Successful cord blood transplantation using a reduced-intensity conditioning regimen for advanced childhood-onset cerebral adrenoleukodystrophy

被引:10
作者
Awaya, Tomonari [1 ]
Kato, Takeo [1 ]
Niwa, Akira [1 ]
Hiramatsu, Hidefumi [1 ]
Umeda, Katsutsugu [1 ]
Watanabe, Ken-ichiro [1 ]
Shibata, Minoru [1 ]
Yamanaka, Yasunari [1 ]
Maruya, Etsuko [2 ]
Saji, Hiroh [2 ]
Nakahata, Tatsutoshi [1 ]
Adachi, Souichi [1 ]
机构
[1] Kyoto Univ, Grad Sch Med, Dept Pediat, Kyoto 6068507, Japan
[2] HLA Lab, Kyoto, Japan
关键词
adrenoleukodystrophy; cord blood stem cell transplantation; hematopoietic stem cell transplantation; reduced-intensity stem cell transplantation; X-LINKED ADRENOLEUKODYSTROPHY; BONE-MARROW-TRANSPLANTATION; STEM-CELL TRANSPLANTATION; HIGH-DOSE BUSULFAN; CYCLOPHOSPHAMIDE; ABNORMALITY; PROGNOSIS; CHILDREN; LEUKEMIA; THERAPY;
D O I
10.1111/j.1399-3046.2009.01188.x
中图分类号
R72 [儿科学];
学科分类号
100202 ;
摘要
The CCALD, which is caused by a mutation of the ABCD1 gene that encodes a peroxisomal membrane protein, progresses to a stage where the patient is in a vegetative state and can cause death within 3-5 yr after the appearance of neurological symptoms. Although HSCT is the only means of preventing the progression of this disease, HSCT is currently recommended only for cases diagnosed in the early stages. Previous reports on HSCT in advanced CCALD have indicated that the complications of the HSCT procedure seem to outweigh its benefits with respect to survival and neurological outcome. In this case, we successfully treated advanced CCALD with CBT using a reduced-intensity conditioning regimen to reduce regimen-related toxicity and transplant-associated morbidity and mortality. Neither neurological deterioration nor deterioration of MRI abnormalities were observed during the clinical course. We report that CBT using the reduce-dintensity conditioning regimen was well tolerated, stopped disease progression and contributed to a good neuropsychological outcome in this case of advanced CCALD.
引用
收藏
页码:E116 / E120
页数:5
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