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Trial Outcomes (trial + outcome)
Selected AbstractsLessons learned from a randomized trial of airway secretion clearance techniques in cystic fibrosis,PEDIATRIC PULMONOLOGY, Issue 3 2010Marci K. Sontag PhD Abstract Rationale Airway secretion clearance therapies are a cornerstone of cystic fibrosis care, however longitudinal comparative studies are rare. Our objectives were to compare three therapies [postural drainage and percussion: (postural drainage), flutter device (FD), and high frequency chest wall oscillation: (vest)], by studying (1) change in pulmonary function; (2) time to need for intravenous (IV) antibiotics, (3) use of pulmonary therapies, (4) adherence to treatment, (5) treatment satisfaction, and (6) quality of life. Methods Participants were randomly assigned to one of three therapies twice daily. Clinical outcomes were assessed quarterly over 3 years. Results Enrollment goals were not met, and withdrawal rates were high, especially in postural drainage (51%) and FD (26%), compared to vest (9%), resulting in early termination. FEV1 decline, time to need IV antibiotics, and other pulmonary therapies were not different. The annual FEF25,75% predicted rate of decline was greater in those using vest (P,=,0.02). Adherence was not significantly different (P,=,0.09). Overall treatment satisfaction was higher in vest and FD than in postural drainage (P,<,0.05). Health-related quality of life was not different. The rate of FEV1 decline was 1.23% predicted/year. Conclusions The study was ended early due to dropout and smaller than expected decline in FEV1. Patients were more satisfied with vest and FD. The longitudinal decline in FEF25,75% was faster in vest; we found no other difference in lung function decline, taken together this warrants further study. The slow decline in FEV1 illustrates the difficulty with FEV1 decline as a clinical trial outcome. Pediatr Pulmonol. 2010; 45:291,300. © 2010 Wiley-Liss, Inc. [source] Understanding clinical trial outcomes: design, analysis, and interpretationDERMATOLOGIC THERAPY, Issue 2 2007Heidi T. Jacobe ABSTRACT:, Outcomes (a variable intended for comparison between groups) are integral to the design, conduct, and data analysis of a clinical trial. They are broadly divided into four categories: physician based, patient reported, economic based, and technology based. Each is used in dermatology to some degree, but no consensus exists as to what type of outcome or degree of validation should be employed. This is problematical because poor quality outcomes or their incorrect use may invalidate the results of a clinical trial. Despite their importance, outcome measures in dermatology receive little attention. The present authors aim to provide an overview of important considerations for outcome measures and a practical approach to their analysis. [source] Skeletal Estimation and Identification in American and East European Populations,JOURNAL OF FORENSIC SCIENCES, Issue 3 2008Erin H. Kimmerle Ph.D. Abstract:, Forensic science is a fundamental transitional justice issue as it is imperative for providing physical evidence of crimes committed and a framework for interpreting evidence and prosecuting violations to International Humanitarian Law (IHL). The evaluation of evidence presented in IHL trials and the outcomes various rulings by such courts have in regard to the accuracy or validity of methods applied in future investigations is necessary to ensure scientific quality. Accounting for biological and statistical variation in the methods applied across populations and the ways in which such evidence is used in varying judicial systems is important because of the increasing amount of international forensic casework being done globally. Population variation or the perceived effect of such variation on the accuracy and reliability of methods is important as it may alter trial outcomes, and debates about the scientific basis for human variation are now making their way into international courtrooms. Anthropological data on population size (i.e., the minimum number of individuals in a grave), demographic structure (i.e., the age and sex distribution of victims), individual methods applied for identification, and general methods of excavation and trauma analysis have provided key evidence in cases of IHL. More generally, the question of population variation and the applicability of demographic methods for estimating individual and population variables is important for American and International casework in the face of regional population variation, immigrant populations, ethnic diversity, and secular changes. The reliability of various skeletal aging methods has been questioned in trials prosecuted by the International Criminal Tribunal for the Former Yugoslavia (ICTY) in The Prosecutor of the Tribunal against Radislav Krsti, (Case No. IT-98-33, Trial Judgment) and