Busulfan Pharmacokinetics in ADA SCID Gene Therapy

Busulfan (1,4-butanediol dimethanesulfonate) is a bifunctional alkylating agent that is cytotoxic to hematopoietic stem and progenitor cells (HSPC).1 Busulfan (BU) is widely used for cytoreduction (full or partial) in both myeloablative and reduced intensity allogeneic hematopoietic stem cell transplant (HSCT) conditioning regimens and prior to autologous gene therapy (GT). Appropriate BU dosing is critical to the success of HSCT, because low BU plasma exposure can result in engraftment failure and high BU exposure can increase regimen-related toxicity.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research