Our population-based study showed that the median survival in patients with LAM from the onset of symptoms or diagnosis is much longer than previously described. This has important implications for life choices and treatment decisions regarding medication use and lung transplantation for patients with LAM.

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Introduction: The inflammatory hypothesis of atherosclerosis is appealing in acute coronary syndromes, but the dynamics and precise role are not established.Objectives: The study investigates the levels of C reactive protein (CRP), interleukin 1b (IL-1b) and stromal-derived factor 1a (SDF-1a) at the time of acute myocardial infarction (AMI) and at 1 and 6 months afterwards, compared with a control group. Results: In the acute phase of AMI, CRP and SDF-1a were significantly higher, while IL-1b showed lower levels compared with controls. CRP positively correlated with coronary stenosis severity (rho ¼ 0.3, p¼.05) and negatively related with left ventricle ejection fraction (LVEF) at 1 month (rho¼ À0.43, p¼.05). IL-1b weakly correlated with the severity of coronary lesions (rho ¼0.29, p¼.02) and strongly with LVEF (rho¼ À0.8, p¼.05). SDF-1a, slightly correlated with LVEF at 1 month (rho ¼ 0.22, p¼.01) and with the severity of coronary atherosclerosis (rho¼ À0.41, p¼.003). Conclusions: CRP, IL-1b and SDF-1a have important dynamic in the first 6 months after AMI and CRP and SDF-1a levels correlated with the severity of coronary lesions and LVEF at 1 month after the acute ischaemic event.

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.