Randomized clinical trials in medical rehabilitation research.

The purpose of this article is to provide researchers and clinicians with a basic understanding of randomized clinical trials and to discuss their potential application to and limitations in the field of physical medicine and rehabilitation. A brief history of the development of randomized clinical trials, definitions of clinical trials, types of trials, and overview of methodological issues related to design are offered. Information is provided about the need to establish clear and concise study objectives and to explicitly define interventions and expected outcomes. Recommendations for developing clinical protocols and determining adequate sample size are presented, and various statistical considerations, including power, are discussed. Issues related to sampling strategies, and recruitment are reviewed. Importance of randomization and blinding is emphasized. Readers are also referred to other resources available on this topic. Finally, the authors describe shortfalls associated with the use of this design in rehabilitation research. These are further explored and discussed in terms of the actual benefits and limitations of randomized clinical trials in physical medicine and rehabilitation research. Recommendations are made regarding the use of this methodology to address relevant needs in clinical practice.     

Strategies for evaluating clinical change: implications for practice and research

The methods and procedures of evaluation research using single-system or small-N designs are defined and described. Various components of single-system evaluation research, including specifying the problem, measuring and recording the data, selecting an appropriate design, and analyzing the data, are presented and discussed. The argument is made that single-system evaluation research methods are ideally suited to document clinical change on an individual basis and can provide a mechanism for establishing therapeutic accountability. The relationship between single-system evaluation research and the more traditional experimental procedures is briefly explored, and the implications for establishing an empirically derived basis for clinical practice are discussed.     

Barriers to rehabilitation research,and overcoming them

Although rehabilitation is thought to be underresearched, some data suggest that evaluative randomized controlled trials (RCTs) form a greater proportion of studies in rehabilitation research than in the general medical literature. Various obstacles to more evaluative research are sometimes put forward. These include suggestions that it is unethical, that the personalized nature of rehabilitation coupled with the unique problems of each patient make it impossible to study groups, that patients will not agree to enter trials, and that the funding mechanisms for both service and research are not available. In practice these arguments probably reflect low self-confidence within the profession. Education of the profession, public and purchasers should help in the long term. Meanwhile rehabilitation research would benefit from the development of stable research units of adequate size conjoined with clinical units.     

Severity Grading of Childhood Poisoning: The Multicentre Study of Poisoning in Children (MSPC) Score

The evidence base of clinical toxicology suffers in comparison to other clinical disciplines. There is an excess of case reports and case series with little in the way of case control or cohort studies, and very few randomized controlled trials. While randomized controlled trials are rightly regarded as the gold standard for interventional studies, they have limitations that are particularly evident in the practice of clinical toxicology. Properly conducted observational studies using quantitative, epidemiological methods [nonrandomized trials, cohort studies (prospective and retrospective), case control methods] can provide answers that may be impossible to obtain from randomized controlled trials. Development of a strong evidence base is essential for progress in clinical toxicology. Whether that evidence base is derived from randomized controlled trials or observational studies, it is essential to collect data. Important observations can be made from basic clinical data and systematic collection of those data into some form of electronic database has significant advantages. A clinical database provides accurate information in the areas of clinical practice, quality assurance (audit), and research. In the area of research, an appropriately designed database can be both a source of hypotheses as well as a vehicle to test them. It can also serve as a repository of research data in subsequent randomized controlled trials.     

Establishing recommendations for physical medicine and rehabilitation: the SOFMER methodology.

OBJECTIVE: The development of a rigorous methodology based on published results of clinical trials, evaluation of daily practice in France and multidisciplinary expert opinion to elaborate recommendations for rehabilitation interventions. METHODS AND RESULTS: The following describes the methodology of SOFMER (Société Fran?aise de Médecine Physique et de Réadaptation [French Society of Physical Medicine and Rehabilitation]) for developing recommendations for rehabilitation interventions. The test case was developing recommendations for rehabilitation in hip or knee osteoarthritis (OA) and hip or knee arthroplasty. Physicians in rehabilitation, orthopedic surgery and rheumatology identified, synthesized, and analyzed data from the literature by use of the usual French system of grading trials (the French Agency for Accreditation and Evaluation in Healthcare [ANAES] scale). The data were published results of comparative controlled studies such as randomized controlled trials, controlled clinical trials, cohort studies, case control studies, reviews of clinical trials, and case series, as well as uncontrolled cohort studies. The resulting recommendations were presented to the three annual French national congresses of rehabilitation, rheumatology, and orthopedic surgery for comment and for adapting to French professional practice. Finally, a panel of multidisciplinary experts (physicians in physical medicine and rehabilitation, rheumatologists, orthopedic surgeons, general practitioners, physical therapists, social workers, podologists, occupational therapists, nurses, and patients) validated the recommendations. CONCLUSION: The SOFMER methodology could be an interesting tool for use in developing recommendations elaborated by all the concerned medical and surgical specialists in the wide domain of rehabilitation.     

