Partial and generalized lipodystrophy: comparison of baseline characteristics and response to metreleptin.

ABSTRACT:

Context

Lipodystrophies are extreme forms of metabolic syndrome. Metreleptin was approved in the United States for generalized lipodystrophy (GLD) but not partial lipodystrophy (PLD).

Objective

The objective of the study was to test metreleptin's efficacy in PLD vs GLD and find predictors for treatment response.

Design

This was a prospective, single-arm, open-label study since 2000 with continuous enrollment. Current analysis included metreleptin treatment for 6 months or longer as of January 2014.

Setting

The study was conducted at the National Institutes of Health (Bethesda, Maryland).

Participants

Patients clinically diagnosed with lipodystrophy, leptin less than 8 ng/mL (males) or less than 12 (females), age older than 6 months, and one or more metab

SUBMITTER: Diker-Cohen T 

PROVIDER: S-EPMC4422900 | biostudies-literature | 2015 May

REPOSITORIES: biostudies-literature