Elif A Oral
Endocrinology and Diabetes,
1000 Wall Street, Room 5313, Ann Arbor, MI 48105
Tanzania
Research Article
Efficacy and Safety of Metreleptin in Patients with Partial Lipodystrophy: Lessons from an Expanded Access Program
Author(s): Nevin Ajluni, Moahad Dar, John Xu, Adam H Neidert and Elif A OralNevin Ajluni, Moahad Dar, John Xu, Adam H Neidert and Elif A Oral
Objective: Patients with lipodystrophy have severe metabolic abnormalities (insulin resistance, diabetes, and hypertriglyceridemia) that may increase morbidity and mortality. Metreleptin is approved by the United States Food and Drug Administration for treatment of generalized forms of lipodystrophy. We aimed to determine the efficacy and safety of metreleptin among patients with partial lipodystrophy using an expanded-access model.
Methods: Study FHA101 (ClinicalTrials.gov identifier: NCT00677313) was an open-label, expanded-access, long-term clinical effectiveness and safety study in 23 patients with partial lipodystrophy and diabetes and/or hypertriglyceridemia with no prespecified leptin level. Metreleptin was administered subcutaneously at 0.02 mg/kg twice daily (BID) at Week 1, followed by 0.04 mg/kg B.. View More»