Ultrasound Elastography: The New Frontier in Direct Measurement of Muscle Stiffness

The use of brightness-mode ultrasound and Doppler ultrasound in physical medicine and rehabilitation has increased dramatically. The continuing evolution of ultrasound technology has also produced ultrasound elastography, a cutting-edge technology that can directly measure the mechanical properties of tissue, including muscle stiffness. Its real-time and direct measurements of muscle stiffness can aid the diagnosis and rehabilitation of acute musculoskeletal injuries and chronic myofascial pain. It can also help monitor outcomes of interventions affecting muscle in neuromuscular and musculoskeletal diseases, and it can better inform the functional prognosis. This technology has implications for even broader use of ultrasound in physical medicine and rehabilitation practice, but more knowledge about its uses and limitations is essential to its appropriate clinical implementation. In this review, we describe different ultrasound elastography techniques for studying muscle stiffness, including strain elastography, acoustic radiation force impulse imaging, and shear-wave elastography. We discuss the basic principles of these techniques, including the strengths and limitations of their measurement capabilities. We review the current muscle research, discuss physiatric clinical applications of these techniques, and note directions for future research.     

Lessons learned in participant recruitment and retention: the EXCITE trial

Participant recruitment is considered the most difficult aspect of the research process. Despite the integral role of recruitment in randomized clinical trials, publication of data defining the recruitment effort is not routine in rehabilitation initiatives. The recruitment process for the Extremity Constraint-Induced Therapy Evaluation (EXCITE) trial illustrates obstacles to and strategies for participant accrual and retention that are inherent in rehabilitation clinical trials. The purpose of this article is to increase awareness of the multiple facets of recruitment necessary for successful clinical trials, thus supporting the continued development of evidence-based practice in physical therapy. The Recruitment Index is presented as a variable to measure recruitment efficacy. In addition, ethical aspects of recruitment are explored, including informed consent and the concept of therapeutic misconception.     

A review of evidence-based practice, nursing research and reflection: levelling the hierarchy

Aim. This paper examines the evidence‐based practice movement, the hierarchy of evidence and the relationship between evidence‐based practice and reflective practice. Background. Evidence‐based practice is equated with effective decision making, with avoidance of habitual practice and with enhanced clinical performance. The hierarchy of evidence has promoted randomized control trials as the most valid source of evidence. However, this is problematic for practitioners as randomized control trials overlook certain types of knowledge that, through the process of reflection, provide useful information for individualized and effective practice. Method. A literature search was undertaken using CINAHL, medline and Ovid electronic databases in early 2006. The search terms used were: evidence‐based practice, research evidence, evidence for practice, qualitative research, reflective practice, reflection and evidence. Other sources included handpicking of books on evidence‐based practice, reflection and research. Only material written in English was included. Findings. The hierarchy of evidence that has promoted randomized control trials as the most valid form of evidence may actually impede the use of most effective treatment because of practical, political/ideological and epistemological contradictions and limitations. Furthermore, evidence‐based practice appears to share very similar definitions, aims and procedures with reflective practice. Hence, it appears that the evidence‐based practice movement may benefit much more from the use of reflection on practice, rather than the use of the hierarchical structure of evidence. Conclusion. Evidence‐based practice is necessary for nursing, but its’ effective implementation may be hindered by the hierarchy of evidence. Furthermore, evidence‐based practice and reflection are both processes that share very similar aims and procedures. Therefore, to enable the implementation of best evidence in practice, the hierarchy of evidence might need to be abandoned and reflection to become a core component of the evidence‐based practice movement. Relevance to clinical practice. Provides an elaborated analysis for clinical nurses on the definition and implementation of evidence in practice.     

