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Treosulfan, fludarabine, and low-dose total body irradiation for children and young adults with acute myeloid leukemia or myelodysplastic syndrome undergoing allogeneic hematopoietic cell transplantation: Prospective phase II trial of the pediatric blood and marrow transplant consortium

Biology of Blood and Marrow Transplantation May 27, 2018

Nemecek ER, et al. - In this multicenter study, the researchers sought to assess a treosulfan-based regimen in children and young adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplant (HCT). This study was conducted on forty patients with median age 11 years. Results revealed that no serious organ toxicities were noted. In children and young adults with AML and MDS, this body surface area (BSA)-based treosulfan regimen resulted in excellent engraftment and disease-free survival and minimal toxicity and transplant-related mortality (3%).
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