title: Clinical and therapeutic aspects of childhood narcolepsy-cataplexy: a retrospective study of 51 children.
abstract: OBJECTIVE
  to report on symptoms and therapies used in childhood narcolepsy-cataplexy.
  
  
  METHODS
  retrospective series of 51 children who completed the Stanford Sleep Inventory. HLA-DQB1*0602 typing (all tested, and 100% positive), polysomnography or Multiple Sleep Latency Test (76%), and cerebrospinal fluid hypocretin-1 measurements (26%, all with low levels) were also conducted. Prospective data on medication response was collected in 78% using a specially designed questionnaire.
  
  
  RESULTS
  patients were separated into children with onset of narcolepsy prior to (53%), around (29%), and after (18%) puberty. None of the children had secondary narcolepsy. Clinical features were similar across puberty groups, except for sleep paralysis, which increased in frequency with age. Common features included excessive weight gain (84% ≥ 4 kg within 6 months of onset of narcolepsy) and earlier puberty (when compared with family members), notably in subjects who gained the most weight. Streptococcus-positive throat infections were reported in 20% of cases within 6 months of onset of narcolepsy. Polysomnographic features were similar across groups, but 3 prepubertal children did not meet Multiple Sleep Latency Test diagnostic criteria. Regarding treatment, the most used and continued medications were modafinil (84% continued), sodium oxybate (79%), and venlafaxine (68%). Drugs such as methylphenidate, tricyclic antidepressants, or selective serotonin reuptake inhibitors were often tried but rarely continued. Modafinil was reported to be effective for treating sleepiness, venlafaxine for cataplexy, and sodium oxybate for all symptoms, across all puberty groups. At the conclusion of the study, half of children with prepubertal onset of narcolepsy were treated "off label" with sodium oxybate alone or with the addition of one other compound. In older children, however, most patients needed more than 2 drugs.
  
  
  CONCLUSIONS
  this study reports on the clinical features of childhood narcolepsy and documents the safe use of treatments commonly used in adults in young children.
authors.affiliation: Neuro-Pediatric Unit, Shaare Zedek Medical Center, Jerusalem, Israel.
authors.forename: Adi
authors.initials: A
authors.lastname: Aran
authors.forename: Mali
authors.initials: M
authors.lastname: Einen
authors.forename: Ling
authors.initials: L
authors.lastname: Lin
authors.forename: Guiseppe
authors.initials: G
authors.lastname: Plazzi
authors.forename: Seiji
authors.initials: S
authors.lastname: Nishino
authors.forename: Emmanuel
authors.initials: E
authors.lastname: Mignot
mesh_terms: D000759:Adjuvants, Anesthesia / Q000627:therapeutic use; D000293:Adolescent; D017677:Age Distribution; D017668:Age of Onset; D018687:Antidepressive Agents, Second-Generation / Q000627:therapeutic use; D001559:Benzhydryl Compounds / Q000627:therapeutic use; D000697:Central Nervous System Stimulants / Q000627:therapeutic use; D002648:Child; D003511:Cyclohexanols / Q000627:therapeutic use; D029721:Drosophila Proteins; D004359:Drug Therapy, Combination / Q000379:methods / Q000706:statistics & numerical data; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D000077408:Modafinil; D009290:Narcolepsy / Q000175:diagnosis* / Q000188:drug therapy* / Q000453:epidemiology; D009419:Nerve Tissue Proteins; D009687:Nuclear Proteins; D050177:Overweight / Q000453:epidemiology; D017286:Polysomnography / Q000379:methods / Q000706:statistics & numerical data; D011627:Puberty; D012189:Retrospective Studies; D012978:Sodium Oxybate / Q000627:therapeutic use; D013290:Streptococcal Infections / Q000453:epidemiology; D011795:Surveys and Questionnaires; D014157:Transcription Factors; D014481:United States / Q000453:epidemiology; D000069470:Venlafaxine Hydrochloride; D015430:Weight Gain
chemical_list: D000759:Adjuvants, Anesthesia; D018687:Antidepressive Agents, Second-Generation; D001559:Benzhydryl Compounds; D000697:Central Nervous System Stimulants; D003511:Cyclohexanols; D029721:Drosophila Proteins; D009419:Nerve Tissue Proteins; D009687:Nuclear Proteins; D014157:Transcription Factors; C073099:pros protein, Drosophila; D000069470:Venlafaxine Hydrochloride; D012978:Sodium Oxybate; D000077408:Modafinil