The aim of this study is to analyse cumulative exposure, pharmacokinetics and pharmacogenomics of daily intravenous busulfan in paediatric haematopoietic stem cell transplantation.

Who is it for?
You may be eligible to join this study if you are aged 18 years or below, with underlying disease with recognised HSCT indication and are receiving once daily IV busulfan as a component of HSCT conditioning regimen and have adequate central venous access for blood sampling.

Study details
This study will involve a prospective nonrandomised trial where patients who consent be asked to provide small volumes (7 ml per day, in total 28mL from 28 samples over four days) of blood which will be sent to a central laboratory for analysis. These sample results will be used for study purposes only and will be taken in conjunction with those taken for standard of care per the institutions current practice. Transplant outcome data will be attained at day +30, +100 and +365 post transplant. The blood sample results will not interfere and will not be available to guide clinical treatment of participating patients, however will improve dose optimisation of busulfan in future patients.

This study will help guide optimal dosing regimens and reduce toxicity for paediatric patients who require intravenous busulfan as part of a HSCT regime.