Hematopoietic stem cell transplantation for DOCK8 deficiency: durable immune reconstitution and long-term survival in 47 patients
Background DOCK8 deficiency is a severe autosomal recessive combined immunodeficiency characterized by recurrent infections, severe atopy, immune dysregulation, and an increased risk of malignancy. Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative treatment. We aimed to evaluate the lo...