Known-group validity of patient-reported outcome instruments and hemophilia joint health score v2.1 in US adults with hemophilia: results from the Pain, Functional Impairment, and Quality of life (P-FiQ) study

• Type of Material: Article
• Authors: Tyler W. Buckner, University of Colorado; Michael Wang, University of Colorado; David L. Cooper, Novo Nordisk Inc.; Neeraj N. Iyer, Novo Nordisk Inc.; Christine Kempton, Emory University
• Language: English
• Date: 2017-01-01
• Publisher: Dove Medical Press
• Publication Version: Final Published Version
• Copyright Statement: © 2017 Buckner et al.
• License: Creative Commons Attribution-Noncommercial 3.0 Unported
• Final Published Version (URL): http://dx.doi.org/10.2147/PPA.S141392
• Title of Journal or Parent Work: Patient Preference and Adherence
• ISSN: 1177-889X
• Volume: 11
• Start Page: 1745
• End Page: 1753
• Grant/Funding Information: Writing assistance was provided by Anna Abt, PhD, of ETHOS Health Communications, Yardley, Pennsylvania, and was supported financially by Novo Nordisk Inc., Plainsboro, New Jersey, in compliance with international Good Publication Practice guidelines.; The P-FiQ study was managed by Quintiles Real World and Late Phase, Boston, Massachusetts, and Rockville, Maryland, with statistical analyses provided by Jennifer James, Senior Biostatistician, and was supported financially by Novo Nordisk Inc.
• Keywords: patient-reported outcome; BLEEDING DISORDERS; Medicine, General & Internal; MANAGEMENT; PHYSICAL-ACTIVITY; ACTIVITIES LIST; pain; Science & Technology; RELIABILITY; hemophilia; Life Sciences & Biomedicine; quality of life; ABILITIES; functional impairment; joint health; General & Internal Medicine
• Research Categories: Health Sciences, Medicine and Surgery; Health Sciences, Oncology

Abstract

Background: The Pain, Functional Impairment, and Quality of Life (P-FiQ) study was an observational, cross-sectional assessment of the impact of pain on functional impairment and quality of life in adults with hemophilia in the United States who experience joint pain or bleeding. Objective: To describe known-groups validity of assessment tools used in the P-FiQ study. Patients and methods: Participants completed 5 patient-reported outcome (PRO) instruments (5-level EuroQoL 5-dimensional questionnaire [EQ-5D-5L] with visual analog scale [VAS] , Brief Pain Inventory v2 Short Form [BPI], International Physical Activity Questionnaire [IPAQ] , Short-Form Health Survey [SF-36v2], and Hemophilia Activities List [HAL] ) and underwent a musculoskeletal examination (Hemophilia Joint Health Score [HJHS]) during a routine clinical visit. Results: P-FiQ enrolled 381 adults with hemophilia (median age, 34 years). Participants were predominantly white/non-Hispanic (69.2%), 75% had congenital hemophilia A, and 70.5% had severe hemophilia. Most (n=310) reported bleeding within the past 6 months (mean [SD] number of bleeds, 7.1 [13.00]). All instruments discriminated between relevant known (site-or self-reported) participant groups. Domains related to pain on EQ-5D-5L, BPI, and SF-36v2 discriminated self-reported pain (acute/chronic/both; P,0.05), domains related to functional impairment on IPAQ, SF-36v2, and HAL discriminated self-reported functional impairment (restricted/unrestricted; P,0.05), and domains related to mental health on the EQ-5D-5L and SF-36v2 discriminated self-reported anxiety/depression (yes/no; P,0.01). HJHS ankle and global gait domains and global score discriminated self-reported arthritis/bone/joint problems, percentage of lifetime on prophylaxis, current treatment regimen, and hemophilia severity (P,0.01); knee and elbow domains discriminated all of these (P,0.01) except for current treatment regimen. Conclusion: All assessment tools demonstrated known-group validity and may have practical applicability in evaluating adults with hemophilia in clinical and research settings in the United States.