Ruggeri, Annalisa and Galimard, Jacques-Emmanuel and Paina, Olesya and Fagioli, Franca and Tbakhi, Abdelghani and Yesilipek, Akif and Fernandez Navarro, Jose Maria and Faraci, Maura and Hamladji, Rose-Marie and Skorobogatova, Elena and Al-Seraihy, Amal and Sundin, Mikael and Herrera, Concepcion and Rifon, Jose and Dalissier, Arnaud and Locatelli, Franco and Rocha, Vanderson and Corbacioglu, Selim (2021) Outcomes of Unmanipulated Haploidentical Transplantation Using Post-Transplant Cyclophosphamide (PT-Cy) in Pediatric Patients With Acute Lymphoblastic Leukemia. TRANSPLANTATION AND CELLULAR THERAPY, 27 (5). ISSN 2666-6375, 2666-6367
Full text not available from this repository. (Request a copy)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. (C) 2021 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | STEM-CELL TRANSPLANTATION; BONE-MARROW-TRANSPLANTATION; HEMATOLOGIC MALIGNANCIES; CHILDREN; CRITERIA; RELAPSE; ALWP; Childhood; Acute lymphoblastic leukemia; Haploidentical transplantation; Post-transplantation cyclophosphamide |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 27 Sep 2022 13:10 |
| Last Modified: | 27 Sep 2022 13:10 |
| URI: | https://pred.uni-regensburg.de/id/eprint/48092 |
Actions (login required)
![]() |
View Item |

