Metreleptin beneficial to patients with partial lipodystrophy
Use of metreleptin for the treatment of patients with partial lipodystrophy (PL) is relatively safe and produces improvements in glycaemic control, hypertriglyceridaemia and liver volume, according to the results of an open-label study and its long-term extension phase.
The current analysis included 41 PL patients (median age, 34 years; 97.6 percent female) circulating leptin <12.0 ng/mL, and diabetes mellitus, insulin resistance or hypertriglyceridaemia who received metreleptin doses (once or twice daily) titrated to a mean of 0.124 mg/kg/day.
Treatment yielded significant reductions from baseline to month 12 in the following endpoints of interest: glycated haemoglobin (HbA1c; –0.6 percent) and fasting serum triglycerides (TGs; –20.8 percent), fasting plasma glucose (FPG; –1.2 mmol/L) and liver volume (–13.4 percent; p<0.05 for all).
The observed benefits extended to the subgroup of patients with baseline HbA1c ≥6.5 percent or TGs ≥5.65 mmol/L: HbA1c (−0.9 percent), fasting TGs (−37.4 percent), FPG (−1.9 mmol/L) and liver volume (−12.4 percent; p<0.05 for all). In this subgroup, 67.9 percent of patients achieved a ≥1-percent decrease in HbA1c or ≥30-percent decrease in fasting TGs, while 42.9 percent of patients achieved ≥2-percent or ≥40-percent reduction, respectively.
Long-term treatment with metreleptin in the said subgroup likewise resulted in substantial improvements in HbA1c, fasting TGs and FPG at months 12, 24 and 36 (p<0.05).
In terms of safety, the drug was well tolerated with no new safety signals. Commonly reported treatment-emergent adverse events were abdominal pain, hypoglycaemia and nausea.
The present data demonstrate the usefulness of metreleptin in the treatment of PL, especially for patients with more severe metabolic disease, the researchers said.