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Progression of X‐linked adrenoleukodystrophy under interferon‐β therapy
Author(s) -
Korenke G. C.,
Christen H.J.,
Kruse B.,
Hunneman D. H.,
Hanefeld F.
Publication year - 1997
Publication title -
journal of inherited metabolic disease
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.462
H-Index - 102
eISSN - 1573-2665
pISSN - 0141-8955
DOI - 10.1023/a:1005361607523
Subject(s) - adrenoleukodystrophy , medicine , interferon , gastroenterology , endocrinology , pathology , immunology , peroxisome , receptor
The cerebral phenotype of X‐linked adrenoleukodystrophy (ALD) is a rapidly progressive neurodegenerative disorder characterized by a cerebral inflammatory response and elevated very long‐chain fatty acids (VLCFA). Interferon‐β (INFB) is known to suppress the synthesis of tumour necrosis factor α and interferon‐γ, which have been reported to be elevated in the margin of the areas of demyelination in ALD brains. We report on treatment with interferon‐β in 8 patients with cerebral ALD, who additionally received glycerol trioleate/glycerol trierucate. INFB‐1a (Rebif, Serono, Switzerland) was given subcutaneously once a week, 3 million units for the first 3 months and 6 million units for the next 9 months. All patients showed an unimpeded progression of neurological symptoms during INFB therapy. Therapy was stopped within 6 months in 4 patients because of the fast neurological deterioration with loss of walking. In all patients the MRI demonstrated a progression of demyelination with a qualitatively unchanged gadolinium enhancement. Further studies are needed to elucidate the pathomechanism of demyelination in ALD in order to find an effective therapy for cerebral ALD patients.