Back to Search
Start Over
Effect of Leptin Therapy on Survival in Generalized and Partial Lipodystrophy: A Matched Cohort Analysis.
- Source :
- Journal of Clinical Endocrinology & Metabolism; Aug2021, Vol. 106 Issue 8, pe2953-e2967, 15p
- Publication Year :
- 2021
-
Abstract
- <bold>Context: </bold>Data quantifying the impact of metreleptin therapy on survival in non-human immunodeficiency virus (HIV)-related generalized lipodystrophy (GL) and partial lipodystrophy (PL) are unavailable.<bold>Objective: </bold>This study aimed to estimate the treatment effect of metreleptin on survival in patients with GL and PL.<bold>Design/setting/patients: </bold>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.<bold>Outcome Measures: </bold>This study assessed time-to-mortality and risk of mortality.<bold>Results: </bold>The 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.<bold>Conclusions: </bold>Metreleptin 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. [ABSTRACT FROM AUTHOR]
Details
- Language :
- English
- ISSN :
- 0021972X
- Volume :
- 106
- Issue :
- 8
- Database :
- Complementary Index
- Journal :
- Journal of Clinical Endocrinology & Metabolism
- Publication Type :
- Academic Journal
- Accession number :
- 151494536
- Full Text :
- https://doi.org/10.1210/clinem/dgab216