ESPE Abstracts (2016) 86 RFC3.8

Anthropometric and Endocrine Features in Children and Adolescents with Type 1 Narcolepsy

Virginia Ponziania, Monia Gennaria, Fabio Pizzac,d, Antonio Balsamob, Filippo Bernardib & Giuseppe Plazzic,d


aS. Orsola-Malpighi University Hospital, Pediatric Unit, Bologna, Italy; bS.Orsola Malpighi University Hospital - Department of Medical and Surgical Sciences, University of Bologna, Pediatric Unit, Bologna, Italy; cDepartment of Biomedical and NeuroMotor Sciences, University of Bologna, Bologna, Italy; dIRCCS Institute of Neurological Sciences, Ospedale Bellaria, Bologna, Italy


Background: Childhood NT1 has been associated with endocrine disorders like obesity and precocious puberty. These comorbidities may challenge the diagnosis, require tailored treatments and call for a multidisciplinary approach.

Objective and hypotheses: To evaluate the impact of type 1 narcolepsy (NT1) on anthropometric and endocrine features in childhood/adolescence, focusing on weight, pubertal development and growth, and their follow up in treated and untreated patients.

Method: At diagnosis we collected anthropometric (height, weight, body mass index (BMI) z-scores), pubertal, metabolic and endocrine data from 72 NT1 patients and available premorbid anthropometric pediatric clinical records. Re-evaluation at one-year follow-up was contrasted 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 growth alterations at the diagnosis. Weight gain started closely after NT1 onset. At follow-up sodium oxybate was associated with a significant BMI z-score reduction (−1.29±0.30, P<0.0005) also after correcting for baseline age, sex, sleepiness, and BMI. Treatment did not influence growth.

Conclusion: NT1 in children/adolescents induced a rapid weight gain up to overweight/obesity and precocious puberty without affecting growth. In our study we found that Sodium oxybate reduces weight excess in NT1 overweight/obese patients without compromising their growth.

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