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Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy.

Publication ,  Journal Article
Krivit, W; Shapiro, EG; Peters, C; Wagner, JE; Cornu, G; Kurtzberg, J; Wenger, DA; Kolodny, EH; Vanier, MT; Loes, DJ; Dusenbery, K; Lockman, LA
Published in: N Engl J Med
April 16, 1998

BACKGROUND: Globoid-cell leukodystrophy is caused by a deficiency of galactocerebrosidase, which results in progressive central nervous system deterioration. We investigated whether allogeneic hematopoietic stem-cell transplantation can provide a source of leukocyte galactocerebrosidase and thereby prevent the decline of central nervous system function in patients with the disease. METHODS: Five children with globoid-cell leukodystrophy (one with the infantile type and four with late-onset disease) were treated with allogeneic hematopoietic stem-cell transplantation. Measurement of leukocyte galactocerebrosidase levels, neurologic examinations, neuropsychological tests, magnetic resonance imaging of the central nervous system, cerebrospinal fluid protein assays, and neurophysiologic measurements were performed before and after transplantation, with follow-up ranging from one to nine years. RESULTS: Engraftment of donor-derived hematopoietic cells occurred in all patients and was followed by restoration of normal leukocyte galactocerebrosidase levels. In the four patients with late-onset disease, the central nervous system deterioration was reversed, and in the patient with the infantile form of the disease, signs and symptoms have not appeared. Magnetic resonance imaging showed a decrease in signal intensity in the three patients with late-onset disease who were assessed both before and after transplantation. Abnormalities in cerebrospinal fluid total protein levels were corrected in three patients with late-onset disease and substantially reduced in the patient with the infantile form. CONCLUSIONS: Central nervous system manifestations of globoid-cell leukodystrophy can be reversed by allogeneic hematopoietic stem-cell transplantation.

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Published In

N Engl J Med

DOI

ISSN

0028-4793

Publication Date

April 16, 1998

Volume

338

Issue

16

Start / End Page

1119 / 1126

Location

United States

Related Subject Headings

  • Transplantation, Homologous
  • Male
  • Leukodystrophy, Globoid Cell
  • Leukocytes
  • Infant
  • Humans
  • Hematopoietic Stem Cell Transplantation
  • General & Internal Medicine
  • Galactosylceramidase
  • Female
 

Citation

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Krivit, W., Shapiro, E. G., Peters, C., Wagner, J. E., Cornu, G., Kurtzberg, J., … Lockman, L. A. (1998). Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy. N Engl J Med, 338(16), 1119–1126. https://doi.org/10.1056/NEJM199804163381605
Krivit, W., E. G. Shapiro, C. Peters, J. E. Wagner, G. Cornu, J. Kurtzberg, D. A. Wenger, et al. “Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy.N Engl J Med 338, no. 16 (April 16, 1998): 1119–26. https://doi.org/10.1056/NEJM199804163381605.
Krivit W, Shapiro EG, Peters C, Wagner JE, Cornu G, Kurtzberg J, et al. Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy. N Engl J Med. 1998 Apr 16;338(16):1119–26.
Krivit, W., et al. “Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy.N Engl J Med, vol. 338, no. 16, Apr. 1998, pp. 1119–26. Pubmed, doi:10.1056/NEJM199804163381605.
Krivit W, Shapiro EG, Peters C, Wagner JE, Cornu G, Kurtzberg J, Wenger DA, Kolodny EH, Vanier MT, Loes DJ, Dusenbery K, Lockman LA. Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy. N Engl J Med. 1998 Apr 16;338(16):1119–1126.
Journal cover image

Published In

N Engl J Med

DOI

ISSN

0028-4793

Publication Date

April 16, 1998

Volume

338

Issue

16

Start / End Page

1119 / 1126

Location

United States

Related Subject Headings

  • Transplantation, Homologous
  • Male
  • Leukodystrophy, Globoid Cell
  • Leukocytes
  • Infant
  • Humans
  • Hematopoietic Stem Cell Transplantation
  • General & Internal Medicine
  • Galactosylceramidase
  • Female