TY - JOUR
T1 - Tolerance and efficacy of sodium oxybate in childhood narcolepsy with cataplexy
T2 - A retrospective study
AU - Lecendreux, Michel
AU - Poli, Francesca
AU - Oudiette, Delphine
AU - Benazzouz, Fatima
AU - Donjacour, Claire E H M
AU - Franceschini, Christian
AU - Finotti, Elena
AU - Pizza, Fabio
AU - Bruni, Oliviero
AU - Plazzi, Giuseppe
PY - 2012/5/1
Y1 - 2012/5/1
N2 - 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.
AB - 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.
KW - Childhood
KW - GHB
KW - Hypocretin
KW - Narcolepsy with cataplexy
KW - Sleepiness
KW - Sodium oxybate
KW - Treatment
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U2 - 10.5665/sleep.1836
DO - 10.5665/sleep.1836
M3 - Article
C2 - 22547897
AN - SCOPUS:84860609389
VL - 35
SP - 709
EP - 711
JO - Sleep
JF - Sleep
SN - 0161-8105
IS - 5
ER -