Rare Spontaneous Attenuation of Childhood Inflammatory Cerebral Adrenoleukodystrophy

被引:1
作者
Choi, Hyoung Won [1 ]
Raymond, Gerald Vincent [2 ]
Miller, Weston [3 ]
机构
[1] Univ Minnesota, Div Pediat Neurol, Dept Neurol, Minneapolis, MN 55455 USA
[2] Penn State Univ, Div Pediat Neurol, Dept Pediat & Neurol, Hershey, PA USA
[3] Univ Minnesota, Dept Pediat, Div Pediat Blood & Marrow Transplantat, Minneapolis, MN 55455 USA
关键词
cerebral adrenoleukodystrophy; inflammatory; spontaneous arrest; HEMATOPOIETIC-CELL TRANSPLANTATION; X-LINKED ADRENOLEUKODYSTROPHY; ONSET;
D O I
10.1055/s-0039-1677805
中图分类号
R72 [儿科学];
学科分类号
100202 ;
摘要
X-linked adrenoleukodystrophy (ALD) is a neurodegenerative peroxisomal disorder with variable clinical phenotypes. Childhood cerebral ALD (CCALD) is at the most severe end of the disease spectrum. In CCALD, the clinical manifestations include increasing deficits in behavior, vision, hearing, coordination, and motor function, as well as seizures. Without treatment, CCALD often results in apparent vegetative state within 1 to 2 years of appearance of initial signs and symptoms. We present the case of a boy with classic inflammatory CCALD who exhibited spontaneous attenuation in disease progression. While extremely rare, spontaneous arrest of disease progression may occur in boys with inflammatory CCALD.
引用
收藏
页码:106 / 109
页数:4
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