Effects of Metreleptin in Pediatric Patients With Lipodystrophy.

ABSTRACT:

Context

Lipodystrophy syndromes are rare disorders of deficient adipose tissue. Metreleptin, a human analog of leptin, improved metabolic abnormalities in mixed cohorts of children and adults with lipodystrophy and low leptin.

Objective

Determine effects of metreleptin on diabetes, hyperlipidemia, nonalcoholic fatty liver disease (NAFLD), growth, and puberty in pediatric patients with lipodystrophy and low leptin.

Design

Prospective, single-arm, open-label studies with continuous enrollment since 2000.

Setting

National Institutes of Health, Bethesda, Maryland.

Patients

Fifty-three patients aged 6 months to <18 years with lipodystrophy, leptin level <8 ng/mL (male patients) or <12 ng/mL (female patients), and ≥1 metabolic abnormality (diabetes, insulin re

SUBMITTER: Brown RJ 

PROVIDER: S-EPMC5443330 | biostudies-literature | 2017 May

REPOSITORIES: biostudies-literature