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This is a short tutorial on two key questions that pertain to cluster randomized trials (CRTs): 1) Should I perform a CRT? and 2) If so, how do I derive the sample size? In summary, a CRT is the best option when you "must" (e.g., the intervention can only be administered to a group) or you "should" (e.g., because of issues such as feasibility and contamination). CRTs are less statistically efficient and usually more logistically complex than individually randomized trials, and so reviewing the rationale for their use is critical. The most straightforward approach to the sample size calculation is to first perform the calculation as if the design were randomized at the level of the patient and then to inflate this sample size by multiplying by the "design effect", which quantifies the degree to which responses within a cluster are similar to one another. Although trials with large numbers of small clusters are more statistically efficient than those with a few large clusters, trials with large clusters can be more feasible. Also, if results are to be compared across individual sites, then sufficient sample size will be required to attain adequate precision within each site. Sample size calculations should include sensitivity analyses, as inputs from the literature can lack precision. Collaborating with a statistician is essential. To illustrate these points, we describe an ongoing CRT testing a mobile-based app to systematically engage families of intensive care unit patients and help intensive care unit clinicians deliver needs-targeted palliative care. [ABSTRACT FROM AUTHOR]

We consider four memes, correct within the context of randomized trials but requiring modification for the analysis of the observational databases typically associated with learning health systems: (a) the right answer always requires randomization; (b) a bigger database is always a better database; (c) statistical adjustment always works if based on a large enough database; and (d) always make a formal adjustment when testing multiple hypotheses. The rationale for these memes within the context of randomized trials is discussed, and the memes are restated in a fashion that is consistent with learning health systems. [ABSTRACT FROM AUTHOR]

Approximately 38% of patients with colorectal cancer will develop isolated liver metastases. Sidedness of colon tumor is identified in non-metastatic and unresected metastatic cancers as predictive of survival, yet its dedicated analysis in resected liver metastases is minimal. Our primary aim was to assess whether left-sided primary tumors improve prognosis in stage IV cancer patients undergoing curative-intent liver metastasectomy; it was hypothesized that it would. This is a retrospective, observational cohort study from 1996 to 2016 in a single tertiary-care facility. Survival from diagnosis was calculated via Kaplan–Meier method and compared between the right and left sides via log-rank analysis. Median survival differs significantly between colorectal tumors of the right and left origins after hepatic metastasectomy in 612 patients. In patients with right-sided tumors, median survival from diagnosis was 4.5 years (IQR 4.1–5.3), and 6.3 years (IQR 5.6–6.9) in those with left tumors (HR 1.5, 95% CI 1.38–1.60, p < 0.001). As in studies on earlier-stage or unresected metastatic disease, tumor sidedness is an important prognostic factor in patient survival with liver metastasectomy. Clinical risk scores should include side of primary tumor. Further work is needed to determine the molecular basis for this difference. [ABSTRACT FROM AUTHOR]

Objective:To investigate sexual orientation differences in college men's motivations for HIV testing.Participants:665 male college students in the Southeastern United States from 2006 to 2014.Methods: Students completed a survey on HIV risk factors and testing motivations. Logistic regressions were conducted to determine the differences between heterosexual men (HM) and sexual minority men (SMM).Results:SMM were more motivated to get tested by concern over past condomless sex, while HM were more often cited supporting the testing program “on principle” and wanting a free t-shirt. SMM and HM differed in behaviors that impact HIV risk and other demographics. However, differences in testing motivation by concern over past condomless sex or wanting a free t-shirt persisted when controlling for these demographic and behavioral differences.Conclusions: Appropriately designed HIV prevention interventions on college campuses should target SMM's distinct concern over past condomless sex as a testing motivation. [ABSTRACT FROM PUBLISHER]

In a widely cited article, Ioannidis argued that most published research findings are false; particularly discovery research involving massive testing, genomics being a typical example. However, his argument ignores adjustment for multiple testing and thus should be taken with a large grain of salt. This is a potential example for statistics courses that concentrate on problem formulation. [ABSTRACT FROM AUTHOR]

