Metabolomic Analysis of the Effects of Leptin Replacement Therapy in Patients with Lipodystrophy

Context and Objective Leptin treatment has dramatic clinical effects on glucose and lipid metabolism in leptin-deficient patients with lipodystrophy. Further elucidation of metabolic effects of exogenous leptin therapy will shed light on understanding leptin physiology in humans. Our objective was to utilize metabolomic profiling to examine the changes associated with administration of short-term metreleptin therapy in patients with lipodystrophy. Study Design We conducted a pre-post treatment study in 19 patients (75% female) with varying forms of lipodystrophy (congenital generalized lipodystrophy, n=10; acquired generalized lipodystrophy, n=1; familial partial lipodystrophy, n=8) who received daily subcutaneous metreleptin injections for a period of 16 - 23 weeks. A 3-hour oral glucose tolerance test and body composition measurements were conducted before and after the treatment period, and fasting blood samples were used for metabolomic profiling. The study outcome aimed at measur...研究背景与研究目的 瘦素（leptin）治疗对脂肪营养不良综合征（lipodystrophy）合并瘦素缺乏患者的糖脂代谢具有显著临床效应。进一步阐明外源性瘦素治疗的代谢效应，将有助于深入理解人体瘦素的生理功能。本研究旨在通过代谢组学分析（metabolomic profiling），探究脂肪营养不良综合征患者接受短期美曲普汀（metreleptin）治疗后的相关代谢变化。 研究设计 本研究针对19例不同类型脂肪营养不良综合征患者开展治疗前后对照研究，其中女性占比75%；患者分型包括先天性全身性脂肪营养不良综合征10例、获得性全身性脂肪营养不良综合征1例、家族性部分性脂肪营养不良综合征8例，所有患者均接受每日皮下注射美曲普汀治疗，疗程为16至23周。于治疗前后分别开展3小时口服葡萄糖耐量试验与身体成分测定，并采集空腹血液样本用于代谢组学分析。本研究的结局指标旨在量化...