Matched unrelated donor transplantation versus haploidentical transplantation with post-transplant cyclophosphamide in children with acute myeloid leukemia: a PDWP-EBMT study

Ruggeri, Annalisa and Santoro, Nicole and Galimard, Jacques-Emmanuel and Kalwak, Krzysztof and Algeri, Mattia and Zubarovskaya, Ludmila and Czyzewski, Krzysztof and Skorobogatova, Elena and Sedlacek, Petr and Besley, Caroline and Balduzzi, Adriana and Bertrand, Yves and Peristeri, Julia and Fagioli, Franca and Ifversen, Mariane and Gozdzik, Jolanta and Peters, Christina and Versluijs, Birgitta and Biffi, Alessandra and Prete, Arcangelo and Faraci, Maura and Ghemlas, Ibrahim and Bodova, Ivana and Aleinikova, Olga and Dalissier, Arnaud and Rocha, Vanderson and Corbacioglu, Selim (2024) Matched unrelated donor transplantation versus haploidentical transplantation with post-transplant cyclophosphamide in children with acute myeloid leukemia: a PDWP-EBMT study. HAEMATOLOGICA, 109 (7). pp. 2122-2130. ISSN 0390-6078, 1592-8721

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Abstract

In children with acute myeloid leukemia (AML) who lack a human leukocyte antigen (HLA) identical sibling, the donor can be replaced with an HLA-matched unrelated donor (MUD) or a haploidentical donor (haplo). We compared outcomes of patients <18 years with AML in first and second complete remission (CR1 & CR2) undergoing a hematopoietic stem cell transplantation (HCT) either with a MUD with anti-thymocyte globulin (ATG) (N=420) or a haplo HCT with post -transplant cyclophosphamide (PT-CY) (N=96) after a myeloablative conditioning regimen (MAC) between 2011 and 2021, reported to the European Society for Blood and Marrow Transplantation. A matched pair analysis was performed to adjust for differences among groups. The final analysis was performed on 253 MUD and 95 haplo-HCT. In the matched cohort, median age at HCT was 11.2 and 10 years and median year of HCT was 2017 and 2018, in MUD and haplo-HCT recipients, respectively. The risk of grade III -IV acute graft - versus -host disease (aGVHD) was significantly higher in the haplo group (hazard ratio [HR]=2.33, 95% confidence interval [CI]: 1.18-4.58; P =0.01). No significant differences were found in 2 years overall survival (OS; 78.4% vs . 71.5%; HR=1.39, 95% CI: 0.84-2.31; P =0.19), leukemia -free survival (LFS; 72.7% vs . 69.5%; HR=1.22, 95% CI: 0.76-1.95; P =0.41), CI of relapse (RI; 19.3% vs . 19.5%; HR=1.14, 95% CI: 0.62-2.08; P =0.68) non -relapse -mortality (NRM; 8% vs. 11%; HR=1.39, 95% CI: 0.66-2.93; P =0.39) and graft - versus -host free relapse -free survival (GRFS; 60.7% vs . 54.5%, HR=1.38, 95% CI: 0.95-2.02; P =0.09) after MUD and haplo-HCT respectively. Our study suggests that haplo-HCT with PT-CY is a suitable option to transplant children with AML lacking a matched related donor.

Item Type: Article
Uncontrolled Keywords: STEM-CELL TRANSPLANTATION; BONE-MARROW-TRANSPLANTATION; ACUTE LYMPHOBLASTIC-LEUKEMIA; HEMATOLOGIC MALIGNANCIES; T-CELL; SIBLING DONORS; RISK; DIAGNOSIS; CRITERIA; RELAPSE;
Subjects: 600 Technology > 610 Medical sciences Medicine
Divisions: Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation
Depositing User: Dr. Gernot Deinzer
Date Deposited: 14 Aug 2025 06:40
Last Modified: 14 Aug 2025 06:40
URI: https://pred.uni-regensburg.de/id/eprint/65644

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