Rett syndrome: controlled study of an oral opiate antagonist, naltrexone.
OBJECTIVE
The opiate antagonist, naltrexone, will be beneficial in Rett syndrome.


METHODS
Twenty-five individuals fulfilling the criteria for Rett syndrome.


METHODS
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 beta-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.


CONCLUSIONS
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.
Percy
A K
AK
University of Alabama at Birmingham.
Glaze
D G
DG
Schultz
R J
RJ
Zoghbi
H Y
HY
Williamson
D
D
Frost
J D
JD
Jankovic
J J
JJ
del Junco
D
D
Skender
M
M
Waring
S
S
D000293:Adolescent; D001921:Brain / Q000187:drug effects / Q000503:physiopathology; D002648:Child; D002675:Child, Preschool; D004311:Double-Blind Method; D004594:Electrophysiology; D005260:Female; D006801:Humans; D009271:Naltrexone / Q000627:therapeutic use*; D015518:Rett Syndrome / Q000188:drug therapy* / Q000503:physiopathology
D009271:Naltrexone