RT Journal Article
T1 Outcomes of Unmanipulated Haploidentical Transplantation Using Post-Transplant Cyclophosphamide (PT-Cy) in Pediatric Patients With Acute Lymphoblastic Leukemia.
A1 Ruggeri, Annalisa
A1 Galimard, Jacques-Emmanuel
A1 Paina, Olesya
A1 Fagioli, Franca
A1 Tbakhi, Abdelghani
A1 Yesilipek, Akif
A1 Navarro, Jose Maria Fernandez
A1 Faraci, Maura
A1 Hamladji, Rose-Marie
A1 Skorobogatova, Elena
A1 Al-Seraihy, Amal
A1 Sundin, Mikael
A1 Herrera, Concepcion
A1 Rifon, Jose
A1 Dalissier, Arnaud
A1 Locatelli, Franco
A1 Rocha, Vanderson
A1 Corbacioglu, Selim
K1 Acute lymphoblastic leukemia
K1 Childhood
K1 Haploidentical transplantation
K1 Post-transplantation cyclophosphamide
AB HLA-haploidentical transplantation (haplo-HCT) using post-transplantation-cyclophosphamide (PT-Cy) is a feasible procedure in children with malignancies. However, large studies on Haplo-HCT with PT-Cy for childhood acute lymphoblastic leukemia (ALL) are lacking. We analyzed haplo-HCT outcomes in 180 children with ALL. Median age was 9 years, and median follow-up was 2.7 years. Disease status was CR1 for 24%, CR2 for 45%, CR+3 for 12%, and active disease for 19%. All patients received PT-Cy day +3 and +4. Bone marrow (BM) was the stem cell source in 115 patients (64%). Cumulative incidence of 42-day engraftment was 88.9%. Cumulative incidence of day-100 acute graft-versus-host disease (GVHD) grade II-IV was 28%, and 2-year chronic GVHD was 21.9%. At 2 years, cumulative incidence of nonrelapse mortality (NRM) was 19.6%. Cumulative incidence was 41.9% for relapse and 25% for patients in CR1. Estimated 2-year leukemia free survival was 65%, 44%, and 18.8% for patients transplanted in CR1, CR2, CR3+ and 3% at 1 year for active disease. In multivariable analysis for patients in CR1 and CR2, disease status (CR2 [hazard ratio {HR} = 2.19; P = .04]), age at HCT older than 13 (HR = 2.07; P = .03) and use of peripheral blood stem cell (PBSC) (HR = 1.98; P = .04) were independent factors associated with decreased overall survival. Use of PBSC was also associated with higher NRM (HR = 3.13; P = .04). Haplo-HCT with PT-Cy is an option for children with ALL, namely those transplanted in CR1 and CR2. Age and disease status remain the most important factors for outcomes. BM cells as a graft source is associated with improved survival.
PB Elsevier
YR 2021
FD 2021-01-18
LK http://hdl.handle.net/10668/17765
UL http://hdl.handle.net/10668/17765
LA en
NO Ruggeri A, Galimard JE, Paina O, Fagioli F, Tbakhi A, Yesilipek A, et al. Outcomes of Unmanipulated Haploidentical Transplantation Using Post-Transplant Cyclophosphamide (PT-Cy) in Pediatric Patients With Acute Lymphoblastic Leukemia. Transplant Cell Ther. 2021 May;27(5):424.e1-424.e9
DS RISalud
RD Apr 11, 2025