Lipodystrophies in non-insulin-dependent children: Treatment options and results from recombinant human leptin therapy

Lipodystrophy is a general definition containing different pathologies which, except for those observed in insulin-treated subjects falling outside the scope of this paper, are characterized by total or partial lack of body fat, that, according to the amount of missing adipose tissue, are divided in generalized or partial lipodystrophy. These diseases are characterized by leptin deficiency, which often leads to metabolic derangement, causing insulin resistance, dyslipidemia, and increasing cardiovascular risk. In this narrative review, we presentend the clinical presentation of different types of lipodystrophies and metabolic unbalances related to disease in children and adolescents, focusing on the main treatment options and the novel results from recombinant human leptin (metreleptin) therapy. Milestones in the management of lipodystrophy include lifestyle modification as diet and physical activity, paired with hypoglycemic drugs, insulin, hypolipidemic drugs, and other drugs with the aim of treating lipodystrophy complications. Metreleptin has been recently approved for pediatric patients with general lipodystrophy (GL)> 2 years of age and for children with partial lipodystrophy (PL)> 12 years of age not controlled with conventional therapies. New therapeutic strategies are currently being investigated, especially for patients with PL forms, specifically, liver-targeted therapies. Further studies are needed to achieve the most specific and precise treatment possible.

Automated summary

Lipodystrophy is a group of diseases characterized by total or partial lack of body fat, resulting in metabolic derangement and increased cardiovascular risk. This paper focuses on the clinical presentation, treatment options, and the use of recombinant human leptin therapy. Currently, lifestyle modification, medication, and leptin therapy are the main treatment strategies for lipodystrophy, particularly for children and adolescents. Liver-targeted therapies are being investigated as potential new therapeutic strategies for lipodystrophy, especially for patients with partial lipodystrophy.