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

• Main Authors: Hee Young Ju (Author), Hyoung Jin Kang (Author), Che Ry Hong (Author), Ji Won Lee (Author), Hyery Kim (Author), Sang Hoon Song (Author), Kyung-Sang Yu (Author), In-Jin Jang (Author), June Dong Park (Author), Kyung Duk Park (Author), Hee Young Shin (Author), Joong-Gon Kim (Author), Hyo Seop Ahn (Author)
• Format: Book
• Published: Korean Pediatric Society, 2016-11-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Summary

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/m2) 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.

Item Description

1738-1061
2092-7258
10.3345/kjp.2016.59.11.S57