ArticleJournal of the Endocrine Society2021
Effects of Metreleptin on Patient Outcomes and Quality of Life in Generalized and Partial Lipodystrophy.
Article in Journal of the Endocrine Society, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 2 of them syntheses that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
18 citing papers in PubMed, 2 syntheses or guidelines pooled it, 39 citations in OpenAlex.
- The Evolving Landscape of Discrete Choice Experiments in Health Economics: A Systematic Review.PharmacoEconomics · 2025Pooled it
- Prophylaxis forBMJ open · 2023Pooled it
- Quality of life outcomes in two phase 3 trials of setmelanotide in patients with obesity due to LEPR or POMC deficiency.Orphanet journal of rare diseases · 2022Trial
- Therapeutic Potential of Leptin in Neurodegenerative Disease.Biomedicines · 2025Review
- Parallel Pathways, Divergent Outcomes: Adipose Tissue-Neural Crosstalk in Depression and Obesity.Journal of clinical medicine · 2025Review
- Pathways to improving the awareness, diagnosis and management of lipodystrophy in Brazil: an expert panel discussion.Diabetology & metabolic syndrome · 2025Review
- Adipose Tissue-Derived Adipokines in Vascular Physiology and Pathophysiology: Insights and Implications.Comprehensive Physiology · 2025Review
- Escherichia coli in the production of biopharmaceuticals.Biotechnology and applied biochemistry · 2025Review
- The clinical approach to child and adolescent patients with lipodystrophy: a series of international case discussions.Frontiers in endocrinology · 2025Review
- Novel Leptin-Based Therapeutic Strategies to Limit Synaptic Dysfunction in Alzheimer's Disease.International journal of molecular sciences · 2024Review
- Impact of lipodystrophy on health-related quality of life: the QuaLip study.Orphanet journal of rare diseases · 2024Observational
- GLP-1 receptor agonist improves metabolic disease in a pre-clinical model of lipodystrophy.Frontiers in endocrinology · 2024Article
- Leptin treatment has vasculo-protective effects in lipodystrophic mice.Proceedings of the National Academy of Sciences of the United States of America · 2022Article
- Therapeutic indications and metabolic effects of metreleptin in patients with lipodystrophy syndromes: Real-life experience from a national reference network.Diabetes, obesity & metabolism · 2022Article
- Approach to the Patient With Lipodystrophy.The Journal of clinical endocrinology and metabolism · 2022Article
- Treatment Options for Lipodystrophy in Children.Frontiers in endocrinology · 2022Review
- Suggestive Evidence for an Antidepressant Effect of Metreleptin Treatment in Patients with Lipodystrophy.Obesity facts · 2022Observational
- Molecular and Cellular Bases of Lipodystrophy Syndromes.Frontiers in endocrinology · 2021Review
Corrections and comments
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Authors and funding
7 authors at 2 institutions in 1 country.
Funding
Abstract
Generalized and partial lipodystrophy are rare and complex diseases with progressive clinical and humanistic burdens stemming from selective absence of subcutaneous adipose tissue, which causes reduced energy storage capacity and a deficiency of adipokines such as leptin. Treatment options were limited before leptin replacement therapy (metreleptin) became available. This retrospective study evaluates both clinical and humanistic consequences of the disease and treatment. Chart data were abstracted from a cohort of metreleptin-treated patients with generalized and partial lipodystrophy (n = 112) treated at the US National Institutes of Health. To quantify the quality-of-life consequences of the lipodystrophy disease attributes recorded in chart data, a discrete choice experiment was completed in 6 countries (US, n = 250; EU, n = 750). Resulting utility decrements were used to estimate the quality-adjusted life-year consequences of changes in lipodystrophy attribute prevalence before and after metreleptin. In addition to metabolic impairment, patients with generalized and partial lipodystrophy experienced a range of lipodystrophy consequences, including liver abnormality (94%), hyperphagia (79%), impaired physical appearance (77%), kidney abnormality (63%), reproductive dysfunction (80% of females of reproductive age), and pancreatitis (39%). Improvement was observed in these attributes following initiation of metreleptin. Quality-adjusted life-year gains associated with 12 months of treatment with metreleptin were estimated at 0.313 for generalized and 0.117 for partial lipodystrophy, reducing the gap in quality of life between untreated lipodystrophy and perfect health by approximately 59% and 31%, respectively. This study demonstrates that metreleptin is associated with meaningful clinical and quality-of-life improvements.
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Registered trials
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