Lipodystrophies are a heterogeneous group of disorders characterized by congenital or acquired
loss of adipose tissue. Recently, metreleptin, a recombinant human leptin analog, has been approved
for the treatment of patients with generalized lipodystrophy. Leptin is an adipokine which has
a fundamental role in glucose and lipid homeostasis. Metreleptin treatment has been demonstrated to
improve metabolic abnormalities such as hyperglycemia, hypertriglyceridemia, increased hepatic fat
content and elevated liver enzymes alanine transaminase and aspartate transaminase in patients with generalized lipodystrophy,
and to correct hyperphagia that likely occurs as a result of leptin deficiency. Limited data has also suggested that
metreleptin treatment might be beneficial on metabolic abnormalities in patients with partial lipodystrophy. This review
focuses on potential benefits of metreleptin in various forms of non-HIV associated lipodystrophy. Safety issues have
been discussed. Recent patent submissions have also been reviewed.
Keywords: Diabetes, fat loss, hepatic steatosis, hypertriglyceridemia, insulin resistance, lipodystrophy, metreleptin.
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