Childhood Adrenoleukodystrophy: Failure of Intensive Immunosuppression to Arrest Neurologic Progression
Abstract
Cyclophosphamide in a dosage of 350 to 700 mg/m2/d was administered for five to 11 days to four patients with childhood adrenoleukodystrophy (ALD) and to one patient with the adult cerebral form of the disease. The rate of neurologic progression in the four patients with childhood ALD did not differ from that of 167 untreated patients with childhood ALD surveyed previously.
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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 Naidu et al., 1988
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- Authors
- Naidu, S.
- Corresponding author
- Naidu, Sakkubai
- Venue
- Archives of Neurology
- Publisher
- American Medical Association (AMA)
- Published
- 1988-08-01
- Volume
- 45
- Issue
- 8
- Pages
- 846
- ISSN
- 0003-9942 (ISSN-L)
- OpenAlex
- W1990906452
- Cited by
- 55
- References
- 10
- Citations by year
- 2025: 2 · 2023: 1 · 2019: 1 · 2018: 1 · 2017: 1 · 2013: 1 · 2012: 2
- FWCI
- 2.25
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Archives of Neurology (publishedVersion)
- PubMed (publishedVersion)
- Funders
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (HD-10981)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Clinical Biochemistry › Metabolism and Genetic Disorders
- Health Sciences › Medicine › Physiology › Lysosomal Storage Disorders Research
- Life Sciences › Neuroscience › Cellular and Molecular Neuroscience › Genetic Neurodegenerative Diseases
- Keywords
- cyclophosphamide, clinical course, treatment outcomes
- MeSH terms
- Adrenoleukodystrophy, Adult, Brain, Diffuse Cerebral Sclerosis of Schilder, Child, Clinical Trials as Topic, Coma, Cyclophosphamide, Humans, Immunosuppressive Agents, Male, Nervous System Diseases, Radiography, Time Factors
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