京都大学 / 0048 / 新制・課程博士 / 博士(人間健康科学) / 甲第21705号 / 人健博第71号 / 新制||人健||5(附属図書館) / 京都大学大学院医学研究科人間健康科学系専攻 / (主査)教授 市橋 則明, 教授 黒木 裕士, 教授 妻木 範行 / 学位規則第4条第1項該当 / Doctor of Human Health Sciences / Kyoto University / DFAM

Objectives: To determine the subacute effects of cervicothoracic spinal thrust/non-thrust in addition to shoulder non-thrust plus exercise in patients with subacromial pathology. Methods: This was a randomized, single blinded controlled trial pilot study. This trial was registered at ClinicalTrials.gov (NCT01753271) and reported according to Consolidated Standards of Reporting Trials requirements. Patients were randomly assigned to either shoulder treatment plus cervicothoracic spinal thrust/non-thrust or shoulder treatment-only group. Primary outcomes were average pain intensity (Numeric Pain Rating Scale) and physical function (Shoulder Pain and Disability Index) at 2 weeks, 4 weeks, and patient discharge. Results: 18 patients, mean age 43.1(15.8) years satisfied the eligibility criteria and were analyzed for follow-up data. Both groups showed statistically significant improvements in both pain and function at 2 weeks, 4 weeks, and discharge. The between-group differences for changes in pain or physical function were not significant at any time point. Discussion: The addition of cervicothoracic spinal thrust/non-thrust to the shoulder treatment-only group did not significantly alter improvement in pain or function in patients with subacromial pathology. Both approaches appeared to provide an equally notable benefit. Both groups improved on all outcomes and met the criteria for clinical relevance for both pain and function. Level of Evidence: 2b.

This paper discusses results of a computer simulation to investigate the effect of jittering to simulate measurement error. In addition, the classical F ratio, the bootstrap F and the F for ranked data are compared. Empirical powers and p-values suggest the bootstrap is a good and robust procedure and the rank procedure seems to be too liberal when compared to the classical F ratio.

Indiana University-Purdue University Indianapolis (IUPUI) / Cluster randomization is frequently used in clinical trials for convenience of inter ventional implementation and for reducing the risk of contamination. The opera tional convenience of cluster randomized trials, however, is gained at the expense of reduced analytical power. Compared to individually randomized studies, cluster randomized trials often have a much-reduced power. In this dissertation, I consider ways of enhancing analytical power with historical trial data. Specifically, I introduce a hierarchical Bayesian model that is designed to incorporate available information from previous trials of the same or similar interventions. Operationally, the amount of information gained from the previous trials is determined by a Kullback-Leibler divergence measure that quantifies the similarity, or lack thereof, between the histor ical and current trial data. More weight is given to the historical data if they more closely resemble the current trial data. Along this line, I examine the Type I error rates and analytical power associated with the proposed method, in comparison with the existing methods without utilizing the ancillary historical information. Similarly, to design a cluster randomized trial, one could estimate the power by simulating trial data and comparing them with the historical data from the published studies. Data analytical and power simulation methods are developed for more general situations of cluster randomized trials, with multiple arms and multiple types of data following the exponential family of distributions. An R package is developed for practical use of the methods in data analysis and trial design.

京都大学 / 0048 / 新制・課程博士 / 博士(人間健康科学) / 甲第18908号 / 人健博第22号 / 新制||人健||2(附属図書館) / 31859 / 京都大学大学院医学研究科人間健康科学系専攻 / (主査)教授 菅沼 信彦, 教授 鈴木 眞知子, 教授 中原 俊隆 / 学位規則第4条第1項該当 / Doctor of Human Health Sciences / Kyoto University / DFAM

Patient-reported outcomes (PROs) produce meaningful information about patient-perceived health status reported directly by patients. Routine collection of PROs data is particularly important in chronic conditions, such as heart failure (HF). Major cardiovascular societies and regulatory agencies encouraged PRO inclusion in randomized controlled trials (RCTs), but PROs remain underutilized as a key outcome in these studies. In this systematic review, we aimed to evaluate temporal trends and explore trial characteristics associated with PRO inclusion in HF RCTs published in high-impact medical journals. We also assessed the quality of PRO reporting against the Consolidated Standards of Reporting Trials PRO extension. We found that over half of HF RCTs included a PRO. The proportion of RCTs with PROs increased significantly since 2000. A number of RCT characteristics such as multicentre; medium-sized (n = 51-250 participants); trials coordinated in Central and South America; and that tested health services, devices or surgery, exercise and rehabilitation interventions were independently associated with higher odds of PRO inclusion. The quality of PRO reporting was modest, with better reporting in RCTs with PROs a primary or co-primary endpoint. Consistent PRO inclusion and high-quality reporting are necessary to increase the utility of these findings by patients, clinicians, and health care policy makers. / Thesis / Master of Science (MSc)

No description available.

