Unrelated umbilical cord blood transplant for juvenile metachromatic leukodystrophy: A 5-year follow-up in three affected siblings

被引：20

作者：

Cable, Casey ^{[1
]}

Finkel, Richard S. ^{[2
]}

Lehky, Tanya J. ^{[3
]}

Biassou, Nadia M. ^{[4
]}

Wiggs, Edythe A. ^{[5
]}

Bunin, Nancy ^{[6
]}

Pierson, Tyler Mark ^{[1
]}

机构：

[1] NINDS, Neurogenet Branch, NIH, Bethesda, MD 20892 USA

[2] Childrens Hosp Philadelphia, Div Neurol, Philadelphia, PA 19104 USA

[3] NINDS, EMG Sect, NIH, Bethesda, MD 20892 USA

[4] NIH, Div Neuroradiol, Dept Radiol & Imaging Sci, Bethesda, MD 20892 USA

[5] NINDS, Dev & Metab Neurol Branch, NIH, Bethesda, MD 20892 USA

[6] Childrens Hosp Philadelphia, Div Oncol, Philadelphia, PA 19104 USA

来源：

MOLECULAR GENETICS AND METABOLISM | 2011年 / 102卷 / 02期

关键词：

Umbilical cord blood transplant; Metachromatic leukodystrophy; Neurological disease; Neuroimaging; BONE-MARROW-TRANSPLANTATION; NERVE-CONDUCTION; STABILIZATION; GENETICS; OUTCOMES; CHILDREN; THERAPY;

D O I：

10.1016/j.ymgme.2010.10.002

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Unrelated umbilical cord blood transplantation (UCBT) was used to treat three siblings with juvenile metachromatic leukodystrophy (jMLD). The efficacy of this therapy was measured over a 5-year period with serial neurological examinations, neuroimaging, nerve conduction studies (NCS), and neuropsychological evaluations (NPE). Outcomes were a function of disease stage at time of UCBT with alteration of disease course occurring in the first 2 years after UCBT and then subsequent halting of progression and stabilization of symptoms and disease. Published by Elsevier Inc.

引用

页码：207 / 209

页数：3

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