Abstract
Background:
The Patient Reported Outcomes Measurement Information System (PROMIS) provides measures of health status that assess physical, mental, and social well-being from the patient perspective. As a complement to disease-specific measures, standardized PROMIS measures allow for comparisons across health attributes, health conditions, and with general populations. The validity and utility of PROMIS measures have been established in numerous adult and pediatric patient populations encompassing a broad range of diseases and chronic conditions but has not yet been evaluated in hemophilia.
The aims of this study are to: 1) describe PROMIS measures reported by adults with hemophilia during routine non-acute care encounters, 2) assess how measures compare to those reported by the PROMIS reference sample, and 3) evaluate the sensitivity of PROMIS to indicators of hemophilia clinical severity.
Methods:
Adult hemophilia patients were recruited to participate in this cross-sectional study during routine clinic visits that occurred at either of two U.S. Hemophilia Treatment Centers during October 2017 through June 2018. After consent was obtained, participants completed the PROMIS-29 on a tablet.
The PROMIS-29 short form includes 4 questions for each of 7 domains: depression, anxiety, physical function, pain interference, fatigue, sleep disturbance, and ability to participate in social roles and activities. Questions were based on the previous 7 days and used a 5-point response option. Responses were then scored on a T-score metric with a mean of 50 and a standard deviation (SD) of 10 based on the original PROMIS reference sample of US adults. Higher scores indicate worse severity for depression, anxiety, pain interference, fatigue, and sleep and better functioning for physical and social roles. Scores 5 or more away from 50 indicate at least mild impairment. Demographic characteristics were self-reported. Clinical characteristics including hemophilia severity (defined by factor levels) were obtained from medical records. Patients also completed the Brief Pain Inventory where they rated their average pain from 0-10. Responses were categorized as no (0), low (1-4), and high pain (5-10). Mean PROMIS measures were compared using T-tests or unadjusted linear regression.
Results:
Of 112 participants, 103 were males who completed the PROMIS questionnaire. Median age was 33 years and ranged from 18-75 years. Half had severe hemophilia and 60% reported using routine continuous factor prophylaxis. Two-thirds of patients reported no pain on average while another 14% reported high pain.
Overall PROMIS scores for each domain among the hemophilia cohort were similar to scores reported in the PROMIS normative sample with mean T-scores near 50 (domain means ranged from 45.7-52.9). However, PROMIS domain scores differed significantly by certain characteristics revealing sub-groups of patients who experienced significantly worse health compared to the normative reference sample. Patients reporting higher average pain reported worse health across all 7 domains compared to those with less pain. Physical function was most impacted by pain: those reporting the highest pain reported worse physical function (mean=36.8, SD=7.9) compared to those with low (mean=41.6, SD=8.3) and no pain (mean=50.7, SE=7.7). Patients with HIV reported higher depression, anxiety, pain, and fatigue, and worse physical function compared to patients without HIV. An emergency department visit in the past 6 months was also related to worse depression, anxiety, fatigue, sleep, and social functioning. Scores did not vary by hemophilia severity, prophylaxis use, or current inhibitor status and were similar to those reported by the referent population.
Conclusion:
The 7 PROMIS domains were sensitive to several adult hemophilia disease severity indicators in a non-acute care setting. Overall scores reported by patients with hemophilia generally mirrored the normative PROMIS sample scores. However, patients with hemophilia who experienced pain reported scores significantly worse than the normative sample. PROMIS instruments provide a potentially valuable tool to study the impact of hemophilia and suggest usefulness in research and clinical practice. Additional studies are needed to assess responsiveness in PROMIS score with changes in disease status over time and in women with hemophilia.
No relevant conflicts of interest to declare.
