TY - JOUR
T1 - In-field assessment of sodium oxybate effect in pediatric type 1 narcolepsy
T2 - An actigraphic study
AU - Filardi, Marco
AU - Pizza, Fabio
AU - Antelmi, Elena
AU - Ferri, Raffaele
AU - Natale, Vincenzo
AU - Plazzi, Giuseppe
N1 - Ricercatori distaccato presso IRCCS a seguito Convenzione esclusiva con Università di Bologna (Pizza Fabio, Antelmi Elena, Plazzi Giuseppe).
La Dr.ssa E. Antelmi ha pubblicato anche lavori con affiliazione straniera che descrivono i risultati del progetto di ricerca svolto al “Sobell Department of Motor Neuroscience and Movement Disorders, University College London (UCL) Institute of Neurology, London” nel corso dei 3 anni della sua permanenza precedente presso l’Istituto (2014-2016).
PY - 2018/6/1
Y1 - 2018/6/1
N2 - 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 7 days of actigraphy during the school week. Information on sleepiness, narcolepsy symptoms, and anthropometric features were collected during the same week with questionnaires and semistructured 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.
AB - 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 7 days of actigraphy during the school week. Information on sleepiness, narcolepsy symptoms, and anthropometric features were collected during the same week with questionnaires and semistructured 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.
KW - Actigraphy
KW - Cataplexy
KW - Children
KW - Sodium oxybate
KW - Type 1 narcolepsy
KW - Weight loss
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U2 - 10.1093/sleep/zsy050
DO - 10.1093/sleep/zsy050
M3 - Article
AN - SCOPUS:85052210617
VL - 41
SP - 1
EP - 7
JO - Sleep
JF - Sleep
SN - 0161-8105
IS - 6
M1 - zsy050
ER -