Myeloablative Matched Sibling Donor Hematopoietic Stem Cell Transplantation as a Curative Treatment for Congenital Erythropoietic Porphyria: A Cohort Study from Low-Middle Income Country.

). Peri-transplant period was complicated by febrile neutropenia and mucositis. Neutrophil engraftment was achieved at a median of Day+16 (range 14-18) and platelet engraftment at median of Day+21 (range 18-21). At a median follow up of 240 days (range 116-2555), all patients were alive, transfusion independent with complete resolution of photosensitivity. Additionally, serum and urine porphyrin levels became normal in all patients. This cohort, though very small supports the efficacy of early myeloablative Hematopoietic stem cell transplant with matched sibling donors to prevent irreversible tissue damage and significantly improve clinical outcomes.