Better leukemia-free and overall survival in AML in first remission following cyclophosphamide in combination with busulfan compared with TBI.

Publication Type:

Journal Article


Blood, Volume 122, Issue 24, p.3863-70 (2013)


2013, Acute Disease, Adolescent, Adult, Antineoplastic Combined Chemotherapy Protocols, Busulfan, Child, Child, Preschool, Clinical Research Division, Combined Modality Therapy, Cyclophosphamide, Disease-Free Survival, Female, hematopoietic stem cell transplantation, Humans, Leukemia, Myeloid, Male, March 2014, Middle Aged, Multivariate Analysis, Remission Induction, Siblings, Time Factors, Transplantation, Homologous, Treatment Outcome, Unrelated Donors, Whole-Body Irradiation, Young Adult


Cyclophosphamide combined with total body irradiation (Cy/TBI) or busulfan (BuCy) are the most widely used myeloablative conditioning regimens for allotransplants. Recent data regarding their comparative effectiveness are lacking. We analyzed data from the Center for International Blood and Marrow Transplant Research for 1230 subjects receiving a first hematopoietic cell transplant from a human leukocyte antigen-matched sibling or from an unrelated donor during the years 2000 to 2006 for acute myeloid leukemia (AML) in first complete remission (CR) after conditioning with Cy/TBI or oral or intravenous (IV) BuCy. Multivariate analysis showed significantly less nonrelapse mortality (relative risk [RR] = 0.58; 95% confidence interval [CI]: 0.39-0.86; P = .007), and relapse after, but not before, 1 year posttransplant (RR = 0.23; 95% CI: 0.08-0.65; P = .006), and better leukemia-free survival (RR = 0.70; 95% CI: 0.55-0.88; P = .003) and survival (RR = 0.68; 95% CI: 0.52-0.88; P = .003) in persons receiving IV, but not oral, Bu compared with TBI. In combination with Cy, IV Bu is associated with superior outcomes compared with TBI in patients with AML in first CR.