The Metreleptin Effectiveness and Safety Registry (MEASuRE): concept, design and challenges.

ABSTRACT:

Background

Metreleptin, a recombinant analog of human leptin, is an approved therapy, adjunct to diet, to treat the metabolic complications of leptin deficiency in patients with lipodystrophy - a group of rare diseases characterized by a paucity of adipose tissue. MEASuRE (Metreleptin Effectiveness And Safety Registry) is a post-authorization, voluntary registry that gathers long-term safety and effectiveness data on metreleptin. Here, we present the aims and evolution of MEASuRE.

Methods

MEASuRE was established to collect data from patients receiving commercially supplied metreleptin in the United States (US) and European Union (EU). MEASuRE aims to determine the incidence and severity of safety events and describe the clinical characteristics and therapeutic outcomes in th

SUBMITTER: Haymond MW 

PROVIDER: S-EPMC10214317 | biostudies-literature | 2023 May

REPOSITORIES: biostudies-literature