A New Harmonized Approach to Estimate Busulfan Exposure PredictsSurvival and Toxicity after Hematopoietic Cell Transplantation in Children andYoung Adults: a Multicenter Retrospective Cohort Analysis

摘要

BackgroundIntravenous-busulfan (IV-busulfan) combined with therapeutic drug monitoring to guide dosing improves outcomes after allogeneic hematopoietic cell transplantation (allo-HCT). The best method to estimate busulfan exposure and the optimal exposure in children/young adults remains unclear. We therefore evaluated three approaches to estimate IV-Bu exposure (expressed as cumulative-area-under-the-curve; AUC) and associated busulfan-AUC with clinical outcomes in children/young adults undergoing allo-HCT.