Article
Interim analysis: Open-label extension study of leniolisib for patients with APDS.
The Journal of allergy and clinical immunology - 1 Jan 2024
Rao V Koneti, Kulm Elaine, Šedivá Anna, Plebani Alessandro, Schuetz Catharina, Shcherbina Anna, Dalm Virgil A, Trizzino Antonino, Zharankova Yulia, Webster Sharon, Orpia Alanvin, Körholz Julia, Lougaris Vassilios, Rodina Yulia, Radford Kath, Bradt Jason, Relan Anurag, Holland Steven M, Lenardo Michael J, Uzel Gulbu
Abstract excerpt
BACKGROUND: Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS; or p110δ-activating mutations causing senescent T cells, lymphadenopathy, and immunodeficiency) is an inborn error of immunity caused by PI3Kδ hyperactivity. Resultant immune deficiency and dysregulation lead to recurrent sinopulmonary infections, herpes viremia, autoimmunity, and lymphoproliferation. OBJECTIVE: Leniolisib, a selective...
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