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

Korean J Pediatr 2016 Nov 30;59(Suppl 1):S57-S59. Epub 2016 Nov 30.

Department 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.

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) 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.

Download full-text PDF

Source
http://dx.doi.org/10.3345/kjp.2016.59.11.S57DOI Listing
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5177714PMC
November 2016
76 Reads

Publication Analysis

Top Keywords

targeted busulfan
12
underwent successful
8
administered daily
8
chronic granulomatous
8
granulomatous disease
8
busulfan fludarabine
8
daily consecutive
8
consecutive days
8
days days
8
days
5
area curve
4
myeloablative conditioning
4
conditioning intravenous
4
busulfan administered
4
days target
4
intravenous busulfan
4
target area
4
fludarabine reduced-toxicity
4
failure report
4
report case
4

Similar Publications