Patient vs. Community Engagement: Emerging Issues.

BACKGROUND: The value proposition of including patients at each step of the research process is that patient perspectives and preferences can have a positive impact on both the science and the outcomes of comparative effectiveness research. How to accomplish engagement and the extent to which approaches to community engagement inform strategies for effective patient engagement need to be examined to address conducting and accelerating comparative effectiveness research. OBJECTIVES: To examine how various perspectives and diverse training lead investigators and patients to conflicting positions on how best to advance patient engagement. RESEARCH DESIGN: Qualitative methods were used to collect perspectives and models of engagement from a diverse group of patients, researchers and clinicians. The project culminated with a workshop involving these stakeholders. The workshop used a novel approach, combining World Café and Future Search techniques, to compare and contrast aspects of patient engagement and community engagement. SUBJECTS: Participants included patients, researchers, and clinicians. MEASURES: Group and workshop discussions provided the consensus on topics related to patient and community engagement. RESULTS: Participants developed and refined a framework that compares and contrasts features associated with patient and community engagement. CONCLUSIONS: Although patient and community engagement may share a similar approach to engagement based on trust and mutual benefit, there may be distinctive aspects that require a unique lexicon, strategies, tactics, and activities.

Gene Expression, and Fatigue in Puerto Rican Men during Radiotherapy for Prostate Cancer: an Exploratory Study.

OBJECTIVE: To examine the trajectory of fatigue experienced by 26 Puerto Rican (PR) men over the course of External Beam Radiation Therapy (EBRT) and to assess gene expression changes from baseline to midpoint of EBRT using microarray technology. Design/Research Approach- Prospective exploratory and comparative design study. Setting- RT facility located in San Juan, PR. Sample/Participants-26 PR men with non-metastatic prostate cancer. METHODS: Participants completed 2 paper forms: demographics and the Spanish version of the 13-item FACT-fatigue at baseline, midpoint, and end of EBRT. Wholeblood samples were collected at baseline and at midpoint of EBRT. Descriptive data was analyzed using t-test, Wilcoxon, and Friedman test for repeated measures. Gene expression data was analyzed using the LIMMA package in R; the functional network analysis was conducted using Ingenuity Pathway analysis. Main Research Variable-Fatigue scores, gene expression. RESULTS: Subjects were of ages 52-81 with fatigue scores that remained unchanged during EBRT (baseline=42.38, SD=9.34; midpoint=42.11, SD=8.93, endpoint=43.04, SD=8.62). Three hundred seventy-three genes (130-up regulated and 243-down regulated) were differentially expressed from baseline to mid-point of EBRT (FDR<0.01). The top distinct canonical pathways of the differentially expressed probesets (p<0.0001) were: "Phospholipase C Signaling," "Role of NFAT in Regulation of the Immune Response," and "Gαq Signaling." CONCLUSION: While fatigue did not worsen over the course of EBRT for this sample as a group, there was variability in fatigue across the sample. It is possible that the over expression of the SESN3 gene, known to suppress oxidative damage, may have contributed to the attenuation of fatigue in this clinical population.

Exercise for everyone: a randomized controlled trial of project workout on wheels in promoting exercise among wheelchair users.

OBJECTIVE: To compare the effectiveness of 2 home-based behavioral interventions for wheelchair users to promote exercise adoption and maintenance over 12 months. DESIGN: Randomized controlled trial, with participants stratified into groups based on disability type (stable, episodic, progressive) and support partner availability. SETTING: Exercise occurred in participant-preferred locations (eg, home, recreation center), with physiological data collected at a university-based exercise laboratory. PARTICIPANTS: Inactive wheelchair users (N=128; 64 women) with sufficient upper arm mobility for arm-based exercise were enrolled. Participants on average were 45 years of age and lived with their impairment for 22 years, with spinal cord injury (46.1%) most commonly reported as causing mobility impairment. INTERVENTIONS: Both groups received home-based exercise interventions. The staff-supported group (n=69) received intensive exercise support, while the self-guided group (n=59) received minimal support. Both received exercise information, resistance bands, instructions to self-monitor exercise, regularly scheduled phone calls, and handwritten cards. MAIN OUTCOME MEASURES: The primary outcome derived from weekly self-reported exercise. Secondary outcomes included physical fitness (aerobic/muscular) and predictors of exercise participation. RESULTS: The staff-supported group reported significantly greater exercise (∼17min/wk) than the self-guided group over the year (t=10.6, P=.00), with no significant between-group difference in aerobic capacity (t=.76, P=.45) and strength (t=1.5, P=.14). CONCLUSIONS: Although the staff-supported group reported only moderately more exercise, the difference is potentially clinically significant because they also exercised more frequently. The staff-supported approach holds promise for encouraging exercise among wheelchair users, yet additional support may be necessary to achieve more exercise to meet national recommendations.

