Publicaciones científicas
Outcomes of Unmanipulated Haploidentical Transplantation Using Post-Transplant Cyclophosphamide (PT-Cy) in Pediatric Patients With Acute Lymphoblastic Leukemia
Annalisa Ruggeri 1 , Jacques-Emmanuel Galimard 2 , Olesya Paina 3 , Franca Fagioli 4 , Abdelghani Tbakhi 5 , Akif Yesilipek 6 , José Maria Fernandez Navarro 7 , Maura Faraci 8 , Rose-Marie Hamladji 9 , Elena Skorobogatova 10 , Amal Al-Seraihy 11 , Mikael Sundin 12 , Concepcion Herrera 13 , Jose Rifón 14 , Arnaud Dalissier 2 , Franco Locatelli 15 , Vanderson Rocha 16 , Selim Corbacioglu 17
Abstract
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.
CITA DEL ARTÍCULO Transplant Cell Ther. 2021 May;27(5):424.e1-424.e9. doi: 10.1016/j.jtct.2021.01.016. Epub 2021 Jan 21.