Younger children are considered to be more vulnerable to late effects (LE), which prompted us to study LE in patients after haematopoietic stem cell transplantation (HSCT) for a haematological malignancy before the age of 3. In this multicentre EBMT study, cumulative incidence (CI) and severity of endocrine LE, central nervous system complications and secondary malignancies at 5, 10, 15 and 20 years of follow-up were assessed. Risk factors (RF) like gender, diagnosis, age at and year of HSCT, TBI- or chemo-conditioning and GVHD were analysed. CI of any LE was 0.30, 0.52, 0.66 and 0.72 at 5, 10, 15 and 20 years after HSCT, respectively. In 25% of the patients, LE were severe at a median follow-up of 10.4 years. In multivariate analysis, only TBI was a RF for having any LE and for thyroid dysfunction and growth disturbance. Female gender was a RF for delayed pubertal development. Some more insight could be gained by descriptive analysis regarding the role of TBI and GVHD on the severity of LE. Although only five selected LE have been studied and median follow-up is relatively short, the incidence and severity of these LE are considerable but not different from what has been found in older children and TBI is the main RF.

Children's BMT Unit Great North Children's Hospital Royal Victoria Infirmary Newcastle upon Tyne UK

Department of BMT Great Ormond Street Hospital for Children Foundation Trust London UK

Department of Cell Transplantation and Immunology Hospital for Children and Adolescents Frankfurt am Main Germany

Department of Haematology Hammersmith Hospital Imperial NHS Trust London UK

Department of Haematology Royal Hospital for Sick Children Glasgow UK

Department of Hematology Immunology SCT Astrid Lindgren Children's Hospital Karolinska University Hospital and Karolinska Institutet Stockholm Sweden

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

Department of Paediatric Haematology Oncology King Faisal Specialist Hospital and Research Centre Riyadh Saudi Arabia

Department of Pediatric Hematology Oncology and Research Unit Aix Marseille University and Timone Hospital Marseille France

Department of Pediatric Hematology Oncology Nouvel Hopital Civil Strasbourg France

Department of Pediatric Oncology Haematology Sheffield Children's Hospital NHS Foundation Trust Sheffield UK

EBMT Pediatric Diseases Working Party office Hematology and Cell Therapy Department Saint Antoine Teaching Hospital Paris France

Hematopoietic Stem Cell Transplant and Cell Therapy Unit Children's Hospital Niño Jesus Madrid Spain

Hospital for Children and Adolescents University of Helsinki Helsinki Finland

Pediatric Hematology and Oncology HSCT Unit Instituto G Gaslini Genova Italy

Pediatric Hematology Oncology Fondazione IRCCS Policlinico San Matteo Pavia Italy

SCT Unit St Anna Kinderspital Medical University Vienna Austria

Sydney Children's Hospital Centre for Children's Cancer Sydney NSW Australia

The Children's Hospital at Westmead Oncology Unit Sydney NSW Australia

Wilhelmina Children's Hospital UMCU Utrecht The Netherlands

Willem Alexander Children's Hospital LUMC Leiden The Netherlands

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