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Use of GLP-1 analog in a patient with Prader-Willi syndrome

Introducton Prader-Willi syndrome (PWS) is a rare genetic disease caused by deletions or imprinting defects in the region 15q11-q13 leading to hypothalamic-pituitary dysfunction, hyperphagia with excessive weight gain and behavioral disorders. Obesity is a hallmark of PWS, with consequently high incidence of impaired glucose tolerance and type 2 diabetes (T2D), particularly after puberty. Liraglutide, glucagon-like peptide 1 (GLP-1) analog is efficient in treatment of T2D, but also exhibits positive effect on body weight reduction and appetite suppression. Case Report We present a 17-year-old girl with genetically confirmed diagnosis of PWS (46, XX, ish del (15) (q11q13)) who developed TD2 at the age 15 years. She was never treated with growth hormone. Basal- bolus insulin therapy was introduced but despite good treatment adherence the optimal glycemic control was not achieved. She was also steadily gaining weight although efforts were made to limit caloric intake. At the age of 17 years her body weight was 140 kg (+4.66SD), height 157 cm (-1.63SD), BMI 56.8 kg/m2 (+4.78SD). Diabetes metabolic control was unsatisfactory (HbA1c 7.7%). The treatment with liraglutide (Victoza) was introduced at dose 1.2 mg/day with gradual reduction and discontinuation of insulin therapy. Two months later, she lost 3.1 kg and her HbA1c level was 6.1%. She also reported reduced appetite. For the following 1.5 years her metabolic control was excellent (HbA1c below 6.5%), but after reaching nadir of 135.2 kg (BMI 54.85 kg/m2) she started gaining weight again and currently weights 143.2 kg. No side effects of the treatment were noted during follow up. Conclusion GLP-1 analogs are effective in treatment of T2D in patients with PWS. However, the positive effect on the body weight, BMI and appetite regulation decreased over time. The literature data regarding use of GLP-1 analogs in patients with PWS are scarce, but they all report improved metabolic control of T2D. Nevertheless, there are conflicting results regarding body weight and BMI improvement. The treatment with GLP-1 analogs seems to be safe and effective option for therapy of T2D in PWS. Further studies are necessary to confirm preliminary results and establish the guidelines for use of GLP-1 agonists among PWS patients.

Prader-Willi syndrome ; obesity ; T2DM ; GLP-1 analog

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