Objective.—Use a presurvey of primary care providers (PCPs) enrolled in a continuing medical education (CME) program on headache management to ascertain their existing knowledge, attitudes, and beliefs regarding migraine and use a postsurvey to determine the extent to which the CME program has brought participant knowledge, attitudes, and skills closer to conformance with best evidence. Background.—Migraine is a common and debilitating condition, which PCPs may not always manage satisfactorily. In an effort to improve management, the American Headache Society has developed a CME program called BRAINSTORM that encourages PCPs to adopt the US Headache Consortium Guidelines for headache care. Methods.—A 20-item questionnaire was developed that covered the essential elements of migraine care. The questionnaire was administered before and after a BRAINSTORM presentation to 254 consenting primary care clinicians attending a medical meeting at 1 of 6 sites. A control group of 112 comparable physicians who did not attend the presentation completed the same questionnaire. Prepresentation scores of attendees were compared to scores of nonattendees to assess the generalizability of results. Prepresentation scores on selected questions were used to assess participant baseline knowledge, attitudes, and beliefs. Pre- and postpresentation scores for attendees at all sites were compared using the Mantel-Haenszel statistic to assess the effectiveness of the BRAINSTORM CME. Pre- and postpresentation scores were compared by site using the Breslow-Day test to evaluate any differential impact based on CME location. Results.—Prepresentation scores of attendees and nonattendees were found to be similar. No significant difference in performance was noted across sites. A chi-square analysis revealed a statistically significant difference between pre- and postpresentation scores for 16 of the test's 20 questions. In the pretest, all participants scored <66% on 2 questions related to prevalence, impact, and pathophysiology of migraine, 2 questions pertaining to history taking/physical examination, and 3 migraine management questions. Attendee scores improved to >66% posttest on all except 2 questions related to prevalence, impact, and pathophysiology of migraine. Conclusion.—Our results indicate that PCPs need to acquire greater understanding about the epidemiology and pathophysiology of migraine and may require guidance in history taking and physical examination of migraine patients. Improvement in scores posttest confirms that the BRAINSTORM program has a significant immediate impact on the knowledge, beliefs, and attitudes of participants. The program could be strengthened to improve emphasis in some areas where posttest scores showed no improvement. [ABSTRACT FROM AUTHOR]

This article cites findings of a cost-minimization randomized clinical analysis, comparing azithromycin-based and levofloxacin-based protocols for the treatment of patients who were hospitalized with community-acquired pneumonia (CAP). A randomized trial was completed for treating moderately to severely ill patients hospitalized with CAP. The azithromycin protocol specified IV azithromycin plus IV ceftriaxone for 2 to 5 days followed by oral azithromycin to complete a total of 7 to 10 days. The levofloxacin protocol specified IV levofloxacin for a minimum of 2 days followed by oral levofloxacin to complete a total of 7 to 14 days of therapy. A total of 110 patients in the azithromycin group and 102 patients in the levofloxacin group were used in the trial. The study findings revealed that cost is a significant consideration in determining the most appropriate course of action for any individual patient.

Which of the available antiplatelet therapies should be preferred for secondary prevention of recurrent ischemic stroke has been contentious. Objective: We applied the Duke Stroke Policy Model (DSPM) to reconsider this issue, paying particular attention to the degree of uncertainty in the estimates of their efficacy. The DSPM is a continuous-time simulation model of stroke development and outcome. Methods: We modified the inputs to reflect the cost of the drugs aspirin (ASA), extended release dipyridamole/aspirin (DP/A) and clopidogrel (CLO), as well as their relative risk in preventing subsequent ischemic stroke in comparison with placebo (PBO). These relative risks were derived from published reports from the second European Stroke Prevention Study (ESPS-2) and Clopidogrel Versus Aspiring in Patients at Risk of Ischemic Events studies. Precision was addressed by applying bootstrapping to the above estimates of relative risk. The target population was 70-year-old men with nondisabling stroke. The outcome measures were quality-adjusted life-years (QALYs), costs, and costs per QALY. Results: Results of Base Case Analysis: In large part because of its modest drug cost, ASA was cost-effective in comparison with PBO. DP/A tended to have improved outcomes, but at increased costs. CLO was dominated in the base case. Results of sensitivity analysis: ASA and DP/A cannot be differentiated on a statistical basis alone. In probabilistic sensitivity analysis, CLO was rarely preferred. Conclusions: Either DP/A or ASA appear to be a good value in comparison with no treatment, but there is no clear winner between the two. In the absence of a definitive randomized trial, simulation modeling can help clarify the trade-offs between the various antiplatelet agents, but not beyond the constraints imposed by the imprecision in the estimates that can be obtained from the current evidence base. [ABSTRACT FROM AUTHOR]

