A multi-centre study investigating the pharmacokinetics of treosulfan in children undergoing stem cell transplantation.

This study involves measuring the blood levels of the medication, treosulfan, to improve the way we adjust the dose in children. Who is it for? Children and adolescents with either malignant or non malignant (genetic) diseases can join this study if they are receiving the medication, treosulfan, prior to undergoing blood or marrow transplantation. Trial details A series of blood levels will be collected after the treosulfan dose to see how well their body breaks down and gets rid of the medication. This study does not replace other studies, but simply investigates key medication widely used in the treatment of children's cancer. Participants will be studied for several years after the study to monitor their health. This study will define the best way to adjust the dose of the drug, treosulfan, a key medication in blood or marrow transplantation conditioning regimens. Too much causes toxicity and too little risks the disease being more likely to return.