Risk Factors for Subsequent Central Nervous System (CNS) Tumors in Pediatric Allogeneic Hematopoietic Cell Transplant (HCT): a Study From the Center for International Blood and Marrow Transplant Research (CIBMTR).

Publication Type:

Journal Article


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


Survivors of Hematopoietic cell transplantation (HCT) are at risk of subsequent solid tumors, including central nervous system (CNS) tumors. The risk of CNS tumors post HCT in pediatric HCT recipients is not known. We evaluated the incidence and risk factors for CNS tumors in pediatric recipients of allogeneic HCT reported to the Center for International Blood and Marrow Transplant Research (CIBMTR) between 1976 and 2008. A case control design was used. There were no CNS tumors in the non-malignant cohort (n=4543) or in those undergoing HCT for solid tumors (n=26). There were 59 CNS tumors in 8720 patients transplanted for hematological malignancies. In comparison to the general population, pediatric HCT recipients with hematological malignancies had a 33 times higher than expected rate of CNS tumors (95% confidence interval, 22.98-45.77, p<0.0001). The cumulative incidence of subsequent CNS tumors is 1.29 % (0.87-1.87) at 20 years post HCT. Significant risk factors in the entire cohort were having an unrelated donor (HR 3.35; p=0.0002), CNS disease prior to HCT for both ALL (HR 8.21; p=0.0003) and AML (HR 6.21; p=0.0174). Analysis of the matched cohort showed having an unrelated donor transplant (HR 4.79; p=0.0037), CNS disease prior to HCT (HR 7.67; p=0.0064) and radiotherapy exposure prior to conditioning (HR 3.7; p=0.0234) to be significant risk factors. Chronic Graft vs Host disease was associated with a lower risk (HR 0.29; p=0.0143). Survivors of HCT for non-malignant diseases did not show an increased incidence of CNS tumors, whereas survivors of hematologic malignancies have a markedly increased incidence of CNS tumors that warrants life-long surveillance.