Targeted busulfan and fludarabine-based conditioning for bone marrow transplantation in chronic granulomatous disease.
10.3345/kjp.2016.59.11.S57
- Author:
Hee Young JU
1
;
Hyoung Jin KANG
;
Che Ry HONG
;
Ji Won LEE
;
Hyery KIM
;
Sang Hoon SONG
;
Kyung Sang YU
;
In Jin JANG
;
June Dong PARK
;
Kyung Duk PARK
;
Hee Young SHIN
;
Joong Gon KIM
;
Hyo Seop AHN
Author Information
1. Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine, Seoul, Korea. kanghj@snu.ac.kr
- Publication Type:Case Report
- Keywords:
Chronic granulomatous disease;
Bone marrow transplantation;
Transplantation conditioning;
Busulfan;
Fludarabine
- MeSH:
Antilymphocyte Serum;
Bone Marrow Transplantation*;
Bone Marrow*;
Busulfan*;
Granulomatous Disease, Chronic*;
Hematopoietic Stem Cell Transplantation;
Humans;
Mortality;
Transplantation Conditioning
- From:Korean Journal of Pediatrics
2016;59(Suppl 1):S57-S59
- CountryRepublic of Korea
- Language:English
-
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.
