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Clinical Benefit of Setmelanotide in Patients With Alstrom Syndrome

Obesity (Silver Spring, Md.), 2022-11, Vol.30, p.142-142 [Peer Reviewed Journal]

Copyright Blackwell Publishing Ltd. Nov 2022 ;ISSN: 1930-7381 ;EISSN: 1930-739X

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  • Title:
    Clinical Benefit of Setmelanotide in Patients With Alstrom Syndrome
  • Author: Haqq, Andrea ; Chung, Wendy ; Hu, Shuang ; Cokkinias, Casey ; Malhotra, Sonali ; Dollfus, Hélěne ; Han, Joan
  • Subjects: Body mass index
  • Is Part Of: Obesity (Silver Spring, Md.), 2022-11, Vol.30, p.142-142
  • Description: Background: Alström syndrome is characterized by multiorgan dysfunction and obesity associated with impaired primary cilia function that may disrupt melanocortin-4 receptor (MC4R) pathway signaling. We evaluated the impact of ≥6 months of the MC4R agonist setmelanotide in patients with Alström syndrome. Methods: Patients with Alström syndrome aged ≥6 years were enrolled in Phase 2 and 3 trials of setmelanotide (NCT03013543, NCT03746522). Changes in weight, hunger, and quality of life (QOL) were evaluated. Clinical benefit was defined as meaningful improvement (ie, ≥5% body weight and/or body mass index [BMI] reduction for adults or ≥0.2 decrease in BMI Z score and/or ≥5-percentage point decrease in the percentage of the 95th percentile for BMI for pediatric patients, ≥25% decrease in hunger score, and/or increase in IWQOL-Lite score of >7.7 or increase in PedsQL score of >4.4). Adverse events (AEs) were assessed. Results: Eight patients received >6 months of treatment (range, 9.7-18.2 months). No patients aged >18 years achieved >5% body weight loss/BMI reduction; however, 1 of 2 adults (50%) had >25% decrease in hunger score. Five of 6 (83.3%) and 3 of 6 patients (50%) aged <18 years achieved >0.2- and >0.3-point decrease in BMI Z score, respectively. Three of 4 patients (75%) aged >12 to <18 years achieved >25% hunger score decrease. Two of 8 patients (25%) had improved QOL. Overall, 7 of 8 patients (87.5%) had improvement in >1 measure. Tolerability of setmelanotide was consistent with that in other rare MC4R disease populations. The most common AE was skin hyperpigmentation (10 of 12 patients [83.3%]). Three patients discontinued treatment because of AEs; 1 AE (facial edema) was related to setmelanotide. Conclusions: Most patients with Alström syndrome had positive response regarding clinical benefit of setmelanotide across weight, hunger, and QOL. Further research into the full extent of clinical benefit and specific characteristics of responders is needed.
  • Publisher: Silver Spring: Blackwell Publishing Ltd
  • Language: English
  • Identifier: ISSN: 1930-7381
    EISSN: 1930-739X
  • Source: ProQuest Central
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