【摘 要】
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BACKGROUND: Childhood cerebral X-linked adrenoleukodystrophy is a rapidly progressive neurodegenerative disorder that affects central nervous system myelin
【机 构】
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Guangzhou Women and Children's Medical Center Department of Hematology
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BACKGROUND: Childhood cerebral X-linked adrenoleukodystrophy is a rapidly progressive neurodegenerative disorder that affects central nervous system myelin and the adrenal cortex.Hematopoietic stem cell transplantation is the best available curative therapy if performed during the early stages of disease.Only 30% of patients who require the hematopoietic stem cell transplant will have a full human leukocyte antigen (HLA)-matched donor, which is considered to be the best choice.PATIENT DESCRIPTION: We present a 5-year-old boy with cerebral X-linked adrenoleukodystrophy, whose brain magnetic resonance imaging severity score was 7 and who needed an immediate transplantation without an available full HLA-matched donor.
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