Uncontrolled Study

Abstract: Uncontrolled studies on the effects of a treatment in a series of patients have a tendency to report favourable results, whereas case reports often inform about adverse effects. Accordingly, the recent publication of an open uncontrolled study reporting favourable effects of an inhaled prostacyclin analogue, iloprost, in patients with primary pulmonary hypertension (PPH) 1, did not come as a surprise. What came as a surprise was a methodologically comparable study on the same treatment, in a similar type of patient, in this issue of the European Respiratory Journal , with completely opposite conclusions 2. How is such a discrepancy possible? The answer is straightforward, and holds in a single word: methodology. Phase III controlled clinical trials (CCT) are required by both the scientific community and Regulatory Agencies as definitive proof of the safety and efficacy of new modalities of treatment. Uncontrolled, small phase II studies are performed at the very beginning of the development of new treatments to confirm the expected effects (proof of concept) and to assess the effective dose (dose finding). A well−designed phase III CCT incorporates elements such as prospective randomization, placebo-control and double-blindness, that ensure the reliability of the results. To increase the chance of success, a pivotal CCT needs an appropriate sample size based on the foreseeable changes of predefined primary end-points. Small, pilot phase III studies are usually required to gauge those changes and to test the appropriateness of the study design. A multicentre cooperation ensures a fast enrolment rate and reproducibility of results. The development of the current treatments of patients with pulmonary arterial hypertension (PAH), defined after the World Health Organization classification 3, have not followed all the golden rules of CCT. In fact, the favourable effect of oral anticoagulant therapy in patients with PPH or pulmonary hypertension associated …

Abstract: Objective : To investigate the effectiveness of workplace exercise for employee health by means of health-related physical activity components. Methods : A randomized uncontrolled study with 20 workers was carried out during three months to evaluate a workplace exercise program. The selected outcomes were flexibility, body mass, fat percentage, lean mass, blood pressure, and heart rate. For statistical analysis, the paired t test and the intent-to-treat analysis were used. Results : There was a significant increase in weight, fat percentage, blood pressure, and heart rate. However the clinical significance was 10% in the size of the effect. Conclusion : The changes verified in the outcomes analyzed were not significant; the variables are within normality ranges proposed by academic organizations