Single subject research designs for disability research

Purpose : The objective of this article is to offer an overview of single subject designs and provide clinicians with information regarding the components of single subject designs and how they can be used in clinical and other rehabilitation environments. Method/Result : Three basic designs in single subject research are presented, with corresponding examples illustrating each design. A review of visual and statistical analysis techniques commonly used in single subject designs is provided, and the advantages and limitations of each are noted. Conclusion : Single subject designs are ideally suited for research in the rehabilitation practice environment. If properly applied, these designs can help establish the efficacy of rehabilitation practice and contribute to rehabilitation science.     

The clinical-economic trial: Promise,problems, and challenges

The clinical-economic trial is a study design that is appearing with greater frequency in medical and public health literature. Some experienced investigators view these trials with skepticism; to policy makers they represent a promising step in the control of rising health care costs. The success of clinical-economic trials in meeting the important goal of more rational and efficient use of health care resources will depend on the strengths and limitations of the research method. As part of a report to the Office of Technology Assessment of the U.S. Congress on new health care assessment techniques, we describe the reasons why economic data collection and analysis are being considered in clinical trials, identify and discuss various designs and methods for gathering economic trial data, and evaluate the strengths and limitations of different methods for providing sound data for decision making on appropriate use of health care interventions. Because of the potential significance and increasing visibility of such research, experts in research methods should give more attention to methodological research for clinical-economic trials. Future efforts should be directed at comparing different techniques for collecting data, examining the incremental value of precision in economic measurements and ensuring appropriate interpretation of data from clinical-economic trials.     

Survival analysis

Aims and objectives. This paper describes when and why survival analysis is used and describes the use and interpretation of the techniques most commonly encountered in medical literature. This is performed using examples taken from core medical journals. Background. Survival analysis is widely used in clinical and epidemiological research: in randomised clinical trials for comparing the efficacy of treatments and in observational (non-randomised) research to determine and test the existence of epidemiological association. Design. This paper introduces the principles, practice and terminology of survival analysis. Methods. References are made to examples from open-access medical journals. Results. Survival analysis is a well-established series of methodologies that are widely encountered in medical literature for both observational and randomised studies. Conclusions. Survival analysis represents a more efficient use of clinical data than other forms of analysis which rely on fixed time periods. One of the most widely used techniques is that developed by Kaplan and Meier. This involves the creation of life tables and the plotting of survival curves with comparison made between two or more groups. The log-rank test is commonly used to establish whether there is a statistically significant difference between these groups. The Multivariate Cox proportional hazards extend this approach to give an estimate of effect size (the Hazards Ratio) and can adjust for any potential confounding variables. In this model, the assumption of proportional hazards is of key importance and should always be checked. More advanced techniques are the use of time-dependent variables and the less widely used parametric survival techniques. Care should always be taken when considering the assumptions involved when using such methods. Relevance to clinical practice. As survival analysis is widely used in clinical research, it is important that readers can critically evaluate the use of this technique.     

The Time Is Now: A FASTER Approach to Generate Research Evidence for Technology-Based Interventions in the Field of Disability and Rehabilitation

Current approaches for generating high-quality research evidence for technology-based interventions in the field of disability and rehabilitation are inappropriate. Prevailing approaches often focus on randomized controlled trials as standard and apply clinical trial practices designed for pharmaceuticals; such approaches are unsuitable for technology-based interventions and are counterproductive to the goals of supporting people with disabilities and creating benefits for society. This communication is designed to: (1) advocate for the use of alternative approaches to generating evidence in the development and evaluation of technology-based interventions; (2) propose an alternative framework and guiding principles; and (3) stimulate action by multiple disciplines and sectors to discuss, adopt, and promote alternative approaches. Our Framework for Accelerated and Systematic Technology-based intervention development and Evaluation Research (FASTER) is informed by established innovation design processes, complex intervention development, evaluation, and implementation concepts as well as our collective experiences in technology-based interventions research and clinical rehabilitation practice. FASTER is intended to be meaningful, timely, and practical for researchers, technology developers, clinicians, and others who develop these interventions and seek evidence. We incorporate research methods and designs that better align with creating technology-based interventions and evidence for integration into practice. We propose future activities to improve the generation of research evidence, enable the selection of research methods and designs, and create standards for evidence evaluation to support rigor and applicability for technology-based interventions. With this communication we aim to improve and advance technology-based intervention integration from conception to use, thus responsibly accelerating innovation to have greater positive benefit for people and society.     

