{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T00:22:08Z","timestamp":1791332528179,"version":"4.3.1"},"reference-count":34,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2007,10,8]],"date-time":"2007-10-08T00:00:00Z","timestamp":1191801600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Clinical Endocrinology"],"published-print":{"date-parts":[[2008,4]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p><jats:bold>Aims\/hypothesis\u2003<\/jats:bold> Partial lipodystrophy (PL) is most commonly characterized by loss of subcutaneous fat in the extremities with preservation of truncal fat and is associated with insulin resistance, diabetes and hyperlipidaemia. Recombinant human leptin (r\u2010metHuLeptin) therapy has been shown to be effective in treating metabolic abnormalities associated with congenital or acquired generalized lipodystrophy and PL associated with lamin A\/C (<jats:italic>LMNA<\/jats:italic>) gene mutations or highly active antiretroviral therapy (HAART). Our aim was to assess the effectiveness of leptin therapy in treating metabolic complications of PL associated with heterozygous peroxisome proliferator activated receptor gamma (<jats:italic>PPARG<\/jats:italic>) mutations. This is the first report to detail the clinical response of a patient with PL due to a <jats:italic>PPARG <\/jats:italic>mutation treated with r\u2010metHuLeptin.<\/jats:p><jats:p><jats:bold>Methods\u2003<\/jats:bold> A 36\u2010year\u2010old female with PL associated with a heterozygous <jats:italic>PPARG<\/jats:italic> mutation complicated by poorly controlled diabetes and severe, refractory hypertriglyceridaemia was enrolled in a National Institutes of Health (NIH) protocol to evaluate the role of r\u2010metHuLeptin in lipodystrophy. The patient received escalating doses of r\u2010metHuLeptin until a dose 0\u00b712\u00a0mg\/kg\/day was reached. Metabolic parameters, including serum chemistries, fasting blood glucose, glycated haemoglobin (HbA1c), lipid profile, an oral glucose tolerance test (OGTT), an insulin tolerance test (ITT), liver volume, percentage body fat and energy expenditure were followed at regular time intervals over 18\u00a0months of therapy.<\/jats:p><jats:p><jats:bold>Results\u2003<\/jats:bold> Eighteen months of r\u2010MetHuLeptin therapy was associated with a marked improvement in glucose homeostasis as evidenced by normalization of the fasting blood glucose (baseline\u00a0=\u00a08\u00b73\u00a0mmol\/l; 18\u00a0months\u00a0=\u00a04\u00b79\u00a0mmol\/l), lowering of HbA1c (baseline\u00a0=\u00a09\u00b79%; 18\u00a0months\u00a0=\u00a07\u00b72%) and improved tolerance to an oral glucose load. In addition, a striking amelioration in the patient's refractory, severe hypertriglyceridaemia was observed (baseline\u00a0=\u00a021\u00b715\u00a0mmol\/l; 18\u00a0months\u00a0=\u00a05\u00b796\u00a0mmol\/l).<\/jats:p><jats:p><jats:bold>Conclusion\u2003<\/jats:bold> r\u2010MetHuLeptin is effective in treating metabolic complications associated with PL due to <jats:italic>PPARG<\/jats:italic> mutations. In the context of previously published work, our findings suggest that the response to r\u2010MetHuLeptin is independent of the aetiology in lipodystrophy.<\/jats:p>","DOI":"10.1111\/j.1365-2265.2007.03095.x","type":"journal-article","created":{"date-parts":[[2007,10,8]],"date-time":"2007-10-08T17:55:10Z","timestamp":1191866110000},"page":"547-554","source":"Crossref","is-referenced-by-count":34,"title":["Leptin therapy for partial lipodystrophy linked to a <i>PPAR\u2010\u03b3<\/i> mutation"],"prefix":"10.1111","volume":"68","author":[{"given":"Jean\u2010Marc","family":"Guettier","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean Y.","family":"Park","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elaine 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