Adrenoleukodystrophy: Failure of Immunosuppression to Prevent Neurological Progression
Abstract
Severe immunosuppression with prednisone and cyclophosphamide failed to prevent neurological progression in a patient with adrenoleukodystrophy.
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Various forms of immunosuppressive therapy have been tested for X-ALD, so far without success.
“Various forms of immunosuppressive therapy have been tested, so far without success [Stumpf et al., 1981;Naidu et al., 1988;Cappa et al., 1994;Korenke et al., 1997;Moser et al., 2001].”
From ABCD1 mutations and the X-linked adrenoleukodystrophy mutation database: Role in diagnosis and clinical correlations (2001) · cites this paper as Stumpf et al., 1981
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Aubourg, P., Blanche, S., Jambaqué, I., Rocchiccioli, F., Kalifa, G., Naud-Saudreau, C., Rolland, M.-O., Debré, M., Chaussain, J.-L., Griscelli, C., et al. (1990) · New England Journal of Medicine · doi:10.1056/nejm199006283222607 · Cited by 366 · see citing works
Shapiro, E., Krivit, W., Lockman, L., Jambaqué, I., Peters, C., Cowan, M., Harris, R., Blanche, S., Bordigoni, P., Loes, D., et al. (2000) · The Lancet · doi:10.1016/s0140-6736(00)02629-5 · Cited by 329 · see citing works
Kemp, S., Pujol, A., Waterham, H. R., van Geel, B. M., Boehm, C. D., Raymond, G. V., Cutting, G. R., Wanders, R. J. A., & Moser, H. W. (2001) · Human Mutation · doi:10.1002/humu.1227 · Cited by 332 · see citing works
Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy
Gupta, A. O., Raymond, G., Pierpont, E. I., Kemp, S., McIvor, R. S., Rayannavar, A., Miller, B., Lund, T. C., & Orchard, P. J. (2022) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2022.2124857 · Cited by 67 · see citing works
Naidu, S. (1988) · Archives of Neurology · doi:10.1001/archneur.1988.00520320032011 · Cited by 55 · see citing works
- Authors
- Stumpf, D. A.
- Corresponding author
- Stumpf, David A.
- ORCID
- Stumpf, David A.
- Venue
- Archives of Neurology
- Publisher
- American Medical Association (AMA)
- Published
- 1981-01-01
- Volume
- 38
- Issue
- 1
- Pages
- 48
- ISSN
- 0003-9942 (ISSN-L)
- OpenAlex
- W2159972914
- Cited by
- 41
- References
- 0
- Citations by year
- 2025: 1 · 2022: 1 · 2016: 1 · 2013: 1 · 2012: 1
- FWCI
- 1.06
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Archives of Neurology (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Clinical Biochemistry › Metabolism and Genetic Disorders
- Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Adrenal Hormones and Disorders
- Life Sciences › Immunology and Microbiology › Immunology › Immunodeficiency and Autoimmune Disorders
- Keywords
- adrenoleukodystrophy, immunosuppressive therapy, prednisone, cyclophosphamide, treatment failure
- MeSH terms
- Adrenal Insufficiency, Diffuse Cerebral Sclerosis of Schilder, Child, Preschool, Cyclophosphamide, Drug Therapy, Combination, Humans, Prednisone
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