Premium
Rett syndrome: Controlled study of an oral opiate antagonist, naltrexone
Author(s) -
Percy Alan K.,
Glaze Daniel G.,
Schultz Rebecca J.,
Zoghbi Huda Y.,
Williamson Daniel,
Frost James D.,
Jankovic Joseph J.,
del Junco Deborah,
Skender Martha,
Waring Steve,
Myer Edwin C.
Publication year - 1994
Publication title -
annals of neurology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 4.764
H-Index - 296
eISSN - 1531-8249
pISSN - 0364-5134
DOI - 10.1002/ana.410350415
Subject(s) - rett syndrome , naltrexone , placebo , crossover study , psychology , anesthesia , opiate , medicine , antagonist , gene , biochemistry , chemistry , receptor , alternative medicine , pathology
Hypothesis: The opiate antagonist, naltrexone, will be beneficial in Rett syndrome. Subjects: Twenty‐five individuals fulfilling the criteria for Rett syndrome. Method: Randomized, double‐blind, placebo‐controlled crossover trial with two treatment periods, 4 months each, and an intervening 1‐month washout period. Clinical stage, motor and cognitive development, motor–behavioral analysis, neurophysiological parameters (computerized electroencephalographic analysis, breathing characteristics, quantification of stereotyped hand movements, and sleep characteristics), and cerebrospinal fluid β‐endorphin measurements were evaluated at baseline and at the end of each treatment period. Results: Only data from the first period of this study were analyzed due to significant sequence effects in the crossover design. This analysis indicated positive effects on certain respiratory characteristics including decreased disorganized breathing during wakefulness. Four (40%) of the individuals receiving naltrexone progressed one or more clinical stages versus none of the individuals receiving placebo. The adjusted (for baseline value and Rett stage) end of treatment psychomotor test age (Bayley Scales) was significantly higher for the placebo group. There was no significant change for the other parameters. Conclusion: Naltrexone may modify some of the respiratory disturbance in Rett syndrome. Declines in motor function and more rapid progression of the disorder suggest a deleterious effect.