Treatment of Severe Forms of LPS-Responsive Beige-like Anchor Protein (LRBA) Deficiency by Allogeneic Hematopoietic Stem Cell Transplantation

Capsule summary: LRBA deficiency, a life-threatening immunodeficiency/autoimmunity disorder, was treated by HSCT in 12 patients, four of whom died from transplant-related causes. Six patients were cured without further need of immunosuppression, and two are in partial remission.

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