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PI3K-delta syndrome; PASLI disease

Pathology: - hyperactive signaling of PI3K-delta Clinical manifestations: - infections - lymphocyte proliferation (lymphocytosis) - autoimmunity - enteropathy - bronchiectasis - increased risk of lymphoma - early mortality Management: - leniolisib (Joenja) FDA-approved

General

immunologic disease syndrome

References

  1. Rao VK et al A randomized, placebo-controlled phase 3 trial of the PI3Kdelta inhibitor leniolisib for activated PI3Kdelta syndrome Blood. 2023 Mar 2;141(9):971-983 PMID: 36399712 PMCID: PMC10163280 Free PMC article