Construct validity of patient-reported outcome instruments in US adults with hemophilia: results from the Pain, Functional Impairment, and Quality of life (P-FiQ) study

Abstract

BackgroundPeople with hemophilia (PWH) experience frequent joint bleeding, resulting in pain and functional impairment. Generic and disease-specific patient-reported outcome (PRO) instruments have been used in clinical studies, but rarely in the comprehensive hemophilia care setting.ObjectiveThe objective of this study was to assess construct validity of PRO instruments measuring pain, functional impairment, and health-related quality of life in US PWH with a history of joint pain/bleeding.MethodsAdult male PWH completed 4 PRO instruments (EQ-5D-5L with visual analog scale, Brief Pain Inventory v2 Short Form [BPI], SF-36v2, Hemophilia Activities List [HAL]) and underwent a musculoskeletal examination (Hemophilia Joint Health Score v2.1 [HJHS]). Construct validity between index and domain scores was evaluated by Pearson product-moment correlation coefficient.ResultsA total of 381 PWH were enrolled. EQ-5D-5L Mobility correlated with BPI, SF-36v2, and HAL domains related to pain, physical function, and activity of the lower extremities. EQ-5D-5L Self-Care correlated only with HAL Self-Care. EQ-5D-5L Usual Activities correlated with BPI Pain Interference and domains within SF-36v2 and HAL related to pain and physical function/activities (particularly those involving the lower extremities). EQ-5D-5L Pain/Discomfort correlated with Bodily Pain and Physical Summary on SF-36v2, HAL Overall Activity, and all BPI pain domains. EQ-5D-5L Anxiety/Depression correlated with social/emotional/mental aspects of SF-36v2. On BPI, most pain domains correlated with Bodily Pain and Physical Health Summary on SF-36v2 and Overall Activity on HAL. On SF-36v2, Physical Functioning, Role Physical, Bodily Pain, and Physical Health summary scores correlated with all the domains of HAL except Self-Care. For HJHS, Ankle and Total scores correlated with SF-36v2 Physical Functioning and HAL Lying/Sitting, Leg Function, Complex Lower Extremity Activity, and Overall Activity.ConclusionAll PRO instruments have high construct validity but provide different levels of detail in describing effects of hemophilia. Instrument choice may depend on individuals’ symptoms, treatment planning goals, or outcome tracking research objectives, with consideration for administrative burden.

Patient‐reported outcomes and joint status across subgroups of US adults with hemophilia with varying characteristics: Results from the Pain, Functional Impairment, and Quality of Life (P‐FiQ) study

“…An important aim of the study was to validate the use of generic and hemophilia‐specific PRO instruments in measuring pain, functional impairment, and HRQoL in PWH. Previous reports from P‐FiQ demonstrated validity and reliability of PRO instruments in this study population. In addition, significant differences in PRO scores and joint status as measured by the Hemophilia Joint Health Score v2.1 (HJHS) were observed between various known groups (eg, self‐reported pain levels), also supporting sufficient known‐group validity .…”

Assessments of pain, functional impairment, anxiety, and depression in US adults with hemophilia across patient‐reported outcome instruments in the Pain, Functional Impairment, and Quality of Life (P‐FiQ) study

“…This analysis incorporates multiple PRO instruments to evaluate the prevalence and characteristics of pain, functional impairment, and anxiety and depression in 381 adult males with hemophilia enrolled in the P‐FiQ study. Assessment of the reliability and validity of PRO instruments in previous P‐FiQ publications demonstrates the accuracy of these tools in measuring pain and HRQoL in this study population . Here, we report trends across the PRO instruments specific to patient‐reported pain, functional impairment, and anxiety and depression among P‐FiQ respondents.…”

Factors associated with pain severity, pain interference, and perception of functional abilities independent of joint status in US adults with hemophilia: Multivariable analysis of the Pain, Functional Impairment, and Quality of Life (P‐FiQ) study

“…Previously published data from P‐FiQ demonstrated the prevalence of pain in the study population, with 32% of respondents reporting having experienced both acute and chronic pain in the prior 6 months . In other reports from P‐FiQ, convergent validity, known‐group validity, internal consistency, item‐total correlation, and test‐retest reliability of 5 PRO instruments and the Hemophilia Joint Health Score v2.1 (HJHS) were demonstrated in the P‐FiQ study population, indicating the potential utility of these instruments for assessing pain and functional impairment and their effect on overall quality of life in PWH . In addition, important correlations were found between participant characteristics and pain or functional outcomes.…”

“…For example, respondents who reported both acute and chronic pain were more likely to report depression compared with respondents who had acute or chronic pain only, or no pain . Additionally, joint disease as measured by HJHS correlated with PRO scores related to functional impairment and pain …”