Growing Up with Type 1 narcolepsy: Its anthropometric and endocrine features

Study Objectives: To evaluate the effect of type 1 narcolepsy (NT1) on anthropometric and endocrine features in childhood/adolescence, focusing on patterns and correlates of weight, pubertal development, and growth in treated and untreated patients. Methods: We collected anthropometric (height, weight, body mass index (BMI) z-scores), pubertal, metabolic, and endocrine data from 72 NT1 patients at diagnosis and all available premorbid anthropometric parameters of patients from their pediatric files (n = 30). New measurements at 1-y reassessment in patients undergoing different treatments were compared with baseline data. Results: We detected a high prevalence of overweight (29.2%), obesity (25%), metabolic syndrome (18.8%), and precocious puberty (16.1%), but no signs of linear growth alterations at diagnosis. According to anthropometric records, weight gain started soon after NT1 onset. At 1-y follow-up reassessment, sodium oxybate treatment was associated with a significant BMI z-score reduction (-1.29 \ub1 0.30, p < 0.0005) after adjusting for baseline age, sex, sleepiness, and BMI. Conclusions: NT1 onset in children/adolescents is associated with rapid weight gain up to overweight/obesity and precocious puberty without affecting growth. In our study, sodium oxybate treatment resulted in a significant weight reduction in NT1 overweight/obese patients at 1-y follow-up

BMI changes in paediatric type 1 narcolepsy under sodium oxybate treatment

Paediatric Type 1 Narcolepsy (NT1) is often associated with overweight and obesity. Sodium oxybate (SO), approved for the treatment of narcolepsy with cataplexy from the age of 7 years old in US, has been associated with weight loss, although longitudinal paediatric studies are lacking. We report a retrospective cohort of 129 consecutive patients with a 4 years follow-up, to analyse the impact of different pharmacological treatments on BMI z-score. At baseline the prevalence of obesity and overweight was 26.4 % (34/129) and 29.5% (38/129), respectively. Patients were divided in 3 groups: children treated with SO alone (group 1), with SO-combined therapy (group 2), and without SO (group 3). At the end of the first year of follow-up, group 1 and group 2 showed a significant BMI z-score reduction compared to baseline: from 1.2±1.1 to 0.4±1.4 for group 1 (p < 0.001), and from 1.4±1.1 to 1±1.3 for group 2 (p = 0.002), independently from baseline clinical features. In the second year only group 2 experienced a further and significant BMI z-score decrease (from 1.0±1.2 to 0.6 ± 1.2, p = 0.037). No further significant BMI z-score changes were observed in SO treated patients in the following years. Instead, children treated without SO developed a significant weight increase between the second and third year of therapy (BMI z-score from 0.3±0.9 to 0.5±0.9). In conclusion, SO treatment in paediatric NT1 is associated with a favourable weight reduction in the first year of treatment