1.
Myeloablative conditioning for allo-HSCT in pediatric ALL: FTBI or chemotherapy?-A multicenter EBMT-PDWP study
Willasch, A. M., Peters, C., Sedlacek, P., Dalle, J. H., Kitra-Roussou, V., Yesilipek, A., Wachowiak, J., Lankester, A., Prete, A., Hamidieh, A. A., et al
Bone marrow transplantation. 2020
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.
2.
Outcomes of advanced Hodgkin lymphoma after umbilical cord blood transplantation: a Eurocord and EBMT Lymphoma and Cellular Therapy & Immunobiology Working Party study
Paviglianiti, A., Maio, K. T., Rocha, V., Gehlkopf, E., Milpied, N., Esquirol, A., Chevallier, P., Blaise, D., Gac, A. C., Leblond, V., et al
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation. 2018
Abstract
Allogeneic stem cell transplantation is an alternative for patients with relapsed or refractory Hodgkin lymphoma (HL) but only limited data on unrelated umbilical cord blood transplantation (UCBT) are available. We analyzed 131 adults with HL who underwent UCBT in EBMT centers from 2003 to 2015. Disease status at UCBT was complete remission (CR) in 59 (47%) and almost all patients had received a previous autologous stem cell transplantation. The 4-year PFS and OS were 26% (95% CI 19-34%) and 46% (95% CI 37-55%), respectively. Relapse incidence was 44% (95% CI 36-54%) and non-relapse mortality (NRM) was 31% (95% CI 23-40%) at 4 years. In multivariate analysis, refractory/relapsed disease status at UCBT was associated with increased relapse incidence (HR=3.14 [95% CI 1.41-7.00], p=0.005) and NRM (HR=3.61 [95% CI 1.58-8.27], p=0.002), lower PFS (HR=3.45 [95% CI 1.95-6.10], p<0.001) and OS (HR=3.10 [95% CI 1.60-5.99], p=0.001). Conditioning regimen with cyclophospamide+fludarabine+2Gy total body irradiation (Cy+Flu+2 GyTBI) was associated with decreased risk of NRM (HR=0.26 [95% CI 0.10-0.64], p=0.004). Moreover, Cy+Flu+2 GyTBI conditioning regimen was associated with a better OS (HR=0.25 [95% CI 0.12-0.50], p<0.001) and PFS (HR=0.51 [95% CI 0.27-0.96], p=0.04). UCBT is feasible in heavily pretreated patients with HL. The reduced intensity conditioning regimen with Cy+flu+2 GyTBI is associated with a better OS and NRM. However, outcomes are poor in patients not in CR at UCBT.
3.
Unrelated Cord Blood Transplantation for Acute Leukemia Diagnosed in the First Year of Life: Outcomes and Risk Factor Analysis
Ruggeri, A., Volt, F., Locatelli, F., Michel, G., Diaz de Heredia, C., Abecasis, M., Zecca, M., Vora, A., Yakouben, K., O'Brien, T. A., et al
Biology of Blood & Marrow Transplantation. 2017;23(1):96-102
Abstract
Infant acute leukemia still has a poor prognosis, and allogeneic hematopoietic stem cell transplantation is indicated in selected patients. Umbilical cord blood (UCB) is an attractive cell source for this population because of the low risk of chronic graft-versus-host disease (GVHD), the strong graft-versus-leukemia effect, and prompt donor availability. This retrospective, registry-based study reported UCB transplantation (UCBT) outcomes in 252 children with acute lymphoblastic leukemia (ALL; n=157) or acute myelogenous leukemia (AML; n=95) diagnosed before 1 year of age who received a single-unit UCBT after myeloablative conditioning between 1996 and 2012 in European Society for Blood and Marrow Transplantation centers. Median age at UCBT was 1.1 years, and median follow-up was 42 months. Most patients (57%) received a graft with 1 HLA disparity and were transplanted in first complete remission (CR; 55%). Cumulative incidence function (CIF) of day 100 acute GVHD (grades II to IV) was 40%+/-3% and of 4-year chronic GVHD was 13%+/-2%. CIF of 1-year transplant-related mortality was 23%+/-3% and of 4-year relapse was 27%+/-3%. Leukemia-free-survival (LFS) at 4 years was 50%+/-3%; it was 40% and 66% for those transplanted for ALL and AML, respectively (P=.001). LFS was better for patients transplanted in first CR, regardless of diagnosis. In multivariate model, diagnosis of ALL (P=.001), advanced disease status at UCBT (<.001), age at diagnosis younger than 3 months (P=.012), and date of transplant before 2004 were independently associated with worse LFS. UCBT is a suitable option for patients diagnosed with infant acute leukemia who achieve CR. In this cohort, patients with AML had better survival than those with ALL. Copyright © 2017 The American Society for Blood and Marrow Transplantation. Published by Elsevier Inc. All rights reserved.