Late effects of total body irradiation and hematopoietic stem cell transplant in children under 3 years of age.

BACKGROUND: Total body irradiation (TBI) is an important component of hematopoietic stem cell transplant (SCT) for pediatric malignancies. With increasing survival rates, late effects of SCT become more important. Younger children may be at particular risk of late effects of radiation and SCT.
METHODS: We retrospectively reviewed outcomes of children less than 3 years of age who received TBI as part of their preparative regimen for SCT at Children's Hospital Colorado. Clinical information including the date of last follow-up, most recent lab values, and physiologic tests were extracted from the medical record.
RESULTS: Of 81 patients who underwent SCT, 19 received TBI and of those, 15 were long-term survivors available for review. Late effects occurring in greater than 50% of the children included abnormalities involving endocrine, metabolic, renal, cataracts, and neurocognitive systems. Other organs involved less commonly included liver, skeletal, and cardiac abnormalities. Solid tumors were a rare finding with only one patient developing a benign osteochondroma and no identified secondary malignancies.
CONCLUSIONS: TBI has been shown to be an important part of the preparative regimen for patients undergoing SCT. Our results, similar to other studies, suggest TBI in patients less than 3 years of age will likely result in multi-organ dysfunction including endocrine, metabolic, renal, eye, and neurocognitive abnormalities. A longitudinal study with standardized testing of these systems would further clarify the late effects concerns in this patient population.