ArticleThe Journal of clinical endocrinology and metabolism2021
Effect of Leptin Therapy on Survival in Generalized and Partial Lipodystrophy: A Matched Cohort Analysis.
Article in The Journal of clinical endocrinology and metabolism, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
What it found
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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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
18 citing papers in PubMed, 1 synthesis or guideline pooled it, 26 citations in OpenAlex.
- Clinical Spectrum ofCells · 2023Pooled it
- Clinical Guidance for Lipodystrophy Syndromes: From Diagnosis and Work-Up to Treatment.Current diabetes reports · 2025Review
- Liraglutide use in pediatric type 2 familial partial lipodystrophy caused by LMNA mutation: a case report.BMC pediatrics · 2025Article
- Brazilian expert consensus on the diagnosis, classification, screening for complications and treatment of familial partial lipodystrophy.Diabetology & metabolic syndrome · 2025Article
- Metabolic Improvements With Tirzepatide in Lipodystrophy: A Novel Option?Diabetes care · 2025Observational
- Adipokines: masterminds of metabolic inflammation.Nature reviews. Immunology · 2025Review
- Effects of Metreleptin in Patients With Generalized Lipodystrophy Before vs After the Onset of Severe Metabolic Disease.The Journal of clinical endocrinology and metabolism · 2025Article
- The clinical approach to child and adolescent patients with lipodystrophy: a series of international case discussions.Frontiers in endocrinology · 2025Review
- Navigating Lipodystrophy: Insights from Laminopathies and Beyond.International journal of molecular sciences · 2024Review
- Efficacy and Safety of Glucagon-Like Peptide 1 Agonists in a Retrospective Study of Patients With Familial Partial Lipodystrophy.Diabetes care · 2024Article
- GLP-1 receptor agonist improves metabolic disease in a pre-clinical model of lipodystrophy.Frontiers in endocrinology · 2024Article
- Metreleptin Robustly Increases Resting-state Brain Connectivity in Treatment-naïve Female Patients With Lipodystrophy.Journal of the Endocrine Society · 2023Article
- Is it possible to achieve an acceptable disease control by dietary therapy alone in Berardinelli Seip type 1? Experience from a case report.Frontiers in endocrinology · 2023Article
- Natural history and comorbidities of generalised and partial lipodystrophy syndromes in Spain.Frontiers in endocrinology · 2023Article
- Leptin treatment has vasculo-protective effects in lipodystrophic mice.Proceedings of the National Academy of Sciences of the United States of America · 2022Article
- Approach to the Patient With Lipodystrophy.The Journal of clinical endocrinology and metabolism · 2022Article
- Leptin as a key regulator of the adipose organ.Reviews in endocrine & metabolic disorders · 2022Review
- Treatment Options for Lipodystrophy in Children.Frontiers in endocrinology · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 6 institutions in 3 countries.
Funding
Abstract
contextData quantifying the impact of metreleptin therapy on survival in non-human immunodeficiency virus (HIV)-related generalized lipodystrophy (GL) and partial lipodystrophy (PL) are unavailable.
objectiveThis study aimed to estimate the treatment effect of metreleptin on survival in patients with GL and PL. DESIGN/SETTING/PATIENTS: Demographic and clinical characteristics were used to match metreleptin-treated and metreleptin-naïve patients with GL and PL. Differences in mortality risk were estimated between matched cohorts of metreleptin-treated and metreleptin-naïve patient cohorts using Cox proportional hazard models. Sensitivity analyses assessed the impact of study assumptions and the robustness of results. OUTCOME MEASURES: This study assessed time-to-mortality and risk of mortality.
resultsThe analysis evaluated 103 metreleptin-naïve patients with characteristics matched to 103 metreleptin-treated patients at treatment initiation. Even after matching, some metabolic and organ abnormalities were more prevalent in the metreleptin-treated cohort due to bias toward treating more severely affected patients. A Cox proportional hazards model associated metreleptin therapy with an estimated 65% decrease in mortality risk (hazard ratio [HR] 0.348, 95% confidence interval (CI): 0.134-0.900; P = 0.029) even though the actual number of events were relatively small. Results were robust across a broad range of alternate methodological assumptions. Kaplan-Meier estimates of time-to-mortality for the metreleptin-treated and the matched metreleptin-naïve cohorts were comparable.
conclusionsMetreleptin therapy was associated with a reduction in mortality risk in patients with lipodystrophy syndromes despite greater disease severity in treated patients, supporting the view that metreleptin can have a positive disease-modifying impact. Confirmatory studies in additional real-world and clinical datasets are warranted.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.