Clinical trials : journal of the Society for Clinical Trials
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Benign prostatic hyperplasia (BPH), a common condition among older men, confers its morbidity through potentially bothersome lower urinary tract symptoms. Treatments for BPH include drugs such as alpha-adrenergic receptor blockers and 5-alpha reductase inhibitors, minimally invasive therapies that use heat to damage or destroy prostate tissue, and surgery including transurethral resection of the prostate. Complementary and alternative medicines are gaining popularity in the US. Two phytotherapies commonly used for BPH are extracts of the fruit of Serenoa repens, the Saw palmetto dwarf palm that grows in the Southeastern US, and extracts of the bark of Pygeum africanum, the African plum tree. ⋯ Trial design occurs in an environment of continually evolving information. In this case, emerging results from another trial suggested that a study of long-term efficacy was premature, and that an effective dose and preparation of S. repens had to be established before proceeding to a long-term clinical trial.
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Clinical research in trauma patients poses multiple challenges in study design. These reflect the heterogeneity of injury and treatment, the paucity of acceptable study endpoints aside from mortality, and the difficulties inherent in obtaining informed consent in acutely ill populations. A current example of this problem is the study of recombinant factor VIIa (rFVIIa), which has attracted considerable interest as a systemic procoagulant agent for use in trauma patients with exsanguinating hemorrhage. ⋯ Research in acutely hemorrhaging trauma patients presents numerous scientific and ethical challenges. The methodology of the CONTROL study is presented as an example of how some of these challenges can be approached and managed, and of the pitfalls that may arise.
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Randomized Controlled Trial Comparative Study
Design, conduct, and analyses of Breast International Group (BIG) 1-98: a randomized, double-blind, phase-III study comparing letrozole and tamoxifen as adjuvant endocrine therapy for postmenopausal women with receptor-positive, early breast cancer.
Aromatase inhibitors provide superior disease control when compared with tamoxifen as adjuvant therapy for postmenopausal women with endocrine-responsive early breast cancer. ⋯ BIG 1-98 is an example of an enriched design, involving complementary analyses addressing different questions several years apart, and subject to evolving analytic plans influenced by new data that emerge over time.
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Comparative Study
Evaluating the benefit of event adjudication of cardiovascular outcomes in large simple RCTs.
Event adjudication in randomized controlled trials is thought to be a necessary step to remove noise and potential bias from the results [1,2]. However, this hypothesis has not been widely evaluated. We conducted a meta-analysis of a series of cardiovascular outcomes trials and estimated the effect of adjudication on treatment estimates and on the number of outcomes included the trials. ⋯ This systematic meta-analysis failed to detect any effect of event adjudication on study conclusions and the numbers of events included in the final analyses were minimally changed. Given the considerable effort required to perform adjudication, there is a need to demonstrate that this process does indeed increase the sensitivity of trials. There is a need to conduct more systematic analyses of the effect of event adjudication in other trials to determine if this process is truly worthwhile.
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Chronic Obstructive Pulmonary Disease (COPD) is a progressive illness characterized by airflow obstruction and dyspnea that afflicts over 12 million people and represents a leading cause of death in the United States. Not surprisingly, COPD is often associated with emotional distress and reduced psychosocial adjustment, which can negatively impact physical functioning and impair quality of life. However, the psychosocial consequences of COPD remain largely untreated. A previous randomized trial from our research team demonstrated that coping skills training (CST) can improve pulmonary-specific quality of life among pulmonary patients awaiting lung transplant (the INSPIRE study). To date, however, no studies have examined the effects of a caregiver-assisted CST intervention in patients with COPD with less severe disease. ⋯ We believe that this novel approach to patient care in which caregivers are used to assist in the delivery of coping skills training to patients with COPD has the potential to change the way in which COPD patients are routinely managed in order to reduce distress, enhance quality of life, and potentially improve medical outcomes.