Hematopoietic Stem Cell Transplantation Achieves Sustained Remission in Pediatric RAS-Associated Autoimmune Lymphoproliferative Disorder with Life-Threatening Complications: a Single-Center Case Series and Literature Review

RAS-associated autoimmune lymphoproliferative disorder (RALD) is a rare disease caused by somatic mutations in KRAS or NRAS genes and is characterized by hepatosplenomegaly, cytopenia, and autoimmune manifestations. Current conventional therapies often yield only transient remission. Herein, we report three pediatric cases of refractory RALD that were successfully treated with allogeneic hematopoietic stem cell transplantation (HSCT). After 21 to 78 months of follow-up, all patients achieved complete recovery of hematopoietic and immune functions, suggesting cure. A concomitant review of the literature identified four additional published RALD cases treated with HSCT, all of whom were successfully rescued. Collectively, these cases expand the known clinical spectrum of RALD and provide preliminary evidence supporting HSCT as a potentially curative option for refractory disease.