Survival and Late Effects after Allogeneic Hematopoietic Cell Transplantation for Hematologic Malignancy at Less Than Three Years of Age.

Publication Type:

Journal Article


Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation (2017)


Very young children undergoing hematopoietic cell transplantation (HCT) are a unique and vulnerable population. We analyzed outcomes of 717 patients from 117 centers who survived relapse-free for ≥1 year following allogeneic myeloablative HCT for hematologic malignancy at <3 years-of-age, between 1987-2012. Median follow-up was 8.3 years (range 1.0-26.4 years); median age at follow-up was 9 years (range 2-29 years). Ten-year overall and relapse-free survival were 87% (95% CI 85-90%) and 84% (95% CI 81-87%). Ten-year cumulative incidence of relapse was 11% (95% CI 9-13%). Of 84 deaths, relapse was the leading cause (43%). Chronic graft-versus-host-disease 1 year after HCT was associated with increased risk of mortality (HR 2.1, 95% CI 1.3-3.3, p=0.0018). Thirty percent of patients experienced ≥1 organ toxicity/late-effect >1 year after HCT. The most frequent late-effects included growth hormone deficiency/growth disturbance (10-year cumulative incidence 23%, 95% CI 19-28%), cataracts (18%, 95% CI 15-22%), hypothyroidism (13%, 95% CI 10-16%), gonadal dysfunction/infertility requiring hormone replacement (3%, 95% CI 2-5%), and stroke/seizure (3%, 95% CI 2-5%). Subsequent malignancy was reported in 3.6%. In multivariable analysis, TBI 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). In summary, those who survived relapse-free ≥1 year after HCT for hematologic malignancy at <3 years-of-age had favorable overall survival. Chronic graft-versus-host-disease and TBI were associated with adverse outcomes. Future efforts should focus on reducing the risk of relapse and late-effects after HCT at early age.