Home > Haematology > EHA 2022 > PI3Kδ inhibitor leniolisib improves symptoms in patients with APDS/PASLI

PI3Kδ inhibitor leniolisib improves symptoms in patients with APDS/PASLI

Presented By
Dr Koneti Rao, National Institutes of Health, MD, USA
Conference
EHA 2022
PI3Kδ inhibitor leniolisib is safe, increases naïve B cells, decreases lymphadenopathy, decreases spleen size, and improves cytopenia in patients with APDS/PASLI, results from a phase 3 trial demonstrate. APDS (activated PI3Kδ syndrome), also known as PASLI (p110δ-activating mutation causing senescent T cells, lymphadenopathy, and immunodeficiency) is a rare primary immunodeficiency caused by pathogenic variants in PIK3CD or PIK3R1, leading to PI3Kδ signalling hyperactivity. Clinical characteristics of APDS are lymphoproliferation, auto-immunity, immunodeficiency, and increased risk of lymphoma. Current treatment options include stem cell transplantation, immunoglobulin replacement therapy, and empirical treatment such as immunomodulatory, antibiotic, and antiviral therapy for symptom relief. Leniolisib is a small-molecule inhibitor of hyperactive PI3Kδ signalling [1]. Dr Koneti Rao (National Institute of Health, MD, USA) presented...


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