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Effect of leptin therapy on survival in generalized and partial lipodystrophy: A matched cohort analysis

Journal of Clinical Endocrinology and Metabolism Apr 07, 2021

Cook K, Ali O, Akinci B, et al. - Researchers conducted the study for estimating the treatment effect of metreleptin on survival in patients with generalized lipodystrophy (GL) and partial lipodystrophy (PL). They used demographic and clinical features to match metreleptin-treated and metreleptin-naïve patients with GL and PL. The analysis assessed 103 metreleptin-naïve patients with characteristics matched to 103 metreleptin-treated patients at treatment initiation. Despite greater disease severity in treated patients, metreleptin therapy was associated with a lower mortality risk in patients with lipodystrophy syndromes, supporting the idea that metreleptin may have a beneficial disease-modifying effect. Confirmatory research in more real-world and clinical datasets is needed.

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