Truth be told: evidence of wheelchair users' accuracy in reporting their height and weight.

OBJECTIVES: To examine whether wheelchair users' self-reports of height and weight differed significantly from direct measurements and whether weight category classifications differed substantially when based on self-reported or measured values. DESIGN: Single group, cross-sectional analysis. Analyses included paired t tests, chi-square test, analysis of variance, and Bland-Altman agreement analyses. SETTING: A university-based exercise lab. PARTICIPANTS: Community-dwelling wheelchair users (N=125). INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURE: Participants' self-reported and measured height, weight, and body mass index. RESULTS: Paired t tests revealed that there were significant differences between wheelchair users' self-reported and measured values for height (difference of 3.1±7.6cm [1.2±3.0in]), weight (-1.7±6.5kg [-3.6±14.2lb]), and BMI (-1.6±3.3). These discrepancies also led to substantial misclassification into weight categories, with reliance on self-reported BMI underestimating the weight status of 20% of the sample. CONCLUSIONS: Our findings suggest that similar to the general population, wheelchair users are prone to errors when reporting their height and weight and that these errors may exceed those noted in the general population.

Bayesian data analysis: estimating the efficacy of T'ai Chi as a case study.

BACKGROUND: Bayesian inference provides a formal framework for updating knowledge by combining prior knowledge with current data. Over the past 10 years, the Bayesian paradigm has become a popular analytic tool in health research. Although the nursing literature contains examples of Bayes' theorem applications to clinical decision making, it lacks an adequate introduction to Bayesian data analysis. METHODS: Bayesian data analysis is introduced through a fully Bayesian model for determining the efficacy of tai chi as an illustrative example. The mechanics of using Bayesian models to combine prior knowledge, or data from previous studies, with observed data from a current study are discussed. RESULTS: The primary outcome in the illustrative example was physical function. Three prior probability distributions (priors) were generated for physical function using data from a similar study found in the literature. Each prior was combined with the likelihood from observed data in the current study to obtain a posterior probability distribution. In each case, the posterior distribution showed that the probability that the control group is better than the tai chi treatment group was low. DISCUSSION: Bayesian analysis is a valid technique that allows the researcher to manage varying amounts of data appropriately. As advancements in computer software continue, Bayesian techniques will become more accessible. Researchers must educate themselves on applications for Bayesian inference, as well as its methods and implications for future research.

A Bayesian comparative effectiveness trial in action: developing a platform for multisite study adaptive randomization.

BACKGROUND: In the last few decades, the number of trials using Bayesian methods has grown rapidly. Publications prior to 1990 included only three clinical trials that used Bayesian methods, but that number quickly jumped to 19 in the 1990s and to 99 from 2000 to 2012. While this literature provides many examples of Bayesian Adaptive Designs (BAD), none of the papers that are available walks the reader through the detailed process of conducting a BAD. This paper fills that gap by describing the BAD process used for one comparative effectiveness trial (Patient Assisted Intervention for Neuropathy: Comparison of Treatment in Real Life Situations) that can be generalized for use by others. A BAD was chosen with efficiency in mind. Response-adaptive randomization allows the potential for substantially smaller sample sizes, and can provide faster conclusions about which treatment or treatments are most effective. An Internet-based electronic data capture tool, which features a randomization module, facilitated data capture across study sites and an in-house computation software program was developed to implement the response-adaptive randomization. RESULTS: A process for adapting randomization with minimal interruption to study sites was developed. A new randomization table can be generated quickly and can be seamlessly integrated in the data capture tool with minimal interruption to study sites. CONCLUSION: This manuscript is the first to detail the technical process used to evaluate a multisite comparative effectiveness trial using adaptive randomization. An important opportunity for the application of Bayesian trials is in comparative effectiveness trials. The specific case study presented in this paper can be used as a model for conducting future clinical trials using a combination of statistical software and a web-based application. TRIAL REGISTRATION: ClinicalTrials.gov Identifier: NCT02260388 , registered on 6 October 2014.

Smoking cessation strategy use among pregnant ex-smokers.

There is strong evidence for the beneficial health effects associated with smoking cessation during pregnancy. Although many pregnant women spontaneously quit smoking during pregnancy, postpartum relapse is high. Evidence suggests that pregnant women do not use smoking cessation strategies as identified by the 40-item Processes of Change Scale as frequently as others who quit smoking. The purpose of this study is to identify factors associated with pregnant exsmokers' use of experiential and behavioral smoking cessation strategies. A cross-sectional survey design is used for the study. From a sample of 201 primarily low-income pregnant women recruited to participate in a larger study, 58 were biologically confirmed ex-smokers. Multiple regression analysis reveals that motivation to quit smoking is the only significant factor explaining the use of experiential and behavioral smoking cessation strategies, accounting for 44% of the variance in experiential processes and 31% of the variance in behavioral processes.