Single subject research designs for disability research

Purpose: The objective of this article is to offer an overview of single subject designs and provide clinicians with information regarding the components of single subject designs and how they can be used in clinical and other rehabilitation environments. Method/Result: Three basic designs in single subject research are presented, with corresponding examples illustrating each design. A review of visual and statistical analysis techniques commonly used in single subject designs is provided, and the advantages and limitations of each are noted. Conclusion: Single subject designs are ideally suited for research in the rehabilitation practice environment. If properly applied, these designs can help establish the efficacy of rehabilitation practice and contribute to rehabilitation science.     

Developing a rehabilitation databank for Germany--conceptional considerations exemplified by UDSMRSM (Uniform Datasystem of Medical Rehabilitation)]

Current research in medical rehabilitation lacks well designed clinical studies to demonstrate the efficacy, effectiveness and efficiency of rehabilitation interventions. Randomized controlled studies (RCTs) are difficult to conduct in rehabilitation research because of multiple variables and difficulties to include appropriate control groups. Therefore, we propose the development of a rehabilitation database and network for Germany to allow data collection from daily rehabilitation practice in the three areas medical-objective, perceptive-subjective and economical-social, designed after the example of the Uniform Datasystem of Medical Rehabilitation (UDSMRSM). This American database uses the Functional Independence Measure (FIM) to monitor functional changes in medical rehabilitation patients. Quarterly reports are sent out to participating institutions and allow comparisons with other regional, national and international institutions. The database demonstrates an initial step towards the concept of "evidence-based rehabilitation" by defining a common rehabilitation outcome and it allows benchmarking with other rehabilitation facilities. This permits the development of quality standards, cost analyses, payment systems and a new research methodology that can identify the most effective rehabilitation method through statistical analysis for focused clinical trials.     

A review of trazodone use in psychiatric and medical conditions

Trazodone is an antidepressant that is FDA-approved for the treatment of depression. It has been used by mental health and primary care providers for the treatment of multiple psychiatric and medical conditions .This review describes trazodone mechanism of action, formulation, dosage and adverse effects and then summarizes the beneficial effects of trazodone in the treatment of various psychiatric and medical conditions such as major depression, as well non-approved FDA indications such as insomnia,anxiety disorders, posttraumatic stress disorder, obsessive compulsive disorder, feeding and eating disorders, substance use disorders, behavioral disturbances associated with cognitive dysfunction, sexual dysfunction, certain pain conditions, and rehabilitation after acute ischemic stroke. Despite trazodone’s favorable effects in the treatment of FDA-unapproved psychiatric and medical conditions, large, randomized controlled clinical trials are still needed to confirm its efficacy in the treatment of the multiple conditions for which it is often used in clinical practice.     

急性心肌梗死PCI术后患者住院期运动康复的最佳证据总结

目的检索并获取急性心肌梗死PCI术后患者住院期运动康复的相关证据,并对最佳证据进行总结。方法应用循证护理的方法,使用计算机、手工相结合的形式检索国内外数据库有关急性心肌梗死PCI术后患者住院期运动康复的相关证据,包括指南、证据总结、临床实践指南、系统评价、随机对照试验等。检索时限为建库至2019年12月1日。由4组循证小组独立进行文献质量评价,有争议的内容由医院循证小组裁决,并提取证据。分别采用AGREEⅡ评分、澳大利亚JBI循证卫生保健中心文献评价标准、证据分级系统,对各类研究进行文献质量评价与等级划分。结果共纳入证据15篇,包括指南4篇、证据总结1篇、系统评价1篇、随机对照试验9篇,最终总结了20条急性心肌梗死PCI术后患者住院期运动康复的最佳证据。结论本研究归纳了关于急性心肌梗死PCI术后患者住院期运动康复的最佳证据,可以为临床开展运动康复提供循证依据,但在实际开展过程中,应结合患者文化背景与医疗机构水平,在保障患者安全的前提下循序渐进地开展。     