Background and Objectives: Missing participant data are likely to bias the results of randomized control trials (RCTs) when the reason for missingness is associated with status on the outcome of interest. Unlike dichotomous MPD in RCTs, which have been thoroughly investigated, knowledge regarding continuous MPD in RCTs is much more limited. Our objectives were 1) using an adapted checklist, to assess the reporting quality of simulation studies comparing methods to deal with continuous MPD; 2) identify optimal methods proposed by biostatisticians and tested in simulations studies for continuous MPD in RCTs; 3) evaluate how authors report MPD, and how they plan and conduct analyses to deal with MPD in RCTs. Methods: We conducted two systematic surveys. The first identified methods papers published till 2015 January that compared statistical approaches to deal with continuous MPD in RCTs using at least one simulation. In this sample, we considered both the quality of reporting and the results. The second survey identified a representative sample of individual RCTs published in 2014 in core journals reporting the results of at least one continuous variable addressing a patient-important outcome. Results and conclusion: Our survey identified important limitations in reporting quality of simulation studies that compared statistical approaches to deal with continuous MPD, particularly in the reporting of simulation procedures. Only one of 60 studies reported the random number generator used and none reported starting seeds or failures during simulation. Less then half reported software used to perform simulation (41.7%) or analysis (48.3%), and only 4 (5%) reported justification of number of simulations. When facing continuous MPD in RCTs, results of simulation studies demonstrate that trialists seeking optimal approaches may choose robust regression or mixed models and avoid using last observation caring forward. Continuous MPD frequently occurs in RCTs and the extent is typically substantial (median greater than 10%). Methods sections in trial reports typically do not provide adequate detail on how they dealt with MPD in their primary analysis. Among methods actually implemented to deal with MPD, most authors use only available data, thus excluding MPD from the analysis. Seldom do investigators apply statistical approaches to impute or taking into account of MPD nor conduct sensitivity analysis to address the impact of it. A comprehensive knowledge synthesis summarizing current available statistical approaches and its relative merits, as well as the current used methods in RCTs provide clear implications on how the practise of using methods to handle continuous MPD should shift in individual RCTs. Trialists should use mixed models and robust regressions and avoid using last observation caring forward method. / Thesis / Doctor of Philosophy (PhD)

Importance: Evidence from randomized controlled trials (RCTs) is required to guide treatment of critically ill children. Unfortunately such evidence is not always available. Objectives: To describe the RCT research enterprise in pediatric critical care — the evidence and the process of creating it, along with problems and some solutions. Methods: To meet these objectives I undertook a series of 5 related studies. First a scoping review to describe the output of the research enterprise. Second, a social network analysis of coauthorship patterns to describe the community of researchers who produce this evidence. Third, a survey to investigate the importance of RCTs in clinicians’ decision-making. Fourth, a survey of trialists to identify barriers and facilitators of high quality RCTs. Fifth, a qualitative interview study to identify acceptable, feasible and effective strategies to improve the evidence available from RCTs in pediatric critical care. Results and conclusions: The number of RCTs in pediatric critical care is increasing but there is a preponderance of small, single-centred RCTs focusing on laboratory or physiological outcomes that are often stopped early because of feasibility problems or futility. The research community is highly fragmented and highly clustered. Experienced trialists identified approaches to improve the pediatric critical care research enterprise, including building a sense of community and ensuring key training and relevant practical experiences for new investigators. Because of the barriers that researchers face and their ethical obligation to undertake trials that are feasible and make a meaningful contribution to advancing the care of critically ill children, individuals and groups must take an active role in building a healthy research community. Only by changing how we function as a research community can we train the next generation of investigators and undertake the type of trials needed to improve the care of critically ill children. / Thesis / Doctor of Philosophy (PhD) / Evidence from randomized controlled trials (RCTs) is required to guide treatment of critically ill children. Unfortunately such evidence is not always available. My objectives in this thesis are to describe the RCT research enterprise in pediatric critical care — the evidence and the process of creating it, along with problems and some solutions. To meet these objectives I undertook a series of 5 related studies: to identify and describe the RCTs, describe how researchers collaborate, understand how clinicians use RCTs, identify barriers and facilitators of conducting high quality RCTs, and understand how we can improve the evidence available from RCTs in pediatric critical care. We found that the number of RCTs is increasing but there are opportunities to improve the methods, outcome measures, and quality of reporting. We identified strategies that researchers can adopt to facilitate the rigorous RCTs that are needed to improve the care of critically ill children.

No description available.