An increasing number of children with non-malignant diseases can be cured by allogeneic haematopoietic stem cell transplantation (HSCT). Treosulfan (L-treitol-1,4-bis-methanesulfonate) is being used more frequently for conditioning, owing to its' lower toxicity profile compared with conventional myeloablative regimens. A retrospective analysis was performed of children registered in the EBMT database, who received treosulfan before HSCT between January 2005 and 2010, to identify possible dose-related toxicity and determine the incidence of engraftment, treatment-related mortality and overall survival (OS). Results from 316 transplants from 11 different countries are presented. Ninety-five (30%) were under 1 year of age at the time of transplant. OS was 83% and event-free survival was 76%; 3-year OS and event-free survival of infants below 1 year were 79% and 73%, respectively. No association was found with age at transplant, dose of treosulfan given, other agents used in combination with treosulfan, donor type, stem cell source, or second or subsequent transplant. In this report of the largest number of children to date receiving treosulfan for non-malignant diseases, treosulfan is shown to be a safe and effective agent even for those under 1 year of age at the time of transplant. Further prospective studies are needed using precisely defined protocols with pharmacokinetic monitoring and detailed chimerism analysis. In addition, long-term studies will be vital to determine long-term effects, for example, on fertility in comparison with other regimens.

Department of Paediatric Hematology and Oncology Teaching Hospital Motol 2nd Medical School Charles University Motol Prague Motol Prague Czech Republic

Department of Paediatric Hematology Oncology Hannover Medical School Hannover Germany

Department of Paediatrics Leiden University Medical Center RC Leiden The Netherlands

Department of Paediatrics St Anna Kinderspital and Children's Cancer Research Institute Medical University of Vienna Vienna Austria

Paediatric HSCT Great Ormond Street Hospital for Children National Health Service Trust London UK

Paediatric Immunology Department Paediatric HSCT Great North Children's Hospital Newcastle upon Tyne UK

Sackler Faculty of Medicine Paediatric Hematology Oncology Schneider Children's Medical Center of Israel Tel Aviv University Tel Aviv Israel

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