Targeted busulfan and fludarabine-based conditioning for bone marrow transplantation in chronic granulomatous disease

Chronic granulomatous disease (CGD) is a primary immunodeficiency disease caused by impaired phagocytic function.Hematopoietic stem cell transplantation (HSCT) is a definitive cure for CGD; however, the use of HSCT is limited because of associated problems, including transplantation-related mortality and engraftment failure.We report a case of a patient with CGD who underwent successful HSCT following a targeted busulfan and fludarabine reduced-toxicity myeloablative conditioning.Intravenous busulfan was administered once daily for 4 consecutive days (days -8 to -5), and the target area under the curve was 75,000 µg•hr/L.Fludarabine (40 mg/m 2 ) was administered once daily for 6 consecutive days from days -8 to -3.Antithymocyte globulin (2.5 mg/kg/day) was administered from days -4 to -2.The patient underwent successful engraftment and did not have any severe toxicity related to the transplantation.Conditioning with a targeted busulfan and fludarabine regimen could provide a better outcome for HSCT in CGD, with close regulation of the busulfan dose.