Matched unrelated donor transplantation versus haploidentical transplantation with post-transplant cyclophosphamide in children with acute myeloid leukemia: a PDWP-EBMT study
Annalisa Ruggeri,
Nicole Santoro,
Jacques-Emmanuel Galimard,
Krzysztof Kalwak,
Mattia Algeri,
Ludmila Zubarovskaya,
Krzysztof Czyzewski,
Elena Skorobogatova,
Petr Sedlacek,
Caroline Besley,
Adriana Balduzzi,
Yves Bertrand,
Julia Peristeri,
Franca Fagioli,
Mariane Ifversen,
Jolanta Gozdzik,
Christina Peters,
Birgitta Versluijs,
Alessandra Biffi,
Arcangelo Prete,
Maura Faraci,
Ibrahim Ghemlas,
Ivana Bodova,
Olga Aleinikova,
Arnaud Dalissier,
Vanderson Rocha,
Selim Corbacioglu
Affiliations
Annalisa Ruggeri
IRCCS San Raffaele Scientific Institute, Milano
Nicole Santoro
Hematology Unit, Department of Oncology and Hematology, Santo Spirito Hospital, 65124 Pescara
Jacques-Emmanuel Galimard
EBMT Statistical Unit, Paris
Krzysztof Kalwak
Department of Pediatric Hematology, Oncology and Bone Marrow Transplantation, Wroclaw Medical University, Wroclaw
Mattia Algeri
Department of Pediatric Hematology Oncology, IRCCS Bambino Gesu Children' s Hospital, Rome, Italy; Department of Health Sciences, Magna Graecia University, Catanzaro
Ludmila Zubarovskaya
RM Gorbacheva Research Institute, Pavlov University, St. Petersburg
Krzysztof Czyzewski
Department of Pediatric Hematology and Oncology, Collegium Medicum, Nicolaus Copernicus University Torun, Bydgoszcz
Elena Skorobogatova
The Russian Children' s Research Hospital, Department of Bone Marrow Transplantation, Moscow
Petr Sedlacek
University Hospital Motol Department of Paediatric Haematology and Oncology, Prague, Czech Republic
Caroline Besley
Bristol Royal Hospital for Children Dept. of Paediatric Oncology/BMT, Bristol
Adriana Balduzzi
Hematopoietic Stem Cell Transplantation Unit, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy; Department of Medicine and Surgery, Milano-Bicocca University, Monza
Yves Bertrand
Institut d’Hematologie et d’Oncologie Pediatrique, Lyon
Julia Peristeri
St Sophia Children’s hospital Oncology center Athens
Franca Fagioli
Onco-Ematologia Pediatrica Centro Trapianti Cellule Staminali, Torino
Mariane Ifversen
Copenhagen University Hospital, Rigshospitalet, Dept of Children and Adolescents Medicine Copenhagen, Denmark
Jolanta Gozdzik
Department of Clinical Immunology and Transplantation Jagiellonian University Medical College, Children's Hospital in Krakow
Christina Peters
St. Anna Children's Hospital, Department of Pediatrics, Medical University of Vienna, Vienna
Birgitta Versluijs
Prinses Maxima Centrum, Utrecht
Alessandra Biffi
Clinica di Oncoematologia Pediatrica, Dipartimento di Pediatria, Padova
Arcangelo Prete
IRCCS-Azienda Ospedaliero Universitaria, Bologna
Maura Faraci
HSCT Unit, Department Hemato-Oncology, IRCSS Istituto G. Gaslini; Genova
Ibrahim Ghemlas
Pediatric Hematology/Oncology, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
Ivana Bodova
Pediatric University Teaching Hospital BMT Unit, II Children’s Clinic Bratislava, Slovakia
Olga Aleinikova
Belorussian Centre for Paediatric Oncology and Hematology Minsk
In children with acute myeloid leukemia (AML) who lack an 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 globuline (ATG) (n=420) or a haplo HCT with PT-CY (n=96) after a myeloablative conditioning regimen (MAC) between 2011 and 2021, reported to EBMT. A matched pair analysis was performed to adjust for differences among groups. The final analysis was performed on 253 MUD and 95 haplo-HCTs. 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 aGvHD was significantly higher in the haplo group (HR=2.33, 95%CI1.18-4.58, p=0.03). No significant differences were found in 2 years overall survival (OS; 78.4%vs71.5%; HR 1.39, 0.84-2.31, p=0.19), leukemia-free-survival (LFS; 72.7%vs69.5%; HR1.22, 0.76-1.95, p=0.41), CI of relapse (RI; 19.3%vs19.5%; HR=1.14, 0.62-2.08, p=0.68) non-relapse-mortality (NRM; 8%vs11%; HR=1.39, 0.66-2.93, p=0.39) and graft versus host free-relapse free survival (GRFS; 60.7%vs54.5%, HR=1.38, 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.
