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Allogeneic bone marrow transplantation for Alexander’s disease

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Abstract

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.

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Authors and Affiliations

  1. Department of Pediatric Hematology/Oncology, University of Chicago Children’s Hospital, University of Chicago Hospitals, Chicago, IL, USA

    M-J Staba & S Goldman

  2. Pediatric Hematology/Oncology, Oregon Children’s Cancer Center, Oregon Health Sciences University, Portland, OR, USA

    FL Johnson

  3. Department of Pediatrics, Neurology, and Pediatric Neurology, University of Chicago Children’s Hospital, University of Chicago Hospitals, Chicago, IL, USA

    PR Huttenlocher

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  1. M-J Staba
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  2. S Goldman
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  3. FL Johnson
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  4. PR Huttenlocher
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Staba, MJ., Goldman, S., Johnson, F. et al. Allogeneic bone marrow transplantation for Alexander’s disease. Bone Marrow Transplant 20, 247–249 (1997). https://doi.org/10.1038/sj.bmt.1700871

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  • DOI: https://doi.org/10.1038/sj.bmt.1700871

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