Immune response in leukodystrophies

Pediatr Neurol. 2007 Oct;37(4):235-44. doi: 10.1016/j.pediatrneurol.2007.06.011.

Abstract

Although the genetics and biochemistry of leukodystrophies have been extensively explored, the immune response in these disorders has received relatively little attention. Both the disease course and its response to treatment may be highly dependent on the immune system. In this review, we compare three common leukodystrophies, each with a different immune response: (1) X-linked adrenoleukodystrophy, which demonstrates a severe, lymphocytic inflammatory response; (2) metachromatic leukodystrophy, which yields a histiocytic response; and (3) vanishing white-matter disease, in which no inflammation is typically seen. We highlight the biochemical, pathologic, and clinical differences, while focusing on the immune response in each disease. We also review the response of leukodystrophies to immunomodulatory therapies and interventions such as hematopoietic stem-cell transplantation. Future studies may delineate specific inflammatory markers as possible candidates for therapeutic intervention.

Publication types

  • Review

MeSH terms

  • Adrenoleukodystrophy / immunology
  • Antibody Formation
  • Brain Diseases / immunology*
  • Brain Diseases / metabolism
  • Brain Diseases / pathology
  • Brain Diseases / therapy
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Immunologic Factors / therapeutic use
  • Leukodystrophy, Metachromatic / immunology

Substances

  • Immunologic Factors