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

doi:10.1111/j.1399-3046.2009.01188.x

Article

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

Pediatric transplantation - 1 Sept 2011

Awaya Tomonari, Kato Takeo, Niwa Akira, Hiramatsu Hidefumi, Umeda Katsutsugu, Watanabe Ken-Ichiro, Shibata Minoru, Yamanaka Yasunari, Maruya Etsuko, Saji Hiroh, Nakahata Tatsutoshi, Adachi Souichi

Abstract excerpt

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...

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