Evidence map›Paper›PMID 41060524›Full record

ArticleAdvances in therapy2025

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

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

Abstract read
In one paragraph

Article in Advances in therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors.

Nicholas L RiderVirginia Tech Carilion School of Medicine, Roanoke, VA, USA.
François LalibertéGroupe d'analyse, SRI, Montréal, QC, Canada.
Guillaume GermainGroupe d'analyse, SRI, Montréal, QC, Canada.
Ana UrosevicGroupe d'analyse, SRI, Montréal, QC, Canada.
Malena MahendranGroupe d'analyse, SRI, Montréal, QC, Canada.
Amanda HarringtonPharming Healthcare, Inc., 10 Independence Blvd, #300, Warren, NJ, 07059, USA. A.Harrington@pharming.com.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionThis 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.

methodsPatients 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.

resultsMean (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.

conclusionHigher HRU and health care costs among patients with APDS vs controls reflect the high clinical and economic burden of APDS.

Indexed as

Health Care CostsHealth ResourcesPatient Acceptance of Health CarePrimary Immunodeficiency DiseasesAdolescentAdultAmbulatory CareCase-Control StudiesChildClass I Phosphatidylinositol 3-KinasesEmergency Service, HospitalFemaleHospitalizationHumansMaleMiddle AgedClass I Phosphatidylinositol 3-KinasesActivated phosphoinositide 3-kinase delta syndrome (APDS)Burden of illnessCostsHealth care resource utilizationImmunodeficiencyInborn error of immunityReal-world evidence

Identifiers

PMID41060524
PMCPMC12618449

What Socratic holds

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LicenceCC BY-NC
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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.