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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

Authors Buckner TW, Wang M, Cooper DL, Iyer NN, Kempton CL

Received 9 May 2017

Accepted for publication 7 September 2017

Published 11 October 2017 Volume 2017:11 Pages 1745—1753

DOI https://doi.org/10.2147/PPA.S141392

Checked for plagiarism Yes

Review by Single-blind

Peer reviewers approved by Dr Amy Norman

Peer reviewer comments 3

Editor who approved publication: Dr Johnny Chen

Tyler W Buckner,1 Michael Wang,1 David L Cooper,2 Neeraj N Iyer,2 Christine L Kempton3

1Hemophilia and Thrombosis Center, University of Colorado School of Medicine, Aurora, CO, 2Novo Nordisk Inc., Clinical, Medical, and Regulatory Affairs, Plainsboro, NJ, 3Departments of Pediatrics and Hematology and Medical Oncology, Emory University School of Medicine, Atlanta, GA, USA

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.

Keywords: hemophilia, pain, functional impairment, quality of life, patient-reported outcome, joint health

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