title: Incidence and severity of crucial late effects after allogeneic HSCT for malignancy under the age of 3 years: TBI is what really matters.
abstract: 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.
authors.affiliation: Willem-Alexander Children's Hospital, LUMC, Leiden, The Netherlands.
authors.forename: D
authors.initials: D
authors.lastname: Bresters
authors.affiliation: SCT Unit, St Anna Kinderspital, Medical University, Vienna, Austria.
authors.forename: A
authors.initials: A
authors.lastname: Lawitschka
authors.affiliation: Pediatric Hematology-Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
authors.forename: C
authors.initials: C
authors.lastname: Cugno
authors.affiliation: SCT Unit, St Anna Kinderspital, Medical University, Vienna, Austria.
authors.forename: U
authors.initials: U
authors.lastname: Pötschger
authors.affiliation: EBMT Pediatric Diseases Working Party office, Hematology and Cell Therapy Department, Saint-Antoine Teaching Hospital, Paris, France.
authors.forename: A
authors.initials: A
authors.lastname: Dalissier
authors.affiliation: Department of Pediatric Hematology Oncology and Research Unit, Aix-Marseille University and Timone Hospital, Marseille, France.
authors.forename: G
authors.initials: G
authors.lastname: Michel
authors.affiliation: Hospital for Children & Adolescents, University of Helsinki, Helsinki, Finland.
authors.forename: K
authors.initials: K
authors.lastname: Vettenranta
authors.affiliation: Department of Hematology/Immunology/SCT, Astrid Lindgren Children's Hospital, Karolinska University Hospital and Karolinska Institutet, Stockholm, Sweden.
authors.forename: M
authors.initials: M
authors.lastname: Sundin
authors.affiliation: Department of Paediatric Haematology/Oncology, King Faisal Specialist Hospital & Research Centre, Riyadh, Saudi Arabia.
authors.forename: A
authors.initials: A
authors.lastname: Al-Seraihy
authors.affiliation: Pediatric Hematology and Oncology HSCT Unit, Instituto G Gaslini, Genova, Italy.
authors.forename: M
authors.initials: M
authors.lastname: Faraci
authors.affiliation: Department of Paediatric Haematology & Oncology, University Hospital Motol, Prague, Czech Republic.
authors.forename: P
authors.initials: P
authors.lastname: Sedlacek
authors.affiliation: Wilhelmina Children's Hospital, UMCU, Utrecht, The Netherlands.
authors.forename: A B
authors.initials: AB
authors.lastname: Versluys
authors.affiliation: Department of Pediatric Oncology/Haematology, Sheffield Children's Hospital NHS Foundation Trust, Sheffield, UK.
authors.forename: A
authors.initials: A
authors.lastname: Jenkins
authors.affiliation: Department of Pediatric Hematology Oncology, Nouvel Hopital Civil, Strasbourg, France.
authors.forename: P
authors.initials: P
authors.lastname: Lutz
authors.affiliation: Department of Haematology, Royal Hospital for Sick Children, Glasgow, UK.
authors.forename: B
authors.initials: B
authors.lastname: Gibson
authors.affiliation: Department of BMT, Great Ormond Street Hospital for Children Foundation Trust, London, UK.
authors.forename: A
authors.initials: A
authors.lastname: Leiper
authors.affiliation: Hematopoietic Stem Cell Transplant and Cell Therapy Unit, Children's Hospital Niño Jesus, Madrid, Spain.
authors.forename: M A
authors.initials: MA
authors.lastname: Diaz
authors.affiliation: The Children's Hospital at Westmead, Oncology Unit, Sydney, NSW, Australia.
authors.forename: P J
authors.initials: PJ
authors.lastname: Shaw
authors.affiliation: Children's BMT Unit, Great North Children's Hospital Royal, Victoria Infirmary, Newcastle upon Tyne, UK.
authors.forename: R
authors.initials: R
authors.lastname: Skinner
authors.affiliation: Sydney Children's Hospital, Centre for Children's Cancer, Sydney, NSW, Australia.
authors.forename: T A
authors.initials: TA
authors.lastname: O'Brien
authors.affiliation: Department of Haematology, Hammersmith Hospital, Imperial NHS Trust, London, UK.
authors.forename: N
authors.initials: N
authors.lastname: Salooja
authors.affiliation: Department of Cell Transplantation and Immunology, Hospital for Children and Adolescents, Frankfurt am Main, Germany.
authors.forename: P
authors.initials: P
authors.lastname: Bader
authors.affiliation: SCT Unit, St Anna Kinderspital, Medical University, Vienna, Austria.
authors.forename: C
authors.initials: C
authors.lastname: Peters
mesh_terms: D002675:Child, Preschool; D003430:Cross-Sectional Studies; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease; D019337:Hematologic Neoplasms / Q000150:complications / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects* / Q000379:methods; D006801:Humans; D015994:Incidence; D007223:Infant; D008297:Male; D012042:Registries; D012307:Risk Factors; D012737:Sex Factors; D013997:Time Factors; D014184:Transplantation, Homologous; D014916:Whole-Body Irradiation / Q000009:adverse effects*