title: Leptin treatment: facts and expectations.
abstract: Leptin has key roles in the regulation of energy balance, body weight, metabolism, and endocrine function. Leptin levels are undetectable or very low in patients with lipodystrophy, hypothalamic amenorrhea, and congenital leptin deficiency (CLD) due to mutations in the leptin gene. For these patients, leptin replacement therapy with metreleptin (a recombinant leptin analog) has improved or normalized most of their phenotypes, including normalization of endocrine axes, decrease in insulin resistance, and improvement of lipid profile and hepatic steatosis. Remarkable weight loss has been observed in patients with CLD. Due to its effects, leptin therapy has also been evaluated in conditions where leptin levels are normal or high, such as common obesity, diabetes (types 1 and 2), and Rabson-Mendenhall syndrome. A better understanding of the physiological roles of leptin may lead to the development of leptin-based therapies for other prevalent disorders such as obesity-associated nonalcoholic fatty liver disease, depression and dementia.
authors.affiliation: The John Curtin School of Medical Research, The Australian National University, Canberra, Australia. Electronic address: gilbertjpf@hotmail.com.
authors.forename: Gilberto
authors.initials: G
authors.lastname: Paz-Filho
authors.affiliation: The John Curtin School of Medical Research, The Australian National University, Canberra, Australia. Electronic address: Claudio.mastronardi@anu.edu.au.
authors.forename: Claudio A
authors.initials: CA
authors.lastname: Mastronardi
authors.affiliation: South Australian Health and Medical Research Institute and Flinders University, Adelaide, Australia. Electronic address: Julio.licinio@sahmri.com.
authors.forename: Julio
authors.initials: J
authors.lastname: Licinio
mesh_terms: D000818:Animals; D001835:Body Weight / Q000187:drug effects; D004734:Energy Metabolism / Q000187:drug effects; D020249:Hormone Replacement Therapy / Q000379:methods; D006801:Humans; D020738:Leptin / Q000378:metabolism / Q000494:pharmacology* / Q000627:therapeutic use*
chemical_list: D020738:Leptin
keywords: Congenital leptin deficiency; Leptin; Metreleptin; Myalept; Obesity