A Case of Allogeneic Stem Cell Transplantation for Chronic Granulomatous Disease.
- Author:
Jae Hee LEE
1
;
Mee Jung LEE
;
Eun Seok CHOI
;
Soo Jong HONG
;
Chan Jeoung PARK
;
Jong Jin SEO
Author Information
1. Department of Pediatrics, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Korea. jjseo@amc.seoul.kr
- Publication Type:Case Report
- Keywords:
Bone marrow transplantation;
Chronic granulomatous disease;
Children
- MeSH:
Bone Marrow Transplantation;
Busulfan;
Child;
Child, Preschool;
Chimerism;
Cyclophosphamide;
Granulomatous Disease, Chronic*;
Humans;
Male;
Mortality;
NADP;
Nitroblue Tetrazolium;
Oxidoreductases;
Phagocytes;
Stem Cell Transplantation*;
Stem Cells*;
Trimethoprim, Sulfamethoxazole Drug Combination;
Unrelated Donors
- From:Korean Journal of Pediatric Hematology-Oncology
2003;10(2):282-286
- CountryRepublic of Korea
- Language:Korean
-
Abstract:
Chronic granulomatous disease (CGD) is a rare primary immunodeficiency in children caused by an abnormal function of the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase in the phagocytic cells, which results in an increased susceptibility to severe bacterial and fungal infections. Prophylactic trimethoprim-sulfamethoxazole improves medium-term survival, but cannot prevent inflammatory sequelae. It still shows high morbidity and mortality. Bone marrow transplantation (BMT) is currently the only curative treatment for CGD. We report on a 29-month-old boy with CGD who was successfully treated with allogeneic BMT from an HLA-identical unrelated donors following a conditioning regimen consisting of busulfan and cyclophosphamide. One year after post-transplantation, the boy is in excellent clinical and hematological condition with complete chimerism and normal nitroblue tetrazolium (NBT) test.
