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.