A qualitative investigation of fatigue among healthy working adults.

Despite considerable attention to fatigue in acute and chronic illnesses, little is known about fatigue in a healthy population. A detailed exploration of fatigue, therefore, was conducted to answer the broad question of what fatigue is to a basically healthy population and to lay the groundwork for establishing an empirically based definition of fatigue. Qualitative and quantitative methods were used. Results of the qualitative phase of the study, in which 40 persons were interviewed, are presented here. Categories and themes of the experience of fatigue were identified. Based on the qualitative findings, we propose that fatigue in generally healthy adults is an acute, subjective, sometimes overwhelming, but temporary state (with physical, emotional, and behavioral manifestations) caused by stress and overwork in one's life roles, which disrupts activity and alerts the person to take restorative measures.

Quality of life in hospice patients with terminal illness.

To better understand quality of life (QOL) and its important correlates among patients with terminal illness, a cross-sectional correlational design was used in a study based on Stewart, Teno, Patrick, and Lynn's conceptual model of factors affecting QOL of dying patients and their families. Sixty participants were recruited from two local hospice programs in the midwestern region of the United States. Data were collected at the participants' homes. The participants had an above average QOL. Living with the caregiver, spirituality, pain intensity, physical performance status, and social support as a set explained 38% of the variance in their QOL. Among these five predictors, living with the caregiver, spirituality, and social support statistically were significant predictors of the QOL of these participants. Participants who did not live with their caregivers experienced less pain intensity, perceived higher spirituality, had more social support, and had a significantly better QOL. Important contributions of these findings are discussed.

The Greater Plains Collaborative: a PCORnet Clinical Research Data Network.

The Greater Plains Collaborative (GPC) is composed of 10 leading medical centers repurposing the research programs and informatics infrastructures developed through Clinical and Translational Science Award initiatives. Partners are the University of Kansas Medical Center, Children's Mercy Hospital, University of Iowa Healthcare, the University of Wisconsin-Madison, the Medical College of Wisconsin and Marshfield Clinic, the University of Minnesota Academic Health Center, the University of Nebraska Medical Center, the University of Texas Health Sciences Center at San Antonio, and the University of Texas Southwestern Medical Center. The GPC network brings together a diverse population of 10 million people across 1300 miles covering seven states with a combined area of 679 159 square miles. Using input from community members, breast cancer was selected as a focus for cohort building activities. In addition to a high-prevalence disorder, we also selected a rare disease, amyotrophic lateral sclerosis.

An exercise trial for wheelchair users: project workout on wheels.

There is growing interest in promoting health for people with disabilities, yet evidence regarding community-based interventions is sparse. This paper describes the design details of a randomized controlled trial (RCT) that will test the effectiveness of a multi-component behaviorally based, intervention to promote exercise adoption (over 6 months) and maintenance (up to one year) among wheelchair users and includes descriptive data on participant characteristics at baseline. Participants were randomly assigned to either a staff-supported intervention group or a self-guided comparison group. The primary study aim is to assess the effectiveness of the multi-component behaviorally based intervention for promoting physical activity adoption and maintenance. The RCT will also assess the physical and psychosocial effects of the intervention and the complex interplay of factors that influence the effectiveness of the intervention. Therefore, the primary outcome derives from participant reports of weekly exercise (type, frequency, duration) over 52 weeks. Secondary outcomes collected on four occasions (baseline, 3 months, 6 months, 12 months) included physiological outcomes (VO(2) peak, strength), disability-related outcomes (pain, fatigue, participation), and psychosocial outcomes (exercise self-efficacy, exercise barriers, quality of life, depression, mood). This study will provide evidence regarding the effectiveness of a multi-component behaviorally based intervention for promoting exercise adoption among people with mobility impairments that necessitate wheelchair use.

Preparedness of the CTSA's structural and scientific assets to support the mission of the National Center for Advancing Translational Sciences (NCATS).

The formation of the National Center for Advancing Translational Sciences (NCATS) brings new promise for moving basic science discoveries to clinical practice, ultimately improving the health of the nation. The Clinical and Translational Science Award (CTSA) sites, now housed with NCATS, are organized and prepared to support in this endeavor. The CTSAs provide a foundation for capitalizing on such promise through provision of a disease-agnostic infrastructure devoted to clinical and translational (C&T) science, maintenance of training programs designed for C&T investigators of the future, by incentivizing institutional reorganization and by cultivating institutional support.