Endocrine Abstracts

We report new estimates of the disutility associated with GL and PL, and the quality of life (QoL) impact of leptin replacement therapy (LRT) based on two methods. The first approach estimates utility decrements associated with a range of impairments related to lipodystrophy through a discrete choice experiment (DCE) conducted with members of the general public; the second elicits QoL estimates from 8 PL patients using the SF-36 instrument. To characterize QoL consequences of ...

Objective: To evaluate the real-world experience of patients with generalized (GL) and partial (PL) lipodystrophy initiating treatment with metreleptin as part of an early access program (EAP).Methods: A retrospective data collection was conducted from four countries for patients enrolled in the EAP. A descriptive analysis was performed on selected patient characteristics, baseline organ impairments and complications, and response to metreleptin therapy,...