Registry data compare rapamycin and leniolisib in APDS
A retrospective analysis of the ESID-APDS registry evaluated real-world use of rapamycin (81 patients) and leniolisib (28 patients) in Activated PI3K-delta syndrome, primarily for lymphoproliferation. The study reported overall response rates of 67% with rapamycin and 64% with leniolisib for lymphoproliferation, with lower response rates (35% and 50%, respectively) for other disease manifestations; treatment-limiting adverse events occurred only in the rapamycin group, and lymphoma cases were observed during follow-up. The authors state that prospective long-term studies are needed to clarify the role of these therapies and their impact on malignancy risk.
Most relevant for: Patients with Activated PI3K-delta syndrome (APDS) being managed for immune dysregulation and lymphoproliferation.
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