Introduction
bone marrow failure, toxicity, non-malignant disease, PID, haemoglobinopathies
PID, BONE MARROW FAILURE, HBP, SYNDROMES, HAEMOGLOBINOPATHIES, INBORN ERRORS OF METABOLISM
Allogeneic hematopoietic stem cell transplantation (HSCT) provides a curative treatment for pediatric patients affected with non-malignant diseases like primary immunodeficiencies (PID), haemoglobinopathies (HBP), bone marrow failure (BMF) syndromes, or inborn errors of metabolism (IEM) [Treosulfan’s (L-threitol-1,4-bis-methanesulfonate) potential for myeloablative conditioning with low toxicity was first demonstrated in adults [We prospectively compared safety and efficacy of treosulfan/fludarabine with busulfan/fludarabine myeloablative conditioning in children with non-malignant disease. The trial was conducted in accordance with the approved European pediatric investigational plan for treosulfan (PIP; EMEA-000883-PIP01-10) including a pharmacokinetic (PK) sub-study.