The design and analysis of a conventional cancer clinical trial can be improved by adopting a robust statistical procedure that enables clinically meaningful interpretations of the treatment effect. The RMST-based quantitative method may be used as a primary tool for future cancer trials or to help us to better understand the clinical interpretation of the HR even when its model assumption is plausible.

BackgroundWhen transplanted into failing heart, autologous somatic tissue–derived cells yield functional recovery via paracrine effects that enhance native regeneration. However, the therapeutic effects are modest. We developed a method in which scaffold‐free cell sheets are attached to the epicardial surface to maximize paracrine effects. This Phase I clinical trial tested whether transplanting autologous cell–sheets derived from skeletal muscle is feasible, safe, and effective for treating severe congestive heart failure.Methods and ResultsFifteen ischemic cardiomyopathy patients and 12 patients with dilated cardiomyopathy, who were in New York Heart Association functional class II or III and had been treated with the maximum medical and/or interventional therapies available, were enrolled. Scaffold‐free cell sheets of 3 to 9×108 cells derived from autologous muscle were transplanted over the LV free wall via left thoracotomy, without additional interventional treatments. There were no procedure‐related major complications during follow‐up. The majority of the ischemic cardiomyopathy patients showed marked symptomatic improvement in New York Heart Association classification (pre: 2.9±0.5 versus 6 months: 2.1±0.4, P<0.01; 1 year: 1.9±0.3, P<0.01) and the Six‐Minute Walk Test with significant reduction of serum brain natriuretic peptide level (pre: 308±72 pg/mL versus 6 months: 191±56 versus 1 year: 182±46, P<0.05), pulmonary artery pressure, pulmonary capillary wedge pressure, pulmonary vein resistance, and left ventricular wall stress after transplantation instead of limited efficacy in dilated cardiomyopathy patients.ConclusionsCell‐sheet transplantation as a sole therapy was feasible for treating cardiomyopathy. Promising results in the safety and functional recovery warrant further clinical follow‐up and larger studies to confirm this treatment's efficacy for severe congestive heart failure.Clinical Trial Registration URL: http://www.umin.ac.jp/english/. Unique identifier: UMIN000003273.

Background: Capturing epidemiological signatures is essential to document burdens of disease and to design health care services, including prevention measures, clinical interventions, and policies. There are large geographical and ethnic variations in the epidemiology of allergic and immunological diseases. Various data are available from North America and Europe, but the epidemiology of allergic and immunological diseases in Asia is not well documented. Objective: To characterize epidemiological signatures of allergic and immunological disease in young children in Japan. Methods: This was a national, multicenter, prospective birth cohort study: Japan Environment and Children's Study (JECS). A general population of 103,060 women was enrolled during pregnancy. Allergic and immunological outcomes were assessed among young children using questionnaire data. Results: The prevalence of caregiver-reported immediate food allergy was 7.6%, 6.7%, and 4.9% at age 1, 2, and 3 years, respectively. Hen egg allergy was most common (5.4% prevalence at age 1 year) followed by allergies to cow milk and wheat. Several patterns of allergic symptom clusters were identified. Physician diagnosed, as reported by the caregiver, non-IgE mediated gastrointestinal food allergy affected 0.5% of infants. By contrast, caregiver-reported gastrointestinal food allergies affected 1.4% of children. Kawasaki disease affected 0.3% and 0.4% children, respectively, at age 1 and 3 years. Primary immunodeficiency disorders affected 0.005% children at age 3 years. Conclusion: These data provide important epidemiological signatures of allergy and immunology in young Japanese children including the age-specific prevalence of allergic disease, Kawasaki disease, and primary immune deficiency.

No abstract

No abstract