title: A 12-week open-label, multicenter study evaluating the safety and patient-reported efficacy of sodium oxybate in patients with narcolepsy and cataplexy.
abstract: OBJECTIVE
  This study aimed to evaluate safety and efficacy of sodium oxybate (SXB) titrated to effect.
  
  
  METHODS
  SXB-naive patients who had participated in a randomized SXB clinical trial and had not been titrated to adequate clinical effect were initiated on open-label SXB at 4.5 g/night and titrated in 1.5-g increments to 6, 7.5, or 9 g/night or down to 3 g/night, based on individual clinical response. Treatment was 12 weeks; safety was the primary outcome. Efficacy was evaluated using the Narcolepsy Symptom Assessment Questionnaire (NSAQ), a five-point scale ("much improved" to "much worse") that assessed changes from baseline in specific symptoms. Response was defined as "much improved" or "somewhat improved" overall at weeks 6 and 12.
  
  
  RESULTS
  Of 202 patients, 171 (85%) completed treatment; final doses were 3 g (n = 5), 4.5 g (n = 29), 6 g (n = 80), 7.5 g (n = 66), and 9 g (n = 22). Adverse events (AEs) were reported in 114 patients (56%), serious AEs in five (2%). The most common AEs were nausea (10%), headache (7%), and dizziness (5%). Response rate was 92% at week 6 and 90% at week 12; most patients reported improvements in all individual symptoms. Overall, 60% of patients rated their symptoms at 12 weeks as "much improved," and this improvement was dose dependent.
  
  
  CONCLUSIONS
  The SXB safety profile was consistent with parent trials. Ninety percent of patients reported improvements as measured by the NSAQ.
authors.affiliation: Department of Psychiatry, University of Toronto, Toronto, ON, Canada. Electronic address: m.mamelak@utoronto.ca.
authors.forename: Mortimer
authors.initials: M
authors.lastname: Mamelak
authors.affiliation: Neurology and Sleep Medicine Consultants of Houston, University of Texas-Houston School of Medicine, Houston, TX, USA.
authors.forename: Todd
authors.initials: T
authors.lastname: Swick
authors.affiliation: George Washington University School of Medicine, Washington, DC, USA; The Center for Sleep & Wake Disorders, Chevy Chase, MD, USA.
authors.forename: Helene
authors.initials: H
authors.lastname: Emsellem
authors.affiliation: Department of Psychiatry, Université de Montréal, Montréal, Québec, Canada.
authors.forename: Jacques
authors.initials: J
authors.lastname: Montplaisir
authors.affiliation: Jazz Pharmaceuticals, Inc., Palo Alto, CA, USA.
authors.forename: Chinglin
authors.initials: C
authors.lastname: Lai
authors.affiliation: Jazz Pharmaceuticals, Inc., Palo Alto, CA, USA; Stanford Center for Sleep Research and Medicine, Stanford, CA, USA.
authors.forename: Jed
authors.initials: J
authors.lastname: Black
mesh_terms: D000759:Adjuvants, Anesthesia / Q000627:therapeutic use*; D000328:Adult; D002385:Cataplexy / Q000150:complications / Q000188:drug therapy* / Q000523:psychology; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D063868:Patient Outcome Assessment; D012978:Sodium Oxybate / Q000627:therapeutic use*; D063189:Symptom Assessment; D016896:Treatment Outcome
chemical_list: D000759:Adjuvants, Anesthesia; D012978:Sodium Oxybate
keywords: Attention; Cataplexy; Efficacy; Excessive daytime sleepiness; Narcolepsy; Open-label; Patient-reported outcomes; Safety; Sleep quality; Sodium oxybate