Real-World Health Care Resource Utilization and Costs Among Patients with Activated Phosphoinositide 3-Kinase Delta (PI3Kδ) Syndrome in the United States.

IF 4 3区医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL

Advances in Therapy Pub Date : 2025-10-08 DOI:10.1007/s12325-025-03377-3

Nicholas L Rider, François Laliberté, Guillaume Germain, Ana Urosevic, Malena Mahendran, Amanda Harrington

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引用次数: 0

Abstract

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.

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美国活化磷酸肌肽3-激酶δ (PI3Kδ)综合征患者的现实世界卫生保健资源利用和成本

本研究旨在描述活化磷酸肌肽3-激酶δ综合征（APDS）患者的医疗保健资源利用（HRU）和治疗使用情况，并评估相对于非APDS对照队列，与APDS相关的HRU增量和医疗保健费用。方法：通过Symphony Health Solutions Integrated Dataverse数据库对APDS患者进行识别。对照以10:1的比例选择，并与APDS患者相匹配。分别使用泊松回归模型估计的比率比（rr）和伽玛回归模型估计的成本比（cr）比较HRU（住院次数、急诊次数、门诊次数、其他次数）和年卫生保健费用。还评估了与APDS相关的对症治疗的使用情况。结果：APDS患者（n = 42）和对照组（n = 420）的平均（标准差[SD]）年龄分别为16.0（15.1）岁和16.9（17.4）岁。APDS患者的全因住院PPY率显著高于对照组（RR 4.31, 95%可信区间[CI] 1.83-8.03; p）结论：APDS患者的HRU和医疗费用高于对照组，反映了APDS患者较高的临床和经济负担。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Advances in Therapy 医学-药学

CiteScore

7.20

自引率

2.60%

发文量

353

审稿时长

6-12 weeks

期刊介绍： Advances in Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all therapeutic areas. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Advances in Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research.