Real-World Health Care Resource Utilization and Costs Among Patients with Activated Phosphoinositide 3-Kinase Delta (PI3Kδ) Syndrome in the United States
Advances in Therapy, 2025
Introduction
This study sought to describe health care resource utilization (HRU) and treatment use among patients with activated phosphoinositide 3-kinase delta syndrome (APDS) and assess the incremental HRU and health care costs associated with APDS relative to a non-APDS control cohort.
Methods
Patients with APDS were identified via Symphony Health Solutions Integrated Dataverse database. Controls were selected using a 10:1 ratio and matched to patients with APDS. Rates of HRU (hospitalizations, emergency department [ED] visits, outpatient [OP] visits, other visits) per person-year (PPY) and annual health care costs were compared using rate ratios (RRs) estimated from Poisson regression models and cost ratios (CRs) estimated from gamma regression models, respectively. Use of symptomatic treatments associated with APDS was also assessed.
Results
Mean (standard deviation [SD]) age of patients with APDS (n = 42) and controls (n = 420) was 16.0 (15.1) and 16.9 (17.4) years, respectively. Patients with APDS had significantly higher PPY rates of all-cause hospitalizations (RR 4.31, 95% confidence interval [CI] 1.83-8.03; p < 0.001) and OP visits (RR 1.77, 95% CI 1.24-2.61; p < 0.001) vs controls, with OP visits driven by higher rates of OP hospital visits (RR 5.32, 95% CI 3.30-8.29; p < 0.001). Patients with APDS had annual all-cause total health care costs 10.54 times greater (95% CI 4.56-20.67; p < 0.001) than controls, driven by higher medical costs (CR 12.39, 95% CI 5.04-25.82; p < 0.001). Significantly higher costs for hospitalizations and ED, OP (office/clinic and hospital), and other visits were observed in patients with APDS vs controls. HRU rates and costs (except other visits) remained significantly higher in patients with APDS vs controls after excluding visits related to immunoglobulin replacement therapy.
Conclusion
Higher HRU and health care costs among patients with APDS vs controls reflect the high clinical and economic burden of APDS.
Authors
Rider NL, Laliberté F, Germain G, Urosevic A, Mahendran M, Harrington A