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Management of Monogenic and Syndromic Obesity
Joan C Han1, Marcus C Rasmussen2, Alison R Forte2
1Division of Pediatric Endocrinology and Diabetes, Department of Pediatrics, Icahn School of Medicine at Mount Sinai, New York, NY, USA; Diabetes, Obesity, and Metabolism Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA; The Mindich Child Health and Development Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Gastroenterology Clinics of North America
|November 2, 2023
Summary
Lifestyle interventions are key for rare genetic obesity. Targeted therapies like metreleptin and setmelanotide can enhance treatments for leptin pathway disorders and related syndromes.
Area of Science:
- Medical genetics
- Endocrinology
- Metabolic disorders
Background:
- Obesity from rare genetic disorders requires tailored treatment approaches.
- Lifestyle interventions (nutrition, physical activity) are foundational.
- Specific genetic defects disrupt metabolic and hormonal pathways, leading to severe obesity.
Purpose of the Study:
- To review current and investigational therapeutic strategies for rare genetic obesity.
- To highlight the role of the leptin signaling pathway in these disorders.
- To discuss targeted therapies for monogenic and syndromic obesity conditions.
Main Methods:
- Review of existing literature on genetic obesity treatments.
- Analysis of therapeutic targets within the leptin pathway.
- Examination of novel treatments for specific genetic syndromes like Prader-Willi syndrome.
Main Results:
- Lifestyle interventions are effective but often insufficient alone for rare genetic obesity.
- Pharmacotherapies targeting the leptin pathway (metreleptin, setmelanotide) show promise.
- Synergistic effects observed when combining lifestyle changes with targeted therapies for leptin pathway defects.
Conclusions:
- Integrated treatment strategies combining lifestyle modifications and targeted pharmacotherapies are crucial for managing rare genetic obesity.
- Further research into investigational treatments, particularly for Prader-Willi syndrome, is warranted.
- Understanding genetic underpinnings and pathway defects enables personalized therapeutic development.
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