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 and 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.

Belorussian Centre for Paediatric Oncology and Hematology Minsk

BMT Unit Sao Paolo University Sao Paolo Brasil

Bristol Royal Hospital for Children Dept of Paediatric Oncology BMT Bristol

Clinica di Oncoematologia Pediatrica Dipartimento di Pediatria Padova

Copenhagen University Hospital Rigshospitalet Dept of Children and Adolescents Medicine Copenhagen Denmark

Department of Clinical Immunology and Transplantation Jagiellonian University Medical College Children's Hospital in Krakow

Department of Pediatric Hematology and Oncology Collegium Medicum Nicolaus Copernicus University Torun Bydgoszcz

Department of Pediatric Hematology Oncology and Bone Marrow Transplantation Wroclaw Medical University Wroclaw

Department of Pediatric Hematology Oncology IRCCS Bambino Gesu Children' s Hospital Rome Italy; Department of Health Sciences Magna Graecia University Catanzaro

EBMT Paris Study Unit Paris

EBMT Statistical Unit Paris

Hematology Unit Department of Oncology and Hematology Santo Spirito Hospital 65124 Pescara

Hematopoietic Stem Cell Transplantation Unit Fondazione IRCCS San Gerardo dei Tintori Monza Italy; Department of Medicine and Surgery Milano Bicocca University Monza

HSCT Unit Department Hemato Oncology IRCSS Istituto G Gaslini; Genova

Institut d'Hematologie et d'Oncologie Pediatrique Lyon

IRCCS Azienda Ospedaliero Universitaria Bologna

IRCCS San Raffaele Scientific Institute Milano

Onco Ematologia Pediatrica Centro Trapianti Cellule Staminali Torino

Pediatric Hematology Oncology King Faisal Specialist Hospital and Research Centre Riyadh Saudi Arabia

Pediatric University Teaching Hospital BMT Unit 2 Children's Clinic Bratislava Slovakia

Prinses Maxima Centrum Utrecht

RM Gorbacheva Research Institute Pavlov University St Petersburg

St Anna Children's Hospital Department of Pediatrics Medical University of Vienna Vienna

St Sophia Children's hospital Oncology center Athens

The Russian Children' s Research Hospital Department of Bone Marrow Transplantation Moscow

University Hospital Motol Department of Paediatric Haematology and Oncology Prague Czech Republic

University of Regensburg Regensburg

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