30% of Very Young HCT Recipients Experience One or More Late Effects

In this multi-center study of patients under 3 years old who underwent myeloablative allogeneic hematopoietic cell transplantation (HCT), those who survived relapse-free for a year or more (n=717) experienced 10-year overall survival of 87% and transplant-related mortality of 5%. However, 30% developed one or more organ toxicity or other late-effect a year or more after transplant.

The 3 most frequent late effects were growth hormone deficiency/growth disturbance (10-year cumulative incidence 23%, 95% CI 19-28%), cataracts (18%, 95% CI 15-22%) and hypothyroidism (13%, 95% CI 10-16%). Subsequent malignancy was reported in 3.6% of patients.

This retrospective study analyzed transplant outcomes reported to CIBMTR® (Center for International Blood and Marrow Transplant Research®). Median follow-up was 8.3 years (range, 1.0-26.4); median age at follow-up was 9 years (range, 2-29).

In a multivariate analysis, total body irradiation was predictive of increased risk of cataracts (HR 17.2, 95% CI 7.4-39.8, p<0.001), growth deficiency (HR 3.5, 95% CI 2.2-5.5, p<0.001), and hypothyroidism (HR 5.3, 95% CI 3.0-9.4, p<0.001). Chronic graft-versus-host disease was also significantly associated with adverse outcomes.

The authors recommend long-term follow-up as “a critical element in the health care of survivors” to decrease the risks of late effects in this growing survivor population.

Vrooman LM, et al. Biol Blood Marrow Transplant