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Treatment Options for Lipodystrophy in Children

Lipodystrophy includes a heterogeneous group of rare diseases characterized by different amounts of adipose tissue loss and several metabolic complications, including hypertriglyceridemia, steatohepatitis and particularly insulin resistance, that may lead to severe morbidity and, sometimes, mortalit...

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Autores principales: Mainieri, Francesca, Tagi, Veronica Maria, Chiarelli, Francesco
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9114741/
https://www.ncbi.nlm.nih.gov/pubmed/35600578
http://dx.doi.org/10.3389/fendo.2022.879979
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author Mainieri, Francesca
Tagi, Veronica Maria
Chiarelli, Francesco
author_facet Mainieri, Francesca
Tagi, Veronica Maria
Chiarelli, Francesco
author_sort Mainieri, Francesca
collection PubMed
description Lipodystrophy includes a heterogeneous group of rare diseases characterized by different amounts of adipose tissue loss and several metabolic complications, including hypertriglyceridemia, steatohepatitis and particularly insulin resistance, that may lead to severe morbidity and, sometimes, mortality. Therefore, therapy for lipodystrophy primarily consists of a conventional approach that involves standard treatments of metabolic abnormalities. Given the evidence of leptin deficiency in lipodystrophy syndromes, leptin replacement therapy has been considered as a treatment option. Long-term studies on the use of therapy with a methionylated analog of human leptin, metreleptin, first on animals and subsequently on human patients, demonstrated enormous improvements of patients’ clinical features and metabolic conditions. Recently, metreleptin was approved by Food and Drug Administration (FDA) for the treatment of generalized lipodystrophy and by European Medicines Agency (EMA) for the treatment of both generalized and partial lipodystrophy. However, further research is being conducted for new and different therapeutic agents, especially helpful for the treatment of patients with partial lipodystrophy, as some of them do not have access to metreleptin therapy or show poor response.
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spelling pubmed-91147412022-05-19 Treatment Options for Lipodystrophy in Children Mainieri, Francesca Tagi, Veronica Maria Chiarelli, Francesco Front Endocrinol (Lausanne) Endocrinology Lipodystrophy includes a heterogeneous group of rare diseases characterized by different amounts of adipose tissue loss and several metabolic complications, including hypertriglyceridemia, steatohepatitis and particularly insulin resistance, that may lead to severe morbidity and, sometimes, mortality. Therefore, therapy for lipodystrophy primarily consists of a conventional approach that involves standard treatments of metabolic abnormalities. Given the evidence of leptin deficiency in lipodystrophy syndromes, leptin replacement therapy has been considered as a treatment option. Long-term studies on the use of therapy with a methionylated analog of human leptin, metreleptin, first on animals and subsequently on human patients, demonstrated enormous improvements of patients’ clinical features and metabolic conditions. Recently, metreleptin was approved by Food and Drug Administration (FDA) for the treatment of generalized lipodystrophy and by European Medicines Agency (EMA) for the treatment of both generalized and partial lipodystrophy. However, further research is being conducted for new and different therapeutic agents, especially helpful for the treatment of patients with partial lipodystrophy, as some of them do not have access to metreleptin therapy or show poor response. Frontiers Media S.A. 2022-05-04 /pmc/articles/PMC9114741/ /pubmed/35600578 http://dx.doi.org/10.3389/fendo.2022.879979 Text en Copyright © 2022 Mainieri, Tagi and Chiarelli https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Endocrinology
Mainieri, Francesca
Tagi, Veronica Maria
Chiarelli, Francesco
Treatment Options for Lipodystrophy in Children
title Treatment Options for Lipodystrophy in Children
title_full Treatment Options for Lipodystrophy in Children
title_fullStr Treatment Options for Lipodystrophy in Children
title_full_unstemmed Treatment Options for Lipodystrophy in Children
title_short Treatment Options for Lipodystrophy in Children
title_sort treatment options for lipodystrophy in children
topic Endocrinology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9114741/
https://www.ncbi.nlm.nih.gov/pubmed/35600578
http://dx.doi.org/10.3389/fendo.2022.879979
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