Off-label treatment of severe childhood narcolepsy-cataplexy with sodium oxybate.

摘要: STUDY OBJECTIVES To evaluate the efficacy and side-effect profile of off-label sodium oxybate (gamma hydroxy butyrate) therapy in severe childhood narcolepsy-cataplexy. DESIGN Retrospective; chart review. SETTING A multidisciplinary tertiary sleep center. PATIENTS group eight children with narcolepsy-cataplexy diagnosed on basis clinical history, nocturnal polysomnography multiple latency test were studied. modified Epworth Sleepiness Scale an arbitrary cataplexy severity scale (1 = minimal weakness, 2 voluntarily preventable falls, 3 falls to ground) utilized. INTERVENTIONS Sodium therapy; concurrent medications maintained. MEASUREMENTS AND RESULTS Before therapy, all subjects had suboptimally controlled sleepiness cataplexy. Following treatment oxybate, 7/8 (88%) improved. Cataplexy frequency decreased from a median 38.5 4.5/ week (p 0.0078). 2.75 1.75 0.06). The Scores improved 19 12.5 0.02). Suicidal ideation, dissociative episodes, tremor constipation occurred one subject each terminal insomnia two. Three 8 (38%) discontinued therapy. Two stopped drug owing side effects due problems postal delivery medication. CONCLUSIONS This is first report Our finding improvement suggests that this medication effective treating