Francois Mifsud, Sarah Chalopin, Emilie Guillon, Pauline Faucher, Jean Muller, Johanne Le Bihan, Karine Clément, Christine Poitou
{"title":"赛特美拉肽治疗成人单基因或综合征性肥胖的实际疗效和安全性：一项前瞻性队列研究。","authors":"Francois Mifsud, Sarah Chalopin, Emilie Guillon, Pauline Faucher, Jean Muller, Johanne Le Bihan, Karine Clément, Christine Poitou","doi":"10.1002/oby.70002","DOIUrl":null,"url":null,"abstract":"<div>\n \n \n <section>\n \n <h3> Objective</h3>\n \n <p>The melanocortin-4 receptor agonist setmelanotide has demonstrated effectiveness in phase 3 clinical trials for patients with monogenic obesity caused by biallelic variants in the leptin receptor (LEPR) and pro-opiomelanocortin (POMC), as well as for individuals with Bardet–Biedl syndrome (BBS). However, real-world evidence remains limited. This study evaluates the long-term effectiveness and safety of setmelanotide in patients who received treatment under a pre-marketing early-access authorization.</p>\n </section>\n \n <section>\n \n <h3> Methods</h3>\n \n <p>This ongoing prospective monocentric cohort includes 17 patients with obesity due to BBS (<i>n</i> = 11) or biallelic variants in <i>LEPR</i> (<i>n</i> = 4) or <i>POMC</i> (<i>n</i> = 2) who either started setmelanotide in routine care between 2022 and 2024 or continued therapy after participating in the RM-493-022 clinical trial. The average follow-up time was 14.4 months.</p>\n </section>\n \n <section>\n \n <h3> Results</h3>\n \n <p>Patients experienced a clinically significant weight reduction of 20% from their highest pre-treatment weight within the first year. Those previously treated in a clinical trial maintained their weight loss over time. Additionally, eating behavior improved, with significant reductions in hunger (−62%), food craving (−41%), and external eating evaluated by DEBQ. The overall safety profile was consistent with phase 3 trials data, without any new safety signals.</p>\n </section>\n \n <section>\n \n <h3> Conclusions</h3>\n \n <p>These findings confirm the drug's long-term clinical benefit and safety profile in a routine-care setting.</p>\n </section>\n </div>","PeriodicalId":215,"journal":{"name":"Obesity","volume":"33 10","pages":"1865-1872"},"PeriodicalIF":4.7000,"publicationDate":"2025-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Real-World Efficacy and Safety of Setmelanotide in Adults With Monogenic or Syndromic Obesity: A Prospective Cohort Study\",\"authors\":\"Francois Mifsud, Sarah Chalopin, Emilie Guillon, Pauline Faucher, Jean Muller, Johanne Le Bihan, Karine Clément, Christine Poitou\",\"doi\":\"10.1002/oby.70002\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div>\\n \\n \\n <section>\\n \\n <h3> Objective</h3>\\n \\n <p>The melanocortin-4 receptor agonist setmelanotide has demonstrated effectiveness in phase 3 clinical trials for patients with monogenic obesity caused by biallelic variants in the leptin receptor (LEPR) and pro-opiomelanocortin (POMC), as well as for individuals with Bardet–Biedl syndrome (BBS). However, real-world evidence remains limited. This study evaluates the long-term effectiveness and safety of setmelanotide in patients who received treatment under a pre-marketing early-access authorization.</p>\\n </section>\\n \\n <section>\\n \\n <h3> Methods</h3>\\n \\n <p>This ongoing prospective monocentric cohort includes 17 patients with obesity due to BBS (<i>n</i> = 11) or biallelic variants in <i>LEPR</i> (<i>n</i> = 4) or <i>POMC</i> (<i>n</i> = 2) who either started setmelanotide in routine care between 2022 and 2024 or continued therapy after participating in the RM-493-022 clinical trial. The average follow-up time was 14.4 months.</p>\\n </section>\\n \\n <section>\\n \\n <h3> Results</h3>\\n \\n <p>Patients experienced a clinically significant weight reduction of 20% from their highest pre-treatment weight within the first year. Those previously treated in a clinical trial maintained their weight loss over time. 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Real-World Efficacy and Safety of Setmelanotide in Adults With Monogenic or Syndromic Obesity: A Prospective Cohort Study
Objective
The melanocortin-4 receptor agonist setmelanotide has demonstrated effectiveness in phase 3 clinical trials for patients with monogenic obesity caused by biallelic variants in the leptin receptor (LEPR) and pro-opiomelanocortin (POMC), as well as for individuals with Bardet–Biedl syndrome (BBS). However, real-world evidence remains limited. This study evaluates the long-term effectiveness and safety of setmelanotide in patients who received treatment under a pre-marketing early-access authorization.
Methods
This ongoing prospective monocentric cohort includes 17 patients with obesity due to BBS (n = 11) or biallelic variants in LEPR (n = 4) or POMC (n = 2) who either started setmelanotide in routine care between 2022 and 2024 or continued therapy after participating in the RM-493-022 clinical trial. The average follow-up time was 14.4 months.
Results
Patients experienced a clinically significant weight reduction of 20% from their highest pre-treatment weight within the first year. Those previously treated in a clinical trial maintained their weight loss over time. Additionally, eating behavior improved, with significant reductions in hunger (−62%), food craving (−41%), and external eating evaluated by DEBQ. The overall safety profile was consistent with phase 3 trials data, without any new safety signals.
Conclusions
These findings confirm the drug's long-term clinical benefit and safety profile in a routine-care setting.
期刊介绍：
Obesity is the official journal of The Obesity Society and is the premier source of information for increasing knowledge, fostering translational research from basic to population science, and promoting better treatment for people with obesity. Obesity publishes important peer-reviewed research and cutting-edge reviews, commentaries, and public health and medical developments.
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