Exploring Patterns of Treatment Utilization and Promis Scores in a Home-Reported Outcomes Study for Individuals with Sickle Cell Disease (SCD)

OBJECTIVES: Patient-reported outcome measures can be utilized to understand general disease burden. This research aims to explore baseline PROMIS Global Health Scale results and patient-reported treatment utilization in a remote observational study for individuals with sickle cell disease (SCD).

METHODS: A remote observational study collecting home-reported outcomes (HROs) within a mobile-based platform allowed participants to track symptom severity and treatment utilization over a 3 month period. Participants at baseline answered the PROMIS Global Health v1.2 and were further categorized based on self-reported health rating. Group A indicated ‘poor’ or ‘fair’ health. Group B indicated ‘good’, ‘very good’, ‘excellent’ health. Average scores of Global Physical Health (GPH) and Global Mental Health (GMH) sub-scales were calculated. Summary statistics of each group’s treatment utilization was analyzed and further categorized by drug class according to the National Drug Code database.

RESULTS: 59 of 82 enrollees completed the PROMIS questionnaire at baseline. For Group A (n= 32), the mean GPH score was 36.3 (STD: 6.0) and the mean GMH was 40.7 (STD: 7.2). For Group B (n=27), the mean GPH score was 45.8 (STD: 7.4) and the mean GMH was 45.9 (STD: 8.5). The top treatment categories tracked in Group A included opioids, antimetabolites, and NSAIDs. 29 participants (90.6%) of participants in Group A skipped at least one treatment. The top reason for skipping an opioid treatment for Group A was ‘Wasn’t needed’ (10.3%). The top treatment categories tracked in Group B included a Hemoglobin S polymerization inhibitor, antimetabolites, and NSAIDs. All 27 participants in Group B tracked skipping a treatment at least once. The top reason for skipping was ‘Forgot today’ (25.9%).

CONCLUSIONS: Patient-reported outcome measures such as PROMIS can provide a point-in-time overview on the state of health for individuals. The utilization of HROs can further contextualize disease burden for individuals with SCD and other rare conditions.