Busulfan- vs. treosulfan-based conditioning for allo-HSCT for pediatric ALL
Kalwak K, Moser LM, Pötschger U, et al. Comparable Outcomes After Busulfan- or Treosulfan-Based Conditioning for Allo-HSCT in Children With ALL. Blood Advances. 2024; (doi: 10.1182/bloodadvances.2024014548).
Research shows that as pediatric patients >4 years of age with acute lymphoblastic leukemia (ALL) prepare for allogeneic hematopoietic stem cell transplantation, busulfan-based and treosulfan-based conditioning are both safe and effective substitutes for total body irrigation (TBI)-based regimens. TBI is recognized as superior to chemotherapy in this setting but may be unavailable in certain geographic areas or contraindicated for some patients. However, evidence points to comparable outcomes when pairing thiotepa-fludarabine with bulsulfan (BU/THIO/FLU) or treosulfan (TREO/THIO/FLU), with no accompanying increase in risk. The data come from the FORUM clinical trial that confirmed TBI's superiority. For the study, scientists studied 180 patients ≤18 years at diagnosis who received BU/THIO/FLU and 128 others who received TREO/THIO/FLU. At a median follow-up of 4.2 years, overall survival between the groups was similar at 0.71 and 0.72, respectively, with event-free survival measured at 0.60 vs. 0.55, respectively. Similarly, the 3-year cumulative rate of relapse was 0.31 vs. 0.36 and non-relapse mortality was 0.08 vs. 0.09.