again in the currently ongoing trial of The Prosecutor of the Tribunal against Zdravko Tolimir, Radivolje Mileti,, Milan Gvero, Vinko Pandurevi,, Ljubisa Beara, Vujadin Popovi,, Drago Nikoli,, Milorad Trbi,, Ljubomir Borovcanin (IT-05-88-PT, Second Amended Indictment). Following the trial of General Krsti,, a collaborative research project was developed between the Forensic Anthropology Center at The University of Tennessee (UT) and the United Nations, International Criminal Tribunal for the Former Yugoslavia, Office of the Prosecutor (ICTY). The purpose of that collaboration was to investigate methods used for the demographic analysis of forensic evidence and where appropriate to recalibrate methods for individual estimation of age, sex, and stature for specific use in the regions of the former Yugoslavia. The question of "local standards" and challenges to the reliability of current anthropological methods for biological profiling in international trials of IHL, as well as the performance of such methods to meet the evidentiary standards used by international tribunals is investigated. Anthropological methods for estimating demographic parameters are reviewed. An overview of the ICTY-UT collaboration for research aimed at addressing specific legal issues is discussed and sample reliability for Balkan aging research is tested. The methods currently used throughout the Balkans are discussed and estimated demographic parameters obtained through medico-legal death investigations are compared with identified cases. Based on this investigation, recommendations for improving international protocols for evidence collection, presentation, and research are outlined. [source] Standardizing Emergency Department,based Migraine Research: An Analysis of Commonly Used Clinical Trial Outcome MeasuresACADEMIC EMERGENCY MEDICINE, Issue 1 2010Benjamin W. Friedman MD Abstract Objectives:, Although many high-quality migraine clinical trials have been performed in the emergency department (ED) setting, almost as many different primary outcome measures have been used, making data aggregation and meta-analysis difficult. The authors assessed commonly used migraine trial outcomes in two ways. First, the authors examined the association of each commonly used outcome versus the following patient-centered variable: the research subject's wish, when asked 24 hours after investigational medication administration, to receive the same medication the next time they presented to an ED with migraine ("would take again"). This variable was chosen as the criterion standard because it provides a simple, dichotomous, clinically sensible outcome, which allows migraineurs to factor important intangibles of efficacy and adverse effects of treatment into an overall assessment of care. The second part of the analysis assessed how sensitive to true efficacy each outcome measure was by calculating sample size requirements based on results observed in previously conducted clinical trials. Methods:, This was a secondary analysis of data previously collected in four ED-based migraine randomized trials performed between 2003 and 2007. In each of these trials, subjects were asked 24 hours after administration of an investigational medication whether or not they would want to receive the same medication the next time they came to the ED with a migraine. Odds ratios (ORs) with 95% confidence intervals (CIs), adjusted for sex and medication received, were calculated as measures of association between the most commonly used outcome measures and "would take again." The sensitivity of each outcome measure to treatment efficacy was determined by calculating the sample size that would be required to detect a statistically significant result using estimates of that outcome obtained in two clinical trials. Results:, Data from 378 subjects were used for this analysis. Adjusted ORs for association of "would take again" and other commonly used primary headache outcomes are as follows: achieving a pain-free state by 2 hours, OR = 3.1 (95% CI = 1.8 to 5.4); sustained pain-free status, OR = 4.5 (95% CI = 1.9 to 11.0); and no need for rescue medication, OR = 3.7 (95% CI = 2.1 to 6.6). An improvement on a standardized 11-point pain scale of ,33% had an adjusted OR = 5.2 (95% CI = 2.2 to 12.4). The best performing alternate outcome, ,33% improvement, correctly classified 288 subjects and misclassified 77 subjects when compared to "would take again." At least 33% improvement and pain-free by 2 hours required the smallest sample sizes, while sustained pain-free and "would take again" required many more subjects. Conclusions:, "Would take again" was associated with all migraine outcome measures we examined. No individual outcome was more closely associated with "would take again" than any other. Even the best-performing alternate outcome misclassified more than 20% of subjects. However, sample sizes based on "would