Objective.—To assess the current level of headache burden and the headache management needs at three diverse clinical sites. Background.—Headache is a common disabling disorder that is costly for the patient and a management challenge for physicians. The determination of whether and how to intervene to improve headache management depends on both the burden of disease and the characteristics of patients that would likely be targeted. Methods.—Patients from three healthcare organizations were identified by administrative records as having either migraine or tension-type headache and then mailed a survey that addressed demographics, headache type, headache-related disability, depression and anxiety, satisfaction with care, general health, worry about headache, problems with headache management, and healthcare utilization. Comparisons were made across sites and between patients with more and less severe headache-related impairments. Results.—Of the 789 patients contacted, 385 (50%) returned a survey. While the socio-demographic characteristics of the patients were diverse, headache-related characteristics were similar. These patients have significant problems with headache management, disability, pain, worry, and dissatisfaction with care. Patients who described higher headache-related impairment experienced significantly greater problems in these areas, perceived themselves to be in worse general health, and had significantly greater use of medical services than those with lower headache severity. Conclusions.—Despite various elements of heterogeneity, we observed across the sites a consistent need for improvement in headache management. Future efforts should be directed at developing and evaluating methods for effectively improving headache management. ( Headache 2005;45:1048-1055) [ABSTRACT FROM AUTHOR]

The quantitative relationship between functional status and self-reported quality of life is relatively unexamined. As part of the 1-, 6-, and 12-month telephone follow-up of consecutive patients in an observational study of patients with stroke, we found that while higher functional status was associated with better quality of life, this relationship was relatively weak (Spearman correlation <0.25). Patients with similar levels of disability reported quite different qualities of life. Any improvement in quality of life over time was modest at best. Mean utilities for patients with minor stroke were near 0.80, while those for patients with major stroke were near 0.60, the latter figure exceeding previous reports. Quality of life with major stroke may not necessarily be as low as that reported before such a stroke occurs. Quality of life after stroke is heterogeneous and depends on more than just level of physical function. [ABSTRACT FROM AUTHOR]

: BackgroundInaccuracy in self-reports of colorectal cancer (CRC) screening procedures (e.g., over- or underreporting) may interfere with individuals adhering to appropriate screening intervals, and can blur the true effects of physician recommendations to screen and the effects of interventions designed to promote screening. We assessed accuracy of self-report of having a fecal occult blood test (FOBT) within a 1-year window based on receipt of FOBT kits among individuals aged 50 and older in the carpentry trade (N = 658) who were off-schedule for having had a FOBT.: MethodIndices of evaluating accuracy of self-reports (concordance, specificity, false-positive and false-negative rates) were calculated relative to receipt of a mailed FOBT. Among those who mailed a completed FOBT, we assessed accuracy of reporting the test result.: ResultsParticipants underestimated having performed a FOBT (false-negative rate of 44%). Accuracy was unrelated to perceptions of getting or worrying about CRC or family history. Self-reports of having a negative FOBT result more consistently matched the laboratory result (specificity 98%) than having a positive test result (sensitivity 63%).: ConclusionsContrary to other findings, participants under- rather than overreported FOBT screening. Results suggest greater efforts are needed to enhance accurate recall of FOBT screening. [Copyright &y& Elsevier]

