Poor-risk (PR) cytogenetic/molecular abnormalities generally direct pediatric patients with acute myeloid leukemia (AML) to allogeneic hematopoietic stem cell transplant (HSCT). We assessed the predictive value of cytogenetic risk classification at diagnosis with respect to post-HSCT outcomes in pediatric patients. Patients younger than 18 years at the time of their first allogeneic HSCT for AML in CR1 between 2005 and 2022 who were reported to the European Society for Blood and Marrow Transplantation registry were subgrouped into four categories. Of the 845 pediatric patients included in this study, 36% had an 11q23 abnormality, 24% had monosomy 7/del7q or monosomy 5/del5q, 24% had a complex or monosomal karyotype, and 16% had other PR cytogenetic abnormalities. In a multivariable model, 11q23 (hazard ratio [HR] = 0.66, P = 0.03) and other PR cytogenetic abnormalities (HR = 0.55, P = 0.02) were associated with significantly better overall survival when compared with monosomy 7/del7q or monosomy 5/del5q. Patients with other PR cytogenetic abnormalities had a lower risk of disease relapse after HSCT (HR = 0.49, P = 0.01) and, hence, better leukemia-free survival (HR = 0.55, P = 0.01). Therefore, we conclude that PR cytogenetic abnormalities at diagnosis predict overall survival after HSCT for AML in pediatric patients.

Anthony Nolan Research Institute Imperial College Healthcare NHS Trust London UK

Blood and Marrow Transplant Unit Department of Paediatric Haematology Royal Manchester Children's Hospital Manchester UK

Bone Marrow Transplant Unit 2 Children's Clinic University Children's Hospital Bratislava Slovakia

Bone Marrow Transplant Unit St Sophia Children's Hospital Oncology Centre Athens Greece

Centro Trapianti Cellule Staminali Onco Ematologia Pediatrica Ospedale Infantile Regina Margherita Turin Italy

CHU Bordeaux Groupe Hospitalier Pellegrin Enfants Bordeaux France

Clinical Department of Paediatric Bone Marrow Transplantation Oncology and Haematology Wrocław Medical University Wrocław Poland

Clinical Research Division Fred Hutchinson Cancer Center Seattle WA USA

Département Hématologie Oncologie Pédiatrique Hôpital de la Timone Marseille France

Department of Bone Marrow Transplantation and Cellular Therapy St Jude Children's Research Hospital Memphis TN USA

Department of Bone Marrow Transplantation Great Ormond Street Hospital NHS Foundation Trust London UK

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

Department of Paediatric Oncology Royal Marsden Hospital London UK

Department of Paediatrics King Faisal Specialist Hospital and Research Centre Riyadh Saudi Arabia

Department of Pediatric Hematology and Oncology IRCCS Ospedale Pediatrico Bambino Gesù Catholic University of the Sacred Heart Rome Italy

Department of Pediatric Hematology Oncology and Stem Cell Transplantation University of Regensburg Regensburg Germany

Department of Pediatrics Niño Jesus Children's Hospital Madrid Spain

Dipartimento di Ematologia Pediatrica Azienda Ospedaliera di Rilievo Nazionale Naples Italy

EBMT Paris Study Unit Hôpital Saint Antoine Paris France

EBMT Statistical Unit Hôpital Saint Antoine Paris France

Hôpital Armand Trousseau APHP Sorbonne Université Paris France

HSCT Unit Department of Hematology and Oncology IRCCS Institute G Gaslini Genoa Italy

King Hussein Cancer Center Amman Jordan

Oncopediatrics department Nantes University Hospital Nantes France

Paediatric Bone Marrow Transplant Service Bristol Royal Hospital for Children Bristol UK

Pediatric Hematology and Immunology Department Hôpital Robert Debré GHU APHP Nord Université Paris Cité Paris France

Pediatric Hematology Oncology and Stem Cell Transplant Division Padova University and Hospital Padua Italy

The Royal Hospital for Children Glasgow UK

Unité de coordination interne et externe Institut d'Hématologie et d'Oncologie Pédiatrique Lyon France

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