Background: Prader-Willi syndrome (PWS) is a genetic disorder associated with developmental delay, obesity, and obsessive behavior related to food consumption. Treatment options for weight control in those patients is limited and there are controversies for a surgical approach. Saxenda® (liraglutide) injection 3 mg is indicated as an adjunct to a reduced-calorie diet and increased physical activity for chronic weight management in adult patients with obesity or overweight in the presence of at least one weight-related comorbid condition.
Objective: The objective of this study is to determine the effect of a 3-month trial of liraglutide on appetite, lipid profile, and weight in youth with PWS.
Methods: Three overweight and obese females with PWS (19-22 years) including maternal uniparental disomy (mUPD) were recruited for a 3-month open-label, non-randomized, longitudinal study conducted at Pusan National University Children's Hospital. Liraglutide was given using standard diabetes dosing without dietary modifications. Body Weight, body mass index (BMI), lipid profile, and appetite were measured over 3 months. Appetite scores was measured using the Dutch Eating Behavior Questionnaire for assessment of restrained, emotional and external eating behavior (DEBQ).
Results: Appetite scores was not significantly decreased from baseline and after 3 months of treatment (26.4±6.8 and 26.1±5.5; P=0.84). Hemoglobin A1c was not decreased after 3 months of treatment (6.63±1.78 and 6.66±1.81; P=0.98), but also weight, BMI z-score, and LDL-cholesterol did not (2.75±2.03 and 2.81±2.17; P=0.97, 3.79±1.44 and 3.82±1.59; P=0.97, 96.0±52.0 and 103.6±24.8; P=0.82, respectively). There was no significant change in waist circumference.
Conclusions: This is the first longitudinal investigation of the effects of liraglutide in subjects with mUPD PWS. It was not effective in decreasing appetite, without change in weight or BMI in the short term. Larger, controlled, longer-term trials in patients with PWS are required to confirm the efficacy and safety of liraglutide and to evaluate whether its use might induce weight loss when given in conjunction with behavioral modification.
19 - 21 Sep 2019
European Society for Paediatric Endocrinology