take again" tended to be larger than other outcome measures. On the basis of these findings and this outcome measure's inherent patient-centered focus, "would take again," included as a secondary outcome in all ED migraine trials, is proposed. ACADEMIC EMERGENCY MEDICINE 2010; 17:72,79 © 2010 by the Society for Academic Emergency Medicine [source] Liability insurance under the negligence ruleTHE RAND JOURNAL OF ECONOMICS, Issue 3 2009Marie-Cécile Fagart We incorporate the concept of evidentiary standard to the analysis of the negligence rule under liability insurance and court errors. When the postaccident evidence is privately contractible and not too noisy, efficiency is achieved by both strict liability and a negligence rule with appropriate due care and evidentiary standards. When the evidence is not directly contractible, trial outcomes represent useful contractible information for the risk-incentives tradeoff in the liability insurance policy. Strict liability is then inefficient and dominated by the negligence rule. The negligence rule can itself be improved upon by decoupling damages from the harm suffered by the victim. [source] Induced hypothermia following out-of-hospital cardiac arrest; initial experience in a community hospitalCLINICAL CARDIOLOGY, Issue 12 2006Brook D. Scott M.D. Abstract Background Successful resuscitation from sudden cardiac death is frequently accompanied by severe and often fatal neurologic injury. Induced hypothermia (IH) may attenuate the neurologic damage observed in patients after cardiac arrest. Hypothesis This study examined a population of nonselected patients presenting to a community hospital following successful resuscitation of sudden cardiac death. We sought to determine whether a program of induced hypothermia would improve the clinical outcome of these critically ill patients. Methods We initiated a protocol of IH at the Oklahoma Heart Hospital in August of 2003. Study patients were consecutive adults admitted following successful resuscitation of out-of-hospital cardiac arrest. Moderate hypothermia was induced by surface cooling and maintained for 24 to 36 h in the Intensive Care Unit with passive rewarming over 8 h. Results Forty-nine patients who were resuscitated and had the return of spontaneous circulation completed the hypothermia protocol. The cause of cardiac arrest was acute myocardial infarction in 24 patients and cardiac arrhythmias in 19 patients. Nineteen patients (39%) survived and were discharged. Sixteen of the patients discharged had no or minimal residual neurologic dysfunction and 3 patients had clinically significant residual neurologic injury. Conclusion A program of induced hypothermia based in a community hospital is feasible, practical, and requires limited additional financial and nursing resources. Survival and neurologic recovery compare favorably with clinical trial outcomes. Copyright © 2006 Wiley Periodicals, Inc. Wiley Periodicals, Inc. [source] Prevention of sudden cardiac deathCLINICAL CARDIOLOGY, Issue S1 2005Eric N. Prystowsky M.D. Abstract It is often unclear why some patients suffer sudden cardiac death (SCD), or even what risk factors correlate best with the syndrome. This review describes current thinking on the prevention of SCD. Most studies have focused on the prevention of potentially fatal ventricular arrhythmias in patients post myocardial infarction (MI). While pharmacotherapy has a role in the prevention of SCD in patients post MI, the interpretation of drug trials can be problematic. This is because not all patients participating in such trials received optimized medical therapy by today's standards. As a result, trial outcomes for new therapies may not reflect their true efficacy when they are added to a background of best medical care. The two principal prophylactic modalities for SCD studied to date are antiarrhythmic drug therapy and use of an implantable cardioverter defibrillator (ICD). At the present time, antiarrhythmic drugs, such as the class III agent amiodarone, seem to display relatively limited efficacy for the primary prevention of sudden death in most patients post MI. Most clinical trials have found that ICD therapy has a significant mortality benefit in patients at high risk for ventricular arrhythmias. This has been demonstrated in primary prevention trials, and in secondary prevention trials such as Antiarrhythmics Versus Implantable Defibrillators (AVID), which studied patients who survived a near-fatal ventricular arrhythmia. Based on an analysis of secondary prevention trials, the single patient characteristic that best predicted an advantage of ICD therapy over antiarrhythmic drug therapy was a left ventricular (LV) ejection fraction , 35%. Cardiac resynchronization therapy has been established as having a mortality benefit in patients with dyssynchronous LV contraction associated with dilated cardiomyopathy. [source] | ||||||||||