A pilot tolerability and efficacy trial of sodium oxybate in ethanol-responsive movement disorders.
Sodium oxybate is currently approved in the United States exclusively for the treatment of cataplexy in narcoleptic patients. In a prior article published in this journal, we reported a patient with severe posthypoxic myoclonus whose myoclonus improved with ethanol and also with treatment with sodium oxybate. We extend this preliminary observation to five other patients with ethanol-responsive movement disorders in an open-label, dose-titration, add-on, 8-week trial. All five patients (one with severe alcohol-responsive posthypoxic myoclonus, two with epsilon-sarcoglycan-linked myoclonus-dystonia, and two with essential tremor) experienced improvement from baseline of 50% or greater as measured by blinded videotape review. Tolerability was satisfactory, with dose-dependent sedation as the most common side effect. Further studies of this drug in hyperkinetic movement disorders are warranted.
Frucht
Steven J
SJ
Department of Neurology, Columbia University Medical Center, New York, New York 10032, USA. sf216@columbia.edu
Bordelon
Yvette
Y
Houghton
William H
WH
Reardan
Dayton
D
D000759:Adjuvants, Anesthesia / Q000009:adverse effects / Q000627:therapeutic use*; D000328:Adult; D000368:Aged; D002492:Central Nervous System Depressants / Q000009:adverse effects / Q000627:therapeutic use*; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D004421:Dystonia / Q000175:diagnosis / Q000188:drug therapy*; D020329:Essential Tremor / Q000175:diagnosis / Q000188:drug therapy*; D000431:Ethanol / Q000009:adverse effects / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D008297:Male; D009207:Myoclonus / Q000175:diagnosis / Q000188:drug therapy*; D010865:Pilot Projects; D012720:Severity of Illness Index; D012978:Sodium Oxybate / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
D000759:Adjuvants, Anesthesia; D002492:Central Nervous System Depressants; D000431:Ethanol; D012978:Sodium Oxybate