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Target Busulfan Exposure in Children With HSCT in China

The First Affiliated Hospital of Soochow University · 2020
ClinicalTrials.gov · Datasets · License: Public Domain · 2020
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hematopoietic-stem-cell-transplantation
Objectives To evaluate the correlation between BU exposure and post-transplant clinical results (efficacy and safety) to establish the optimal BU treatment window for myeloablative conditioning in Chinese pediatrics, provide theoretical basis and the new strategy for BU individualized dosage, further optimize transplant treatment and reduce drug-related toxicity. Population 500 participants of any sex between the age of 0.1 and 18 years. Patients receiving the BU-based myeloablative conditioning before transplantation. Endpoint primary To establish BU pop-PK model and analyze the association between BU AUC and event-free survival (EFS)or overall survival (OS) after transplantation in Chinese pediatrics. Secondary The investigators are also interested in transplantation-related mortality (TRM), acute toxicity and chronic GvHD.
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