Clinical and Experimental Pediatrics

The Korean Pediatric Society (대한소아청소년과학회)
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Targeted busulfan and fludarabine-based conditioning for bone marrow transplantation in chronic granulomatous disease

  • Ju, Hee Young (Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine) ;
  • Kang, Hyoung Jin (Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine) ;
  • Hong, Che Ry (Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine) ;
  • Lee, Ji Won (Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine) ;
  • Kim, Hyery (Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine) ;
  • Song, Sang Hoon (Department of Laboratory Medicine, Seoul National University College of Medicine) ;
  • Yu, Kyung-Sang (Department of Clinical Pharmacology and Therapeutics, Seoul National University College of Medicine) ;
  • Jang, In-Jin (Department of Clinical Pharmacology and Therapeutics, Seoul National University College of Medicine) ;
  • Park, June Dong (Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine) ;
  • Park, Kyung Duk (Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine) ;
  • Shin, Hee Young (Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine) ;
  • Kim, Joong-Gon (Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine) ;
  • Ahn, Hyo Seop (Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine)
  • Received : 2014.05.27
  • Accepted : 2014.09.25
  • Published : 2016.11.15
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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 $75,000{\mu}g{\cdot}hr/L$. Fludarabine ($40mg/m^2$) 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.

Keywords

References

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