Effectiveness of leniolisib in reducing infections among patients with activated phosphoinositide 3-kinase δ syndrome (APDS).
6 affiliations
- 1Division of Pediatric Allergy and Immunology, Duke University, Durham, NC, US.
- 2PANTHERx Rare Pharmacy, Pittsburgh, PA, US.
- 3Groupe d'analyse SRI, Montréal, QC, Canada.
- 4Division of Allergy, Immunology and Rheumatology, Johns Hopkins University, Baltimore, MD, US.
- 5Consultant for Pharming Healthcare, Inc, Warren, NJ, US.
- 6Pharming Healthcare, Inc, Warren, NJ, US. [email protected].
Abstract
APDS is a rare inborn error of immunity characterized by clinical manifestations, including recurrent infections. This real-world, retrospective analysis evaluated patient-reported infection rates before and after leniolisib initiation. Patients (aged ≥ 12 years) newly initiating leniolisib therapy were eligible for inclusion. Data were retrieved from PANTHERx Rare Pharmacy. Patient-reported infection rates during the baseline period (one year before leniolisib initiation) were compared with infection rates after leniolisib initiation (observation period; from initiation to end of data or death) using generalized estimating equations Poisson regression models with robust standard errors. Patients who had an infection assessment collected via a standardized questionnaire during the baseline and observation periods were included in the analysis (N = 71); subgroups for baseline infection rates (lower and higher than the median) and age (12-17 and ≥ 18 years) were also evaluated. The mean (SD) rate of infections per patient-year (PPY) decreased significantly-by 68%-from 5.8 (5.1) during baseline to 1.9 (2.0) in the observation period (rate ratio [RR], 0.32; 95% CI, 0.24-0.42; P < 0.001); infections PPY decreased by 38% in patients with low baseline infection rates (RR, 0.62; 95% CI, 0.43-0.89; P = 0.010) and 76% in those with high baseline infection rates (RR, 0.24; 95% CI, 0.17-0.34; P < 0.001); infections PPY decreased by 70% in those aged 12 - 17 years (RR, 0.30; 95% CI, 0.20-0.45; P < 0.001) and by 67% in those aged ≥ 18 years (RR, 0.33; 95% CI, 0.23-0.49; P < 0.001). Real-world evidence demonstrated that leniolisib treatment provided a significant reduction in infection rates overall and among subgroups.
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BibTeX
@article{patel2026effectiveness,
title = {{Effectiveness of leniolisib in reducing infections among patients with activated phosphoinositide 3-kinase δ syndrome (APDS).}},
author = {Niraj C Patel and Jessica Kuivinen and Sean D MacKnight and M Elizabeth M Younger and Guillaume Germain and Susannah Ripley and François Laliberté and Jesse Fishman and Amanda Harrington},
journal = {Immunologic research},
year = {2026},
month = oct,
doi = {10.1007/s12026-026-09863-2},
url = {https://europepmc.org/article/MED/42925385},
note = {CC BY},
}
RIS
TY - JOUR
TI - Effectiveness of leniolisib in reducing infections among patients with activated phosphoinositide 3-kinase δ syndrome (APDS).
AU - Niraj C Patel
AU - Jessica Kuivinen
AU - Sean D MacKnight
AU - M Elizabeth M Younger
AU - Guillaume Germain
AU - Susannah Ripley
AU - François Laliberté
AU - Jesse Fishman
AU - Amanda Harrington
PY - 2026
DA - 2026/10/24
JO - Immunologic research
DO - 10.1007/s12026-026-09863-2
UR - https://europepmc.org/article/MED/42925385
AB - APDS is a rare inborn error of immunity characterized by clinical manifestations, including recurrent infections. This real-world, retrospective analysis evaluated patient-reported infection rates before and after leniolisib initiation. Patients (aged ≥ 12 years) newly initiating leniolisib therapy were eligible for inclusion. Data were retrieved from PANTHERx Rare Pharmacy. Patient-reported infection rates during the baseline period (one year before leniolisib initiation) were compared with infection rates after leniolisib initiation (observation period; from initiation to end of data or death) using generalized estimating equations Poisson regression models with robust standard errors. Patients who had an infection assessment collected via a standardized questionnaire during the baseline and observation periods were included in the analysis (N = 71); subgroups for baseline infection rates (lower and higher than the median) and age (12-17 and ≥ 18 years) were also evaluated. The mean (SD) rate of infections per patient-year (PPY) decreased significantly-by 68%-from 5.8 (5.1) during baseline to 1.9 (2.0) in the observation period (rate ratio [RR], 0.32; 95% CI, 0.24-0.42; P < 0.001); infections PPY decreased by 38% in patients with low baseline infection rates (RR, 0.62; 95% CI, 0.43-0.89; P = 0.010) and 76% in those with high baseline infection rates (RR, 0.24; 95% CI, 0.17-0.34; P < 0.001); infections PPY decreased by 70% in those aged 12 - 17 years (RR, 0.30; 95% CI, 0.20-0.45; P < 0.001) and by 67% in those aged ≥ 18 years (RR, 0.33; 95% CI, 0.23-0.49; P < 0.001). Real-world evidence demonstrated that leniolisib treatment provided a significant reduction in infection rates overall and among subgroups.
SN - 0257-277X
SN - 1559-0755
ER -