Treatment with Glucagon-Like Peptide-1 Agonist Exendin-4 in a Patient with Hypothalamic Obesity Secondary to Intracranial Tumor

Simmons, Jill H.
Shoemaker, Ashley H.
Roth, Christian L.

^aDivision of Endocrinology, Department of Pediatrics, Vanderbilt University Medical Center, Nashville, Tenn., and ^bSeattle Children's Research Institute, Seattle, Wash., USA

^*Christian L. Roth, MD, Division of Endocrinology, Seattle Children's Research Institute, 1900 Ninth Avenue, Seattle, WA 98101 (USA), Tel. +1 206 987 5428, E-Mail [email protected]

Received March 20, 2012; Accepted May 07, 2012; previously published online July 20, 2012

Hormone Research in Paediatrics 78(1):p 54-58, August 2012. | DOI: 10.1159/000339469

Abstract

Background/Aims:

Patients with hypothalamic tumors frequently experience severe obesity, and its treatment with diet, exercise, and/or pharmacologic treatment has had limited effect. Glucagon-like peptide-1 agonist exenatide (exendin-4), used for treatment of type 2 diabetes, causes persistent weight loss via signaling in the brainstem.

Methods:

We report the case of a 17-year-old patient with obesity resulting from a hypothalamic germ cell tumor. He was treated by chemoradiotherapy and exenatide at a dose of 5 μg subcutaneously twice daily.

Results:

Exenatide resulted in a 29-kg weight loss (BMI reduction from 37.1 to 29.1) after 2.5 years of treatment; significant weight gain occurred shortly after exenatide was discontinued.

Conclusion:

Exenatide resulted in considerable reduction of body weight in a patient with severe hypothalamic obesity. This novel observation requires follow-up clinical studies for establishing the effects of exenatide in patients with disrupted hypothalamic energy regulatory pathways.