Metreleptin treatment of non-HIV lipodystrophy syndromes

Lipodystrophy syndromes (LS) constitute a group of rare diseases of the adipose tissue, characterized by a complete or selective deficiency of the fat mass. These disorders are associated with important insulin resis-tance, cardiovascular and metabolic comorbidities that impact patient's survival and quality of life. Manage-ment is challenging and includes diet, physical activity, and specific pharmacological treatment of LS-associated comorbidities. Because of a common pathophysiology involving decreased concentration of the adipokine leptin, efforts have been made to develop therapeutic strategies with leptin replacement therapy. Metreleptin, a recombinant human leptin analogue, has been proposed in hypoleptinemic patients since the beginning of 2000's. The treatment leads to an improvement in metabolic parameters, more important in gen-eralized than in partial LS forms. In this review, the current knowledge about the development of the drug, its outcomes in the treatment of lipodystrophic patients as well as the peculiarities of its use will be presented. (c) 2021 Published by Elsevier Masson SAS.

Automated summary

Lipodystrophy syndromes are rare diseases of the adipose tissue characterized by fat deficiency, leading to important comorbidities affecting patient survival and quality of life. Treatment includes diet, physical activity, and pharmacological intervention.