Predictors for clinical trial participation in the rare lung disease lymphangioleiomyomatosis

• Published in: Respiratory medicine 2010-04, Vol.104 (4), p.578-583
• Main Authors: Kinder, Brent W, Sherman, A.C, Young, L.R, Hagaman, J.T, Oprescu, N, Byrnes, S, McCormack, Francis X
• Format: Article
• Language: English
• Subjects: Age Factors; Biological and medical sciences; Clinical trial; Clinical Trials as Topic - statistics & numerical data; Female; Health Services Accessibility; Humans; Lung Diseases - therapy; Lymphangioleiomyomatosis; Lymphangioleiomyomatosis - therapy; Medical sciences; Middle Aged; North America; Patient Participation - statistics & numerical data; Patient Selection; Pneumology; Pulmonary/Respiratory; Rare diseases; Rare Diseases - therapy; Research Design; Survey; Surveys and Questionnaires; Trial participation
• ISSN: 0954-6111; 1532-3064
• DOI: 10.1016/j.rmed.2009.09.023

Summary 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 respondents in North America had participated in a clinical trial. Age, disease duration, lack of any college education, use of oxygen therapy, and presentation without chest pain were associated with trial participation in unadjusted analyses. Multivariate analyses indicate that patient age was the strongest independent predictor for trial participation (OR = 2.07, p = 0.004 per decade greater of patient age). Common reasons reported against trial participation included not meeting enrollment criteria (44%), drug toxicity (25%), and stable disease (20%). The most frequent reason reported for trial participation was to help future patients (85%). Conclusions Study entry criteria, drug toxicity, and stability of disease are barriers to trial enrollment among subjects with LAM. Older LAM patients and those with more advanced disease are more likely to have participated in clinical trials. Altruism is commonly a motivating factor.