Considerations when Selecting Patient-Reported Outcome Measures for Assessment of Health-Related Quality of Life in Patients with Pulmonary Hypertension: A Narrative Review
Authors:
Yarlas, A., Mathai, S. C., Nathan, S. D., DuBrock, H. M., Morland, K., Anderson, N., Kosinski, M., Lin, X., and Classi, P.
Abstract:
TOPIC IMPORTANCE: It is well established that pulmonary hypertension (PH) places a substantial burden on patients' health-related quality of life (HRQoL). As more effective treatments have been developed for this condition, there has been an increase in evaluating treatment benefit based on experiences reported by patients regarding their well-being and physical, social, and emotional functioning. A review of the published literature and clinical trials in PH was conducted to identify and evaluate patient-reported outcome measures (PROMs) that assess PH-specific HRQoL for use in clinical studies.
REVIEW FINDINGS: Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR), emPHasis-10, Living with Pulmonary Hypertension Questionnaire (LPHQ), and Pulmonary Arterial Hypertension - Symptoms And Impact (PAH-SYMPACT) were selected for in-depth evaluation with respect to their content validity, psychometric properties, interpretation guidelines, conceptual coverage, and administrative feasibility. Recommendations for clinical study endpoint strategies are provided. The review identified many strengths for each of the PROMs. Content development for all PROMs followed best practices, and any weaknesses in assessment of measurement properties were from a scarcity of available data. While conceptual coverage and patient burden varied greatly across the PROMs, each provided a unique strength relative to the others, and no one PROM was recommended as most appropriate across all contexts of use.
SUMMARY: Optimal endpoint selection for assessing PH-specific HRQoL thus requires consideration of the purpose and situation in which the assessment will be conducted. These recommendations should be considered as a snapshot of a quickly evolving landscape that should be updated as new information emerges.
