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.
Department of Neurology, Columbia University Medical Center, New York, New York 10032, USA. sf216@columbia.edu
Steven J
SJ
Frucht
Yvette
Y
Bordelon
William H
WH
Houghton
Dayton
D
Reardan
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