Prevention of deterioration in metachromatic leukodystrophy by bone marrow transplantation

W. Krivit, M. E. Lipton, L. A. Lockman, M. Tsai, P. J. Dyck, S. Smith, N. K. Ramsay, J. Kersey

Research output: Contribution to journalArticlepeer-review

31 Scopus citations

Abstract

The first girl in a family was affected with late infantile metachromatic leukodystrophy (MLD) and had the expected characteristic central nervous system progressive deterioration, which resulted in decerebration and death. The second girl (propositus) demonstrated similar symptoms and signs at the same age. Both girls had characteristically low arylsulfatase A levels. The propositus underwent allogeneic bone transplantation (BMT) from a normal histocompatible sibling. Two and a half years later, the propositus has not developed the intellectual and neurologic impairment demonstrated by the first sibling, although nerve conduction has continued to worsen. These results suggest that the induction of normal enzyme levels by BMT may be retarding or inhibiting CNS deterioration. These results, confirming earlier results of others, are sufficiently promising to warrant a larger scale critical trial of BMT early in the course of MLD.

Original languageEnglish (US)
Pages (from-to)80-85
Number of pages6
JournalAmerican Journal of the Medical Sciences
Volume294
Issue number2
DOIs
StatePublished - 1987

Bibliographical note

Funding Information:
From the Department of Pediatrics,* Division of Pediatric Neurology,t Department of Laboratory Medicine and Pathology,:J; Peripheral Nerve Laboratory, Mayo Medical School,§ Bone Marrow Transplantation Program at Variety Club Children's Hospital, University Hospitals, University of Minnesota Medical School, Minneapolis, Minnesota. Supported in part by a grant from the NIH (CA 21737), by donations from the public in Mankato, Minnesota, and a gift from Blue Cross/Blue Shield of Minnesota. The authors acknowledge the importance of consultation and review of manuscript by Dr. Gunnar Lund (Neurology), Dr. W. Kennedy (Motor Nerve Conduction), and Dr. F. Torres (EEG labo· ratory). The BMT team of the University of Minnesota, the nursing staff, and the house staff all have contributed. Preliminary data was reported on the patient at Pediatric National Blood Club, June 1985, Washington D.C., in the March of Dimes Birth Defects Series (Vol. 22, No.1, 1986, pp 57-68), and the Child Neurology Society Meeting, Memphis, Tennessee, October 1985. The authors also thank Ms. Sheryl Frankel for typing this manuscript. Reprint requests: William Krivit, MD, PhD, Box 477, University of Minnesota Hospital and Clinic, Minneapolis, MN 55455.

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