Efficacy of leptin therapy in the different forms of human lipodystrophy

Aims/hypothesis Lipodystrophy is a rare disorder characterised by loss of adipose tissue, hypoleptinaemia, severe insulin resistance, diabetes and dyslipidaemia. The aims of this study were to determine whether leptin replacement in lipodystrophy patients ameliorates their metabolic abnormalities over an extended period of time and whether leptin therapy is effective in the different forms of lipodystrophy. Methods We conducted an open-label prospective study of patients with acquired forms of lipodystrophy and inherited forms of lipodystrophy secondary to mutations in the AGPAT2 , SEIPIN (also known as BSCL2 ), LMNA and PPARγ (also known as PPARG ) genes. Between July 2000 and November 2008, 48 patients with lipodystrophy were treated with s.c. recombinant methionyl human leptin. Results Serum triacylglycerol and HbA 1c levels declined dramatically with leptin therapy. Among 35 patients with data at baseline and 12 months, serum triacylglycerol fell by 59% (from 10.18 ± 2.67 mmol/l to 4.16 ± 0.99 mmol/l [means ± SE]; p = 0.008) and HbA 1c decreased by 1.5 percentage points (from 8.4 ± 0.3% to 6.9 ± 0.3%; p