Liraglutide therapy induces 22.2 kg weight loss in young man with Smith-Magenis syndrome and severe obesity.

Patients with Smith-Magenis syndrome, a rare genetic disorder, frequently experience intellectual disability, behavioral challenges, and severe obesity driven by hyperphagia. Conventional dietary and behavioral interventions often prove insufficient for sustainable weight management in this population, especially given the genetic predisposition to excessive hunger. This patient's rapid and persistent weight gain over five years, coupled with emerging signs of metabolic disease including prediabetes, dyslipidemia, and fatty liver disease, highlighted an urgent need for more effective therapeutic strategies. Glucagon-Like Peptide-1 Receptor Agonists (GLP-1RAs) like liraglutide are known to modulate appetite and promote satiety, offering a promising pharmacological avenue for managing such complex cases of obesity.

This case report details the treatment of a young man diagnosed with Smith-Magenis syndrome who presented with significant obesity and associated metabolic concerns. Following a history of accelerated weight gain over five years despite prior attempts at portion control and behavioral modification, the patient was initiated on daily liraglutide. The medication was gradually titrated to its maximum therapeutic dose over a period of five months. The intervention was conducted over a total duration of 11 months, during which the patient also continued with concurrent lifestyle and behavioral modifications. The primary outcome assessed was total body weight loss, alongside monitoring for medication tolerability and adverse gastrointestinal side effects.

Over the 11-month treatment period, the patient demonstrated significant and sustained weight reduction.

The patient achieved a total weight loss of 22.2 kg (49 lbs). This substantial weight loss was observed while the patient concurrently engaged in lifestyle and behavioral modifications, suggesting a synergistic effect with the pharmacological intervention. Importantly, the patient exhibited good tolerability to liraglutide, experiencing no severe gastrointestinal side effects throughout the titration and maintenance phases. This finding is particularly relevant given the potential for GLP-1RA side effects to impact adherence, especially in patients with complex needs. The successful weight management in this case, building upon only one other published report, underscores the potential utility of liraglutide in a challenging patient population with Smith-Magenis syndrome.