Applying evidence standards to rehabilitation research

OBJECTIVE: To describe evidence grading methods employed in the systematic reviews in this special series of articles. To provide an overview of results of these reviews to critique the quality of rehabilitation research. To identify issues in the application of evidence grading methods to rehabilitation. DESIGN: Conceptual review of evidence-based practice and evidence grading methods. English-language research studies on rehabilitation of persons with spinal cord injury, traumatic brain injury, and burn for the 5-yr period of 1999-2004 were reviewed using methods of the American Academy of Neurology supplemented by Cochrane criteria and summarized. RESULTS: Rehabilitation has a shortage of high-level studies. The number of level 1 treatment studies was quite limited (five in spinal cord injury, 15 in traumatic brain injury, 12 in burn rehabilitation), as was the number of level 2 studies (26, 4, and 2, respectively). Despite the large number of correlational studies published, the number of high-level (1 or 2) diagnostic and prognostic studies was surprisingly limited (34, 11, and 5, respectively). The rate of production of high-level studies is rapidly increasing. Problems were encountered in applying standard evidence criteria to complex issues encountered in some studies, suggesting limitations and issues in evidence grading methodology. CONCLUSIONS: Rehabilitation needs more high-level studies. Some improvements in research methodology are relatively affordable (e.g., improved blinding), whereas others are expensive (e.g., large randomized controlled trials). Lower-level investigations reporting promising results need to be followed by more definitive, higher-level trials.     

Systematic reviews in rehabilitation for stroke: issues and approaches to addressing them

OBJECTIVES: To describe the problems encountered when undertaking systematic reviews of rehabilitation and to give advice to reviewers about ways of addressing these issues. BACKGROUND: Evidence-based practice has become established as a way of linking clinical practice with research evidence. The most robust evidence is usually considered to be that from randomized controlled trials, and from systematic reviews of these trials. However, most reviews have focused on drug and surgical interventions, and few reviews have been produced in the field of rehabilitation. This may be due to the unique difficulties that are posed by reviews in this area. EXPERIENCE OF SYSTEMATIC REVIEWS IN APHASIA: We describe the challenges that occurred when carrying out systematic reviews of speech and language therapy for aphasia following stroke, and of ways to deliver rehabilitation services to people who have suffered a stroke. Issues arose at all stages of the reviews and included: using expertise; planning the review; defining the health care problem, patients, outcomes and interventions of interest; searching for, interpreting and synthesizing the results of the studies identified; and deriving implications from the review. Suggestions made to address these challenges include: developing a suitable team of reviewers; using inclusive and broad criteria for inclusion of trials to a review; developing appropriate search strategies; and using caution in arriving at conclusions. It is important to be explicit about decisions made during the review process concerning how to handle the challenges. Practical advice about carrying out reviews in the field of stroke rehabilitation is likely to be helpful to reviewers of other rehabilitative interventions. There are likely to be relatively few studies available for such reviews, and limitations in drawing specific practical conclusions. Such undertakings are, however, useful in setting the agenda for further research.     

Limitations of clinical trials in acute lung injury and acute respiratory distress syndrome

PURPOSE OF REVIEW: To review the challenges and limitations of randomized clinical trials in acute respiratory distress syndrome, with special emphasis on those pertaining to ventilatory management. RECENT FINDINGS: Superbly executed randomized trials of ventilatory strategy have garnered deserved attention from the critical care community and yet have illustrated the limitations of our current approach to clinical research in this area. Inexact definitions, incomplete mechanistic understanding of complex pathophysiology, inappropriate outcome variables, diverse therapeutic environments, lengthy data acquisition time and ethical constraints on trial design limit the applicability of randomized control trial methodology to acute respiratory distress syndrome and acute lung injury. As yet, clinical practice does not seem to have been greatly impacted by the implications of completed randomized controlled trials per se. Recent issues, both ethical and interpretive, regarding control group participants have raised troubling and theoretically important issues that are yet to be fully resolved. SUMMARY: Without tighter definitions of the condition under treatment, more specific targets for interventions to act upon, stratification that recognizes key interactive elements, and cointerventions based on better mechanistic understanding, randomized controlled trials of new drugs, ventilatory strategy, and other management approaches in acute respiratory distress syndrome are likely to remain a blunt instrument for investigation. As valuable as they are for calling important therapeutic principles to attention and for helping to suggest general guidelines for care, the limitations of randomized controlled trials for treating the individual with acute respiratory distress syndrome must be acknowledged.