A study of myeloablative hematopoietic cell transplantation (HCT) performed in children under 3 years old has found that although transplant-related mortality at 10 years is only 5%, recipients require monitoring for late complications. The researchers collected data on late effects in 717 patients undergoing HCT between 1987 and 2012 who survived at least one year following HCT for a hematologic malignancy. Transplant outcomes were reported to CIBMTR (Center for International Blood and Marrow Transplant Research). The median follow-up of survivors was 99 months (range, 12-317).
Overall and disease-free survival at 10 years were 87% and 84%, respectively. Grade II-IV acute GVHD developed in 35% of patients, and chronic GVHD developed in 32% of patients. Late complications included cataracts 10% (95% CI 7-12%), growth hormone deficiency/growth disturbance 15% (95% CI 12-19%), hypothyroidism 9% (95% CI 7-12%), and stroke/seizure 5% (95% CI 3-7%). A new malignancy developed in 3.6% of patients. The researchers concluded that late effects are common in this population, and surveillance for long-term toxicities is needed for survivors.