Abstract
Although most children with acute lymphoblastic leukemia (ALL) receive fractionated total body irradiation (FTBI) as myeloablative conditioning (MAC) for allogeneic hematopoietic stem cell transplantation (allo-HSCT), it is an important matter of debate if chemotherapy can effectively replace FTBI. To compare outcomes after FTBI versus chemotherapy-based conditioning (CC), we performed a retrospective EBMT registry study. Children aged 2–18 years after MAC for first allo-HSCT of bone marrow (BM) or peripheral blood stem cells (PBSC) from matched-related (MRD) or unrelated donors (UD) in first (CR1) or second remission (CR2) between 2000 and 2012 were included. Propensity score weighting was used to control pretreatment imbalances of the observed variables. 3.054 patients were analyzed. CR1 (1.498): median follow-up (FU) after FTBI (1.285) and CC (213) was 6.8 and 6.1 years. Survivals were not significantly different. CR2 (1.556): median FU after FTBI (1.345) and CC (211) was 6.2 years. Outcomes after FTBI were superior as compared with CC with regard to overall survival (OS), leukemia-free survival (LFS), relapse incidence (RI), and nonrelapse mortality (NRM). However, we must emphasize the preliminary character of the results of this retrospective “real-world-practice” study. These findings will be prospectively assessed in the ALL SCTped 2012 FORUM trial.
Highlights
In the CR1 cohort, 213 patients (14%) received chemotherapybased conditioning (CC). These regimens consisted of Busulfan/Cyclophosphamide (Bu/Cy, n = 68), Bu/Cy/Etoposide (Bu/Cy/Eto, n = 66), Bu/Cytarabine (AraC)/+/−Melphalan (Mel, n = 23), Bu/Cy/Mel (n = 20), Bu/Fludarabine (Flu, n = 20), Bu/Cy/Thiotepa (Thio, n = 14), and Bu/Flu/Thio (n = 2)
Five years overall survival (OS) was 68.8% after fractionated total body irradiation (FTBI) and 74.1% after CC (P = 0.25)
Five years leukemia-free survival (LFS) was 63.8% after FTBI and 61.4% after CC (P = 0.83)
Summary
Most children with acute lymphoblastic leukemia (ALL) above 2 years of age being candidate to be treated with allogeneic hematopoietic stem cell transplantation (alloHSCT) receive myeloablative conditioning (MAC) with a fractionated total body irradiation (FTBI)-containing regimen [1,2,3,4,5,6,7,8,9,10,11,12,13,14,15,16,17] It is an important matter of debate if chemotherapy can effectively replace FTBI. The study was initiated and conducted on behalf of the Paediatric Diseases Working Party of the European Society for Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?—A multicenter
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