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Updated: Mar 9, 2026
Busulfan as a Myelosuppressive Agent for Generating Stable High-level Bone Marrow Chimerism in Mice
Published on: April 1, 2015
Targeted busulfan and fludarabine-based conditioning for bone marrow transplantation in chronic granulomatous disease
Hee Young Ju1, Hyoung Jin Kang1, Che Ry Hong1
1Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine, Seoul, Korea.; Cancer Research Institute, Seoul National University College of Medicine, Seoul, Korea.
Insights
Hematopoietic stem cell transplantation (HSCT) offers a cure for Chronic Granulomatous Disease (CGD). A reduced-toxicity conditioning regimen using targeted busulfan and fludarabine demonstrated successful engraftment and minimal toxicity in a CGD patient.
Area of Science:
- Immunology
- Hematology
- Transplantation Medicine
Background:
- Chronic Granulomatous Disease (CGD) is a primary immunodeficiency characterized by impaired phagocytic function.
- Hematopoietic Stem Cell Transplantation (HSCT) is a curative option for CGD but faces challenges like mortality and engraftment failure.
Purpose of the Study:
- To report a successful HSCT in a patient with CGD using a novel reduced-toxicity myeloablative conditioning regimen.
- To evaluate the efficacy and safety of targeted busulfan and fludarabine conditioning for HSCT in CGD.
Main Methods:
- The conditioning regimen involved targeted intravenous busulfan (AUC 75,000 µg·hr/L) for 4 days and fludarabine (40 mg/m²/day) for 6 days.
- Antithymocyte globulin was administered from days -4 to -2.
- The patient underwent HSCT with close monitoring of busulfan dosage.
Main Results:
- Successful engraftment was achieved in the patient.
- The patient experienced no severe transplantation-related toxicity.
- The targeted busulfan and fludarabine regimen proved effective and well-tolerated.
Conclusions:
- Reduced-toxicity myeloablative conditioning with targeted busulfan and fludarabine offers a promising approach for HSCT in CGD.
- Precise regulation of busulfan dosage is crucial for optimizing HSCT outcomes in CGD patients.
Abstract:
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.
