Brehm Center for Diabetes Research, Division of Metabolism, Endocrinology & Diabetes, Department of Internal Medicine, University of Michigan, 1000 Wall Street, Room 5313, Ann Arbor, MI, 48105, USA.
Division of Endocrinology, Department of Internal Medicine, Dokuz Eylul University, Izmir, Turkey.
Curr Diab Rep. 2018 Oct 29;18(12):139. doi: 10.1007/s11892-018-1100-7.
The purpose of this review is to summarize the therapeutic approach for lipodystrophy syndromes with conventional treatment options and metreleptin therapy in detail and to point out the current investigational treatments in development.
The observation of leptin deficiency in patients with lipodystrophy and the potential of leptin replacement to rescue metabolic abnormalities in animal models of lipodystrophy were followed by the first clinical study of leptin therapy in patients with severe lipodystrophy. This and several other long-term studies demonstrated important benefits of recombinant human leptin (metreleptin) to treat metabolic abnormalities of lipodystrophy. These studies ultimately led to the recent FDA approval of metreleptin for the treatment of generalized lipodystrophy and EMA approval for both generalized and partial lipodystrophy. Additional research efforts in progress focus on novel treatment options, predominantly for patients with partial lipodystrophy. Current treatment of generalized lipodystrophy includes metreleptin replacement as an adjunct to diet and standard treatment approach for metabolic consequences of lipodystrophy. Beyond metreleptin, a number of different compounds and treatment modalities are being studied for the treatment of partial lipodystrophy.
本文旨在详细总结治疗脂肪萎缩综合征的常规治疗方案和 metreleptin 疗法，并指出当前正在开发的研究性治疗方法。
观察到脂肪萎缩症患者存在瘦素缺乏，并在脂肪萎缩症动物模型中发现瘦素替代治疗可挽救代谢异常，随后进行了首例严重脂肪萎缩症患者的瘦素治疗临床研究。该研究和其他几项长期研究表明，重组人瘦素（metreleptin）治疗脂肪萎缩症代谢异常具有重要益处。这些研究最终导致 FDA 最近批准 metreleptin 用于治疗全身性脂肪萎缩症，EMA 批准 metreleptin 用于治疗全身性和部分性脂肪萎缩症。正在进行的其他研究侧重于新型治疗方法，主要针对部分性脂肪萎缩症患者。目前，全身性脂肪萎缩症的治疗包括 metreleptin 替代治疗，作为饮食和标准治疗方法的辅助手段，以治疗脂肪萎缩症的代谢后果。除了 metreleptin 之外，还有许多不同的化合物和治疗方式正在研究用于治疗部分性脂肪萎缩症。
