Tolerance and efficacy of sodium oxybate in childhood narcolepsy with cataplexy: A retrospective study

Michel Lecendreux, Francesca Poli, Delphine Oudiette, Fatima Benazzouz, Claire E H M Donjacour, Christian Franceschini, Elena Finotti, Fabio Pizza, Oliviero Bruni, Giuseppe Plazzi

Research output: Contribution to journalArticlepeer-review


Narcolepsy with cataplexy is a sleep disorder characterized by excessive daytime sleepiness, irresistible sleep episodes, and sudden loss of muscle tone (cataplexy) mostly triggered by emotions. Narcolepsy with cataplexy is a disabling lifelong disorder frequently arising during childhood. Pediatric narcolepsy often results in severe learning and social impairment. Improving awareness about this condition increases early diagnosis and may allow patients to rapidly access adequate treatments, including pharmacotherapy and/or non-medication-based approaches. Even though children currently undergo pharmacotherapy, data about safety and efficacy in the pediatric population are scarce. Lacking international guidelines as well as drugs registered for childhood narcolepsy with cataplexy, physicians have no other alternative but to prescribe in an off-label manner medications identical to those recommended for adults. We retrospectively evaluated 27 children ranging from 6 to 16 years old, suffering from narcolepsy with cataplexy, who had been treated with off-label sodium oxybate and had been followed in a clinical setting. Throughout a semi-structured interview, we documented the good efficacy and tolerability of sodium oxybate in the majority of the patients. This study constitutes a preliminary step towards a further randomized controlled trial in childhood narcolepsy with cataplexy.

Original languageEnglish
Pages (from-to)709-711
Number of pages3
Issue number5
Publication statusPublished - May 1 2012


  • Childhood
  • GHB
  • Hypocretin
  • Narcolepsy with cataplexy
  • Sleepiness
  • Sodium oxybate
  • Treatment

ASJC Scopus subject areas

  • Physiology (medical)
  • Clinical Neurology


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