PRESYMPTOMATIC LATE-INFANTILE METACHROMATIC LEUKODYSTROPHY TREATED WITH BONE-MARROW TRANSPLANTATION

被引：16

作者：

PRIDJIAN, G ^{[1
]}

HUMBERT, J ^{[1
]}

WILLIS, J ^{[1
]}

SHAPIRA, E ^{[1
]}

机构：

[1] TULANE UNIV, SCH MED, HUMAN GENET PROGRAM, NEW ORLEANS, LA 70112 USA

来源：

JOURNAL OF PEDIATRICS | 1994年 / 125卷 / 05期

关键词：

D O I：

10.1016/S0022-3476(94)70072-9

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

At 8 months of age, before clinical neurologic deterioration, the younger of two sisters with metachromatic leukodystrophy received a transplant of bone marrow from her haploidentical, heterozygote mother. Compared with the course in the older, affected, untreated sibling, the onset of neurologic regression was delayed 1 year and progressed at a slower rate.

引用

页码：755 / 758

页数：4

共 9 条

[1]

BAYEVER E, 1985, LANCET, V1, P471

[2] INCIDENCE AND GENETICS OF METACHROMATIC LEUCODYSTROPHY IN NORTHERN SWEDEN [J].

GUSTAVSON, KH ;

HAGBERG, B .

ACTA PAEDIATRICA SCANDINAVICA, 1971, 60 (05) :585-+

[3]

HASKINS M, 1991, TREATMENT GENETIC DI

[4] PREVENTION OF DETERIORATION IN METACHROMATIC LEUKODYSTROPHY BY BONE-MARROW TRANSPLANTATION [J].

KRIVIT, W ;

LIPTON, ME ;

LOCKMAN, LA ;

TSAI, M ;

DYCK, PJ ;

SMITH, S ;

RAMSAY, NKC ;

KERSEY, J .

AMERICAN JOURNAL OF THE MEDICAL SCIENCES, 1987, 294 (02) :80-85

[5] TREATMENT OF LATE INFANTILE METACHROMATIC LEUKODYSTROPHY BY BONE-MARROW TRANSPLANTATION [J].

KRIVIT, W ;

SHAPIRO, E ;

KENNEDY, W ;

LIPTON, M ;

LOCKMAN, L ;

SMITH, S ;

SUMMERS, CG ;

WENGER, DA ;

TSAI, MY ;

RAMSAY, NKC ;

KERSEY, JH ;

YAO, JK ;

KAYE, E .

NEW ENGLAND JOURNAL OF MEDICINE, 1990, 322 (01) :28-32

[6]

KRIVIT W, 1993, BONE MARROW TRANSPLA

[7]

Lipton M, 1986, Birth Defects Orig Artic Ser, V22, P57

[8]

Neufeld E F, 1973, Birth Defects Orig Artic Ser, V9, P27

[9]

RINGDEN O, 1990, TRANSPL P, V22, P198

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