Leniolisib and rapamycin in Activated PI3-Kinase-δ-syndrome: a retrospective ESID registry-based analysis
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
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| 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 type | 3 - Human Observational |
| Model system | Human (retrospective multicenter registry, n=95 APDS patients) |
| Journal | Journal of Allergy and Clinical Immunology |
| Year | 2026 |
| Peer reviewed | Yes |
| Record last updated | 2026-10-01 |
| Source | DOI 10.1016/j.jaci.2026.09.014 · PMID 42805311 |
Where this study fits
How this study connects to the pathway map, open questions, people and other studies in the Atlas.
People in the Atlas
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First author of a multicenter registry analysis comparing rapamycin and leniolisib in a rare immune disorder
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Senior author studying primary immunodeficiencies and their treatment
More: 107 other authors
No profile yet: Wolkewitz M, Kusters M, Uhlmann A, Guarnieri V, Abou-Chahla W, Adjaoud D, Aladjidi N, Armari-Alla C, Azarnoush S, Baris S, Barlogis V, Barzaghi F, Baumann U, Bloomfield M, Bodet D, Boutboul D, Boz V, Bozkurt S, Bucciol G, Cancrini C, Catherinot E, Cavazzana M, Cheminant M, Chinello M, Ciznar P, Conti F, Costagliola G, Dalm V, D'Aveni-Piney M, Ekwall O, Eren E, Eric Z, Farmaki E, Fasth A, Fieschi C, Fischer A, Fournier B, Galicier L, Ghosh S, Gkantaras A, Hatchuel Y, Hilfanova A, Hoenig M, Jeziorski E, Jolles S, Karakoc-Aydiner E, Kilic SS, Kiykim A, Körholz J, Le Guenno G, Licciardi F, Lodi L, Lougaris V, Lucena JM, Malphettes M, Marzollo A, Masmas TN, Meinhardt A, Merlin E, Meyts I, Milota T, Moratti M, Moussouni N, Neth O, Nunes-Gomes C, Ocak S, Olbrich P, Yücel EÖ, Özen A, Schmid JP, Palma P, Pellier-Landreau I, Perlat A, Picard C, Prader S, Ricci S, Riviere J, Santilli V, Sarrot-Reynauld F, Schleinitz N, Schrijvers R, Schulz A, Schuetz C, Sediva A, Semeraro M, Sharapova S, Sogkas G, Soomann M, Palacín PS, Suarez F, Tessarin G, Tommasini A, Trück J, van Aerde K, van Montfrans J, Vermont C, Wustrau K, Zharankova Y, Moshous D, Neven B, Seidel MG, Chandra A, Thalhammer J, Kracker S, Ehl S, Mahlaoui N, Elfeky R
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Topics
Extracted findings
These fields were extracted from the paper with AI assistance and have not been checked against the source. Read them as a pointer into the paper, not as curated claims; where they disagree with the curated summary above, the curated summary stands.
| Intervention | Rapamycin (off-label) and/or leniolisib (PI3Kdelta inhibitor) |
| Target | PI3Kdelta / mTOR |
| Model | Human (retrospective registry, 95 APDS patients) |
| Effect | Both drugs were associated with similar response rates for lymphoproliferation (67% vs 64%); treatment-limiting adverse events occurred only with rapamycin. |
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