NOVEL THERAPIES FOR METABOLIC COMPLICATION IN PATIENTS WITH LIPODYSTROPHIES

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Lipodystrophies are a heterogeneous group of disorders characterized by selective loss of adipose tissue. Affected patients frequently have metabolic complications related to insulin resistance such as diabetes, hypertriglyceridemia and hepatic steatosis. Due to the extreme insulin resistance, treatment of these conditions represents a major therapeutic challenge. There is marked paucity of clinical trials evaluating treatment options in patients with lipodystrophy. The purpose of the study is to examine the safety and efficacy of various novel interventions designed to improve or resolve diabetes, dyslipidemia and fatty liver in these patients. The specific interventions to be examined are low fat diet, leptin replacement therapy, cholic acid therapy and treatment with the peroxisome proliferator activated receptors (PPAR) agonist, pioglitazone.