International retrospective study of allogeneic hematopoietic cell transplantation for activated PI3K-delta syndrome

Abstract excerpt

BACKGROUND: Activated phosphoinositide 3-kinase delta syndrome (APDS) is a combined immunodeficiency with a heterogeneous phenotype considered reversible by allogeneic hematopoietic cell transplantation (HCT). OBJECTIVES: This study sought to characterize HCT outcomes in APDS. METHODS: Retrospective data were collected on 57 patients with APDS1/2 (median age, 13 years; range, 2-66 years) who underwent HCT....

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• Share to Hematopoietic Stem Cell Transplantation
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