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Leniolisib and rapamycin in Activated PI3-Kinase-δ-syndrome: a retrospective ESID registry-based analysis

Rivalta B, Maccari ME et al. · 2026 · Journal of Allergy and Clinical Immunology · Atlas ID RIV2026

What this study shows

In a 95-patient ESID registry of Activated PI3Kδ Syndrome (APDS), rapamycin and the targeted PI3Kδ inhibitor leniolisib produced comparable overall response rates for lymphoproliferation (67% vs 64%), but treatment-limiting adverse events occurred only in the rapamycin-treated group; lymphoma cases were observed during follow-up in the cohort.

Abstract

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Activated PI3K-delta syndrome (APDS) is a rare inborn error of immunity characterized by variable immune dysregulation, recurrent infections, and increased risk of malignancy. Clinical management is challenging. Rapamycin has been widely used off-label to control immune dysregulation, while the selective PI3Kδ inhibitor leniolisib has recently become available as a disease-specific targeted therapy. Real-world data on these treatment approaches remain limited.

Read the full abstract on PubMed →

At a glance

Evidence type H Human study Marked H because it is direct evidence from a human clinical trial or human cohort; the code names the kind of study, not its quality -- a small, well-run trial is still H.
Study type3 - Human Observational
Model systemHuman (retrospective multicenter registry, n=95 APDS patients)
JournalJournal of Allergy and Clinical Immunology
Year2026
Peer reviewedYes
Record last updated2026-09-23
SourceDOI 10.1016/j.jaci.2026.09.014 · PMID 42805311

Cite this paper

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Rivalta, B., et al. (2026). Leniolisib and rapamycin in Activated PI3-Kinase-δ-syndrome: a retrospective ESID registry-based analysis. Journal of Allergy and Clinical Immunology. https://doi.org/10.1016/j.jaci.2026.09.014

@article{RIV2026,
  author       = {Rivalta, B. and Maccari, M. E. and others},
  title        = {{Leniolisib and rapamycin in Activated PI3-Kinase-δ-syndrome: a retrospective ESID registry-based analysis}},
  journal      = {Journal of Allergy and Clinical Immunology},
  year         = {2026},
  doi          = {10.1016/j.jaci.2026.09.014},
  note         = {PMID: 42805311},
}

Cite this Atlas record

The record is the Atlas's own work — the evidence label, the extracted findings and the links. It is cited as part of the dataset, not as the paper. 

Barton, O. (2026). Oliver's mTOR Atlas (record RIV2026) [Data set]. https://mtor-atlas.org/study/RIV2026/ · Dataset DOI 10.5281/zenodo.22059963

@misc{atlas_RIV2026,
  author       = {Barton, Oliver},
  title        = {{Oliver's mTOR Atlas}, record RIV2026},
  howpublished = {Data set},
  year         = {2026},
  url          = {https://mtor-atlas.org/study/RIV2026/},
  doi          = {10.5281/zenodo.22059963}
}