Real-World Health Care Resource Utilization and Costs Among Patients with Activated Phosphoinositide 3-Kinase Delta (PI3Kδ) Syndrome in the United States
Bibliographic record
Abstract
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. 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. 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. Higher HRU and health care costs among patients with APDS vs controls reflect the high clinical and economic burden of APDS. Activated phosphoinositide 3-kinase delta syndrome (APDS) is a rare disease caused by harmful changes in certain genes that lead to problems with how the immune system works. To better understand the burden associated with APDS, this study estimated the use of health care resources and health care costs incurred by patients with APDS compared with those without APDS in the USA. This study identified 42 patients with APDS and matched them to 420 patients without APDS from the Symphony Health Integrated Dataverse database. Patients with APDS had four times the rate of hospitalizations and close to two times the rate of outpatient visits compared with those without APDS. Total health care costs were nearly 11 times greater in patients with APDS than those without APDS. Costs for hospitalizations, emergency department visits, outpatient visits, and other visits were much higher among patients with APDS than those without APDS and remained much higher even when the cost of immunoglobulin replacement therapy was removed. Higher health care resource use and health care costs among patients with APDS compared with patients without APDS highlight the substantial clinical and economic burden of APDS.
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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.000 | 0.000 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one teacher head, not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".