Brain-sparing cord blood transplantation for the borderline stage of adrenoleukodystrophy

Yutaro Yada,Michiko Torio,Yuhki Koga,Fumiya Yamashita,Takuya Ichimura,Katsuhide Eguchi,Masataka Ishimura,Yuichi Mushimoto,Akio Hiwatashi,Momoko Sasazuki,Ryutaro Kira,Yasunari Sakai,Shouichi Ohga

Molecular Genetics and Metabolism Reports（2021）

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摘要

•Therapeutic strategies remain to be established for adrenoleukodystrophy.•Multidisciplinary discussions are necessary for making a decision of treatment.•A 6-year-old boy achieved a successful engraftment after the transplantation.•The brain-sparing method might provide favorable outcomes for adrenoleukodystrophy.

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关键词

Adrenoleukodystrophy,Loes score,Reduced-intensity conditioning,Brain-sparing irradiation,Hematopoietic cell transplantation

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