Glucagon like pepetide-1 agonist in a patient with prader-willi syndrome: a Sri Lankan experience

Abstract

BackgroundPrader-Willi Syndrome (PWS) is a genetic disease characterized by morbid obesity and hyperphagia. Although the exact pathophysiological basis of obesity and hyperphagia remains unclear it is thought to be mainly due to hypothalamic dysfunction. Currently the options for medical therapy are limited and most are undergoing clinical trials. Lately glucagon like peptide agonist -1(GLP-1 agonist) has been tried out in this patient population due to its role in causing weight reduction, appetite suppression and glycemic control. We discuss our experience in using GLP-1 agonist on an adolescent with PWS and the challenges faced in managing these patients in a developing nation. Case PresentationA 16 year old Sri Lankan girl with PWS, presented with short stature, primary amenorrhea, morbid obesity (BMI =60) and hyperphagia. She had defaulted clinic follow up during child hood. Investigations revealed hypogonadotrophic hypogonadism and growth hormone deficiency. Cortisol, prolactin and thyroid hormonal levels were normal as was the imaging of the pituitary gland. Glycemic assessment revealed pre diabetes with HbA1c level of 6.2% and fasting blood glucose 120 mg/dl. She was commenced on liraglutide monotherapy which resulted in remarkable control of hyperphagia. She lost 3 kilograms during a period of three months and HbA1c dropped from 6.2% to 5.0%. However due to the high cost of the drug, her caregiver was compelled to discontinue it after 3 months of treatment. She was commenced on replacement of growth hormone, estradiol and metformin. However, hyperphagia recurred and she regained weight. Attempts to refer the patient for behavioral therapy were declined by the patient and her caregiver. ConclusionGLP-1 agonists seem to be a promising drug for hyperphagia and morbid obesity in patients with PWS and warrants further study to demonstrate its efficacy and safety in the long term. Inaccessibility to modern and expensive drugs, social stigma in seeking psychiatric and behavioral therapies, lack of support groups and organized institutional care for such patients are some of the obstacles in managing a rare and challenging disease like PWS in developing countries where the ever rising communicable and non communicable diseases drain the bulk of the health budget.

Keywords

• thalassaemia, iorn overload, delayed adrenache, delayed puburache