van Vulpen, Merel A. Timmer, Michiel Coppens, Samantha C. Gouw, Marjolein Peters, Marieke J.H.A. Kruip, Marjon H. Cnossen, Jelmer J. Muis, Evelien S. van Hoorn, Lotte Haverman, Kathelijn Fischer

*Corresponding author for this work

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Abstract

Background: Legacy hemophilia-specific questionnaires are considered too long, show floor-/ceiling effects, and/or include irrelevant questions. Patient Reported Outcomes Measurement Information System (PROMIS) item banks, including Computer Adaptive Tests (CATs) and short forms, were designed for more efficient outcome assessment. Objectives: Evaluate the feasibility, measurement properties, and relevance of seven PROMIS CATs and two short forms in patients with hemophilia. Patients/Methods: In this cross-sectional study, Dutch adults with hemophilia completed nine PROMIS item banks electronically. Feasibility was assessed by number of items and floor/ceiling effects. Reliability was determined as the proportion of reliable scores (standard error ≤3.2). Construct validity was assessed by comparison with legacy instruments and expected differences between subgroups. Relevance of item banks was determined by proportions of limited scores. Results: Overall, 142 of 373 invited patients (mean age, 47 [range, 18-79], 49% severe hemophilia, 46% receiving prophylaxis) responded. Per CAT item bank, mean number of items answered varied from 5 (range, 3-12) to 9 (range, 5-12), with floor effects in pain interference (26% lowest scores) and depression (18% lowest scores). Construct validity and reliability were good for physical function, pain interference, satisfaction with social roles and activities, and fatigue. The CAT physical function showed the most limited scores (38%). The self-efficacy short forms showed ceiling effects (22%-28%) and no relation with the legacy instruments. Conclusions: The PROMIS CATs physical function, pain interference, satisfaction with social roles and activities, and fatigue are feasible, reliable, and valid alternatives to legacy instruments for patients with hemophilia, with few items and low floor-/ceiling effects.

Original languageEnglish
Article numbere12621
JournalResearch and Practice in Thrombosis and Haemostasis
Volume5
Issue number8
Early online date21 Nov 2021
DOIs
Publication statusPublished - 21 Nov 2021

Bibliographical note

Funding Information:
LFDvV has performed consultancy for Sobi, Tremeau, and CSL Behring and received a research grant from CSL Behring, all paid to the institution. MC reports having received research support from Bayer, UniQure, NovoNordisk, and Roche; and personal fees from Sobi, NovoNordisk, and CSL Behring outside of the submitted work. SG has received an unrestricted research grant from Sobi. MJHAK has received speaker’s fees from Roche and Sobi and has received research support from Bayer, Pfizer, Boehringer Ingelheim, Sobi, and Daiichi Sankyo. All grants, awards, and fees go to the Erasmus MC as institution. MHC has received investigator‐initiated research and travel grants as well as speaker fees over the years from the Netherlands Organisation for Scientific Research, the Netherlands Organization for Health Research and Development, the Dutch “Innovatiefonds Zorgverzekeraars,” Baxter/Baxalta/Shire/Takeda, Pfizer, Bayer Schering Pharma, CSL Behring, Sobi Biogen, Novo Nordisk, Novartis, and Nordic Pharma, and has served as a steering board member for Roche, Bayer and Novartis. All grants, awards, and fees go to the Erasmus MC as institution. LH is part of the Dutch‐Flemish PROMIS group. The Van Creveldkliniek has received speaker’s fees from Bayer, Baxter/Shire, SOBI/Biogen, CSL Behring, and NovoNordisk; has performed consultancy for Bayer, Biogen, CSL‐Behring, Freeline, NovoNordisk, Roche, and SOBI; and has received research support from Bayer, Baxter/Shire, Novo Nordisk, Pfizer, and Biogen for work done by KF. The other authors have no competing interests.

Publisher Copyright:
© 2021 The Authors. Research and Practice in Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis (ISTH).

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