ESPE Abstracts (2014) 82 P-D-2-3-493

Role of Metformin in the Treatment of Hypothalamic Obesity

Arundoss Gangadharana, Mohammed Didia, Barry Pizerb, Lisa Howellb, James Haydenb, Conor Malluccic, Benedetta Pettorinic & Joanne C Blaira


aDepartment of Paediatric Endocrinology, Alder Hey Children’s Hospital, Liverpool, UK; bDepartment of Paediatric Oncology, Alder Hey Children’s Hospital, Liverpool, UK; cDepartment of Paediatric Neurosurgery, Alder Hey Children’s Hospital, Liverpool, UK


Background: Disruption of homeostatic functioning of the hypothalamic centres results in hyperphagia, autonomic imbalance, reduction of energy expenditure, and hyperinsulinemia. A syndrome of rapid, unrelenting weight gain is often observed in patients with structural lesions of the hypothalamus. Hypothalamic obesity syndrome (HOS) is often refractory to standard dietary and lifestyle interventions. It has been reported that metformin induces anorectic effects via an increase in the central sensitivity to leptin. This provides a rationale for novel therapeutic approaches associating leptin and metformin in the treatment of HOS.

Objective: To describe rate of increase in BMI SDS before and during treatment with metformin.

Method: A retrospective study of the rate of BMI gain in patients before and during metformin therapy was undertaken.

Results: Data from five patients (5F) mean age 10.2 years (5.7–13.6) at diagnosis of hypothalamic lesions (hypothalamic hamartoma (n=2), craniopharyngioma (n=2), hypothalamic pilomyxoid astrocytoma+NF1 (n=1)) are reported. Patients were treated with surgery (n=4), adjuvant proton radiotherapy (n=1) and primary fractionated radiotherapy (n=1). Metformin was introduced at a mean age of 13.1 (10.8–15.8) years when mean gain in BMI SDS was 1.3/year (0.1–3.48). After 22 (4–56) months of treatment, mean gain in BMI SDS was 0.0/year (−0.12 to +0.27). Treatment with metformin was well tolerated.

Conclusion: Although metformin is only licensed for treatment of type-2 diabetes, there has been a steady increase of its use in the management of obesity, as an adjuvant to diet and lifestyle measures. We believe metformin had a positive effect on the management of HOS in these five patients and that a randomised control trail should now be considered to rigorously examine its role in the management of HOS.

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