Assessment of treatment effects on cataplexy in peaditaric NT1 throught standadized video recording procedure

Objectives: Cataplexy is the pathognomonic symptom of type 1 narcolepsy (NT1), however current criteria do not require its objective assessment. The aim of this study is to extend the reliability of our standardized video recording procedure for cataplexy assessment to measure response to treatment. Methods: Twenty NT1 children and adolescent were evaluated at baseline and after at least three months of stable treatment with Sodium Oxybate or Pitolisant. Children underwent a standardized video recording procedure consisting of watching funny movies, with the aim of evoking cataplexy. Immediately after the test, an expert sleep technician reviewed the whole video recording and systematically evaluated the occurrence (on a 4-point Likert scale) of head drops, trunk falls, ptosis, and mouth opening separately in the baseline and the under emotional stimulation conditions. Tests results in the two conditions were compared within subjects. Results: Patients (16 males and 4 females) had NT1 onset at age 9.5 ± 3.06 and were studied at a mean age of 11.7 ± 2.6 at baseline and 13.4 ± 2.5 at follow-up. Our standardized video recording procedure objectively documented a significant reduction of cataplexy features (head drops, ptosis, and mouth openings) at follow-up compared to baseline assessment. Conclusions: Our results show that a standardized video recording procedure is able to successfully track of anticataplectic therapies effects in paediatric NT1 patients. This simple and quickly use procedure have the potential to be a reliable tool to objectively assess cataplexy severity across the establishment of pharmacological therapies. Further studies are needed to extend the population and analyze the effect of different treatment strategies alone and in combination.