Predictors for clinical trial participation in the rare lung disease lymphangioleiomyomatosis.

ABSTRACT:

Background

Lymphangioleiomyomatosis (LAM) is a rare, progressive and frequently lethal cystic lung disease that almost exclusively affects women and has no proven therapies. An improved understanding of the pathogenesis has identified promising molecular targets for clinical trials. Although barriers, modifiers, and benefits for clinical trial participation in common diseases such as cancer have been studied, we are unaware of such evaluations concerning rare diseases.

Methods

We performed a survey of a population-based registry of 780 LAM subjects in North America to identify predictors of trial participation. Logistic regression analysis evaluated the association of demographic and clinical features with trial participation.

Results

41 of 263 (16%) LAM patient respo

SUBMITTER: Kinder BW 

PROVIDER: S-EPMC4407662 | biostudies-literature | 2010 Apr

REPOSITORIES: biostudies-literature