In-Field Assessment of Sodium Oxybate Effect in Pediatric type 1 Narcolepsy: An Actigraphic Study

Marco Filardi, Fabio Pizza, Elena Antelmi, Raffaele Ferri, Vincenzo Natale, Giuseppe Plazzi

Research output: Contribution to journalArticlepeer-review


Study Objectives: Sodium oxybate (SXB) is a GABAergic agent widely used as off-label treatment in pediatric type 1 narcolepsy (NT1). Here we aimed at analyzing by wrist actigraphy the sleep/wake profile of NT1 children and adolescents in drug-naïve condition and after 1 year of SXB treatment. As secondary aim, we investigated changes on sleepiness, cataplexy and children's anthropometric profile after 1 year of SXB treatment.

Methods: Twenty-four drug-naïve NT1 children underwent seven days of actigraphy during the school week. Information on sleepiness, narcolepsy symptoms and anthropometric features were collected during the same week with questionnaires and semi-structured clinical interview. Children started SXB treatment and underwent a second evaluation encompassing actigraphy, clinical interview, questionnaires and anthropometric assessment after 1 year of stable treatment.

Results: Actigraphy effectively documented an improvement of nocturnal sleep quality and duration coupled with a reduction of diurnal total sleep time, nap frequency and duration at 1-year follow-up. Reduction of sleepiness, cataplexy frequency and severity and weight loss, mainly in obese and overweight NT1 children, were also observed at the 1-year follow-up.

Conclusions: Actigraphy objectively documented changes in nocturnal sleep quality and diurnal napping behavior after 1-year of SXB treatment, thus representing a valid approach to ecologically assess SXB treatment effect on NT1 children's sleep/wake profile. NT1 symptoms severity and children's anthropometric features also changed as expected. Actigraphy offers the possibility to longitudinally follow up children and has potential to become a key tool to tailor treatment in pediatric patients.

Original languageEnglish
Publication statusE-pub ahead of print - Mar 7 2018


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