Allogeneic bone marrow transplantation for Alexander's disease

Bone Marrow Transplant. 1997 Aug;20(3):247-9. doi: 10.1038/sj.bmt.1700871.


In this case report, we evaluate the efficacy of allogeneic bone marrow transplantation (BMT) in a 7-month-old female with the infantile form of Alexander's disease. Based on research that describes Alexander's disease as a leukodystrophy which may result from an unidentified enzyme deficiency, we attempted marrow transplantation to reverse or arrest the patient's neurological deterioration. Despite an initial return to her pretransplant neurological state, the patient's neurological status deteriorated. Marrow transplantation was not effective in changing her prognosis with Alexander's disease.

Publication types

  • Case Reports

MeSH terms

  • Bone Marrow Transplantation*
  • Demyelinating Diseases / pathology
  • Demyelinating Diseases / physiopathology
  • Demyelinating Diseases / therapy*
  • Female
  • Humans
  • Infant
  • Transplantation, Homologous