Abstract
Background
Hemophilia is marked by frequent joint bleeding, resulting in pain and functional impairment.
Objective
This study aimed to assess the reliability of five patient-reported outcome (PRO) instruments in people with hemophilia (PWH) in a non-bleeding state.
Methods
Adult male PWH of any severity and inhibitor status, with a history of joint pain or bleeding, completed a pain history and five PRO instruments (EQ-5D-5L, Brief Pain Inventory v2 [BPI], International Physical Activity Questionnaire [IPAQ], Short Form 36 Health Survey v2 [SF-36v2], and Hemophilia Activities List [HAL]) during their routine comprehensive care visit. Patients were approached to complete the PRO instruments again at the end of their visit while in a similar non-bleeding state. Concordance of individual questionnaire items and correlation between domain scores were assessed using intra-class correlation coefficient (ICC).
Results
Participants completing the retest (n=164) had a median age of 33.9 years. Median time for completion of the initial survey with PRO instruments was 36.0 minutes and for the five PRO instruments, median retest time was 21.0 minutes. The majority of participants had hemophilia A (74.4%), were white and non-Hispanic (72.6%), and self-reported arthritis/bone/joint problems (61%). Median/mean test-retest concordance was EQ-5D-5L 80.0%/79.1%, BPI 54.5%/58.9%, IPAQ 100%/100%, SF-36v2 77.8%/76.4%, and HAL 77.4%/75.9%. ICCs for test-retest reliability were EQ-5D-5L index 0.890; BPI – severity 0.950; BPI – interference 0.920; IPAQ total activity 0.940; SF-36v2 overall health 0.910; HAL total score 0.970.
Conclusion
All five PRO scales showed acceptable test-retest reliability in adult PWH. Therefore, the choice of instrument to be used for research or clinical care should be driven by instrument characteristics other than reliability.
Supplementary material
Munson Medical Center
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Emory University
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St Vincent Hospital and Health Care Center, Inc.
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Western Institutional Review Board
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Henry Ford Health System
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Vanderbilt University
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Western Institutional Review Board
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University of Colorado Multiple
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Michigan State University
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University of Minnesota
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Rush University Medical Center
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Wake Forest University Health Services
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Georgetown University
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Acknowledgments
The clinical study analyzed was sponsored by Novo Nordisk A/S. Statistical analysis plan and analyses were provided by Jennifer James, MS at Quintiles Outcome under direction of Kari Kastango, PhD, and Pattra Mattox, MS. Writing assistance was provided by Shawn Keogan, PhD, of ETHOS Health Communications in Yardley, Pennsylvania, and was supported financially by Novo Nordisk Inc., Plainsboro, New Jersey, in compliance with international Good Publication Practice guidelines. The abstract of this paper was presented at the International Society of Thrombosis and Haemosta-sis 2015 congress as a poster presentation. The poster’s abstract was published in the Journal of Thrombosis and Haemostasis.
Disclosure
CL Kempton: grant/research support from Novo Nordisk Inc.; consultant for Baxter, Biogen, CSL Behring, Kedrion. M Wang: consultant for Novo Nordisk Inc.. M Recht: grant/research support from Baxter, Biogen Idec, Novo Nordisk Inc., Pfizer; consultant for Biogen, Kedrion, Novo Nordisk Inc. A Neff: grant/research support from Novo Nordisk Inc.; consultant for Alexion, Baxter, CSL Behring, Novo Nordisk Inc. AD Shapiro: consultant for Baxter BioScience, Novo Nordisk Inc., Biogen Idec, ProMetic Life Sciences, Kedrion Biopharma. A Soni: speakers bureau participant for CSL Behring and Novo Nor-disk Inc.; consultant for Bayer. R Kulkarni: grant/research support from Novo Nordisk Inc., Biogen, Baxter, Bayer; consultant for Novo Nordisk Inc., Biogen, Bayer, Baxter, Kedrion, BPL, Pfizer. TW Buckner: consultant for Bax-alta US, Genentech, and Novo Nordisk Inc. K Batt: grant/research support from Novo Nordisk Inc.; scientific advisor for Precision Health Economics. NN Iyer and DL Cooper: employees of Novo Nordisk Inc., the sponsor of the study. The authors report no other conflicts of interest in this work.