Weight loss with glucagon‐like peptide‐1 receptor agonists in <scp>Bardet‐Biedl</scp> syndrome

Ganawa, Shawg; Santhosh, Smrithi Hema; Parry, Lucy J.; Syed, Akheel A.

doi:10.1111/cob.12546

Cited by 10 publications

(8 citation statements)

References 16 publications

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“…It was necessary to maintain the drug in therapy, because it was observed a weight regain after dose reduction. 141 Pre-clinical studies demonstrated that the use of roscovitine and rapamycin was able to rescue renal cysts in zebrafish models of BBS. 142 A novel target for therapy in BBS is glycosphingolipid metabolism, in experimental models of disease.…”

Section: Future Perspectivesmentioning

confidence: 99%

Bardet-Biedl Syndrome: Current Perspectives and Clinical Outlook

Melluso¹,

Secondulfo²,

Capolongo³

et al. 2023

TCRM

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The Bardet Biedl syndrome (BBS) is a rare inherited disorder considered a model of non-motile ciliopathy. It is in fact caused by mutations of genes encoding for proteins mainly localized to the base of the cilium. Clinical features of BBS patients are widely shared with patients suffering from other ciliopathies, especially autosomal recessive syndromic disorders; moreover, mutations in cilia-related genes can cause different clinical ciliopathy entities. Besides the best-known clinical features, as retinal degeneration, learning disabilities, polydactyly, obesity and renal defects, several additional clinical signs have been reported in BBS, expanding our understanding of the complexity of its clinical spectrum. The present review aims to describe the current knowledge of BBS i) pathophysiology, ii) clinical manifestations, highlighting both the most common and the less described features, iii) current and future perspective for treatment.

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Section: Future Perspectivesmentioning

confidence: 99%

Bardet-Biedl Syndrome: Current Perspectives and Clinical Outlook

Melluso¹,

Secondulfo²,

Capolongo³

et al. 2023

TCRM

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“…61 There is one case report of successful weight loss with GLP-1RA therapy in a 28-year-old female with BBS and type 2 diabetes. 62 In our clinical experience, GLP-1RAs are not effective at appetite suppression in this population.…”

Section: Management Of Obesitymentioning

confidence: 81%

Bardet‐Biedl syndrome: A clinical overview focusing on diagnosis, outcomes and best‐practice management

Shoemaker

2024

Diabetes Obesity Metabolism

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Bardet‐Biedl syndrome (BBS) is a genetic disorder characterized by early‐onset obesity, polydactyly, genital and kidney anomalies, developmental delay and vision loss due to rod‐cone dystrophy. BBS is an autosomal recessive disorder with >20 implicated genes. The genotype–phenotype relationship in BBS is not clear, and there may be additional modifying factors. The underlying mechanism is dysfunction of primary cilia. In BBS, receptor trafficking in and out of the cilia is compromised, affecting multiple organ systems. Along with early‐onset obesity, hyperphagia is a prominent symptom and contributes significantly to clinical morbidity and caregiver burden. While there is no cure for BBS, setmelanotide is a new pharmacotherapy approved for treatment of obesity in BBS. The differential diagnosis for BBS includes other ciliopathies, such as Alstrom syndrome, and other genetic obesity syndromes, such as Prader‐Willi syndrome. Careful clinical history and genetic testing can help determine the diagnosis and a multidisciplinary team is necessary to guide clinical management.

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“…Glucagon-like peptide-1 receptor agonists (GLP-1 RA) have also been considered as a weight management treatment in hyperphagic obesity syndromes [26]. Thus far, these drugs have limited evidence of effectiveness to treat hyperphagia or obesity in people with BBS [27 ▪ ]. Given their role in the treatment of obesity and hyperglycaemia (including type 2 diabetes mellitus) in other populations, investigation of the effectiveness of GLP-1 RAs merit further investigation.…”

Section: Discussionmentioning

confidence: 99%

Diabetes mellitus in Bardet Biedl syndrome

Pomeroy

Offenwanger²,

Timmler

2022

Current Opinion in Endocrinology, Diabetes &Amp; Obesity

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Purpose of reviewBardet Biedl syndrome (BBS) is a rare disease characterized by obesity and hyperphagia. Despite the very high prevalence of paediatric and adult obesity in this population, the prevalence of diabetes mellitus is not well described.Recent findingsStudies in small and moderately large cohorts suggest a high prevalence of traditional risk factors for diabetes mellitus in people with BBS. People with BBS appear to have a high prevalence of insulin resistance and metabolic syndrome. Small cohort studies have identified high rates of sleep disordered breathing, including sleep apnoea syndrome. Recent research has characterized traditional behavioural risk factors such as sleep hygiene and physical inactivity in people with BBS. High rates of insufficient sleep and prolonged sedentary time suggest behavioural targets of interventions to treat or prevent diabetes mellitus. Hyperphagia, likely caused by defects in the hypothalamic melanocortin-4 receptor (MC4R) neuronal pathway, pose additional challenges to behavioural interventions to prevent diabetes mellitus.SummaryUnderstanding the prevalence of diabetes mellitus and other metabolic disorders in people with BBS and the impact of traditional risk factors on glucose regulation are important to developing effective treatments in this population.

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Weight loss with glucagon‐like peptide‐1 receptor agonists in Bardet‐Biedl syndrome

Cited by 10 publications

References 16 publications

Bardet-Biedl Syndrome: Current Perspectives and Clinical Outlook

Bardet-Biedl Syndrome: Current Perspectives and Clinical Outlook

Bardet‐Biedl syndrome: A clinical overview focusing on diagnosis, outcomes and best‐practice management

Diabetes mellitus in Bardet Biedl syndrome

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