Twenty-seven year follow-up of Fanconi Anemia from REFAIN (Registry for Fanconi Anemia in India)-Finite disappointment and infinite hope

摘要

Nineteen children were included in study with a median age atdiagnosis of eight years (1 year to 18 years). The median total leukocytecount at presentation was 10,200 (4500 to 500,000). B-myeloid in fifteen,T-myeloid in two and B/T lymphoid in two children were the immuno-phenotype documented. Eleven patients received AML induction therapyand 8 patients received ALL induction therapy. Eight out of thirteen chil-dren who underwent allogeneic hematopoietic stem cell transplantationfrom the best available donor and two of the five patients who did notundergo HSCT are alive. The overall survival in our cohort was 42.1%. Themost common cause of mortality was relapse (66%) followed by regimenrelated toxicity (33%).