ABSTRACT Background: Functional neuroimaging, including positron emission tomography (PET), has been proposed for use in diagnosing Alzheimer's disease-related dementia (AD). Objective: The objective of this study was identify the circumstances under which PET scanning for the diagnosis of AD maximizes health outcomes. Methods: A Markov-model-based decision analysis was conducted using estimates derived from the literature on AD epidemiology, the accuracy of PET, and donepezil treatment efficacy. The target population for the analysis was assumed to be US men and women who either have mild AD or are asymptomatic but at an elevated risk of developing AD owing to disease in a first-degree relative (parent or sibling). The time horizon was the patient lifetime. We compared treatment 1) based on an American Academy of Neurology (AAN) clinical evaluation either alone; 2) in combination with PET scanning; or 3) empirically based on a family history. Outcomes measures were life expectancy, quality-adjusted life-years (QALYs), and (severe) dementia-free life expectancy (SDFLE). Results: For both patient populations, treating all patients based on an AAN evaluation without further testing using PET resulted in the greatest gains in life expectancy, QALYs, and SDFLEs. PET-based testing was the second preferred strategy compared to no intervention. The rankings of the strategies were sensitive to severity of treatment complications: analyses of hypothetical treatments with the potential for severe complications indicated that testing was preferred if the treatment was effective but had moderate complications. Conclusions: These results suggest that current treatments, which are relatively benign and may slow progression of disease, should be offered to patients who are identified as having AD based solely on an AAN clinical evaluation. A clinical evaluation that includes functional neuroimaging based testing will be warranted, however, when new treatments that are effective at slowing disease progression but have the potential for moderate to severe complications become available. [ABSTRACT FROM AUTHOR]

Objective.-To determine the feasibility of developing a headache management program and to assess the outcomes of patients referred to the program for treatment of chronic headache. Background.-Effective headache treatment requires that the patient receives the correct headache diagnosis; that appropriate acute and, if indicated, preventive medications be prescribed; and that the patient receives adequate education, including headache self-management skills. Design/Methods.-A headache management program was established at a northern California staff-model health maintenance organization. Fifty-four patients were enrolled in the program and followed for 6 months. Patients participated in a structured program of group and individual sessions with the program manager. Data collection at baseline and 6 months included the Migraine Disability Assessment (MIDAS), the Short Form-36 Health Survey (SF-36), a patient satisfaction survey, and 2 additional short surveys-one that assessed patient worries about their headaches and another that queried patients on their problems with headache management. Results.-All enrolled patients participated in the initial group visit; 74% had at least one additional visit. All but one patient suffered from more than one headache type. Sixty-one percent of patients suffered from migraine headache and 98% from tension-type headache. At baseline, patients were severely disabled, with a mean MIDAS score of 41. At 6 months, MIDAS scores decreased an average of 21.2 points (P <.005). Patients reported 14.5 fewer days with headache over the preceding 3 months (P < .0001) and experienced clinically significant improvements in 6 of the SF-36 subscales. Patients were significantly more satisfied with their headache care (P <.0001), reported less problems with their headache management (P < .0001), and were less worried about their headaches (P < .01). During the intervention, emergency department visits for headache decreased (P <... [ABSTRACT FROM AUTHOR]

: PurposeRandomized trials have indicated that well-managed anticoagulation with warfarin could prevent more than half of the strokes related to atrial fibrillation. However, many patients with atrial fibrillation who are eligible for this therapy either do not receive it or are not maintained within an optimal prothrombin time–international normalized ratio (INR) range. We sought to determine whether an anticoagulation service within a managed care organization would be a feasible alternative for providing anticoagulation care.: Subjects and methodsWe performed a multi-site randomized trial in six large managed care organizations in the United States. Subjects were aged 65 years or older and had nonvalvular atrial fibrillation. At each site, physician practices were divided into two geographically defined practice clusters; each site was randomly assigned to have one intervention and one control cluster. The intervention cluster received an anticoagulation service that satisfied specifications for high-quality anticoagulation care and was coordinated through the managed care organization. Control clusters continued with their usual provider-based care. We measured the proportion of time that warfarin-treated patients in each of the clusters (intervention and control) were in the target range for the INR at baseline, and again during a follow-up period.: ResultsFive of the six selected sites succeeded at developing an anticoagulation service. Patients in the intervention and control clusters had similar demographic characteristics, contraindications to warfarin, and risk factors for stroke. Among patients (n = 144 in the intervention clusters; n = 118 in the control clusters) for whom data were available during the baseline and follow-up periods, the changes in percentages of time in the target range were similar for those in the intervention clusters (baseline: 47.7%; follow-up: 55.6%) and in the control clusters (baseline: 49.1%; follow-up: 52.3%; intervention effect: 5%; 95% confidence interval: –5% to 14%; P = 0.32).: ConclusionAlthough it was feasible in a managed care organization to implement anticoagulation services that were tailored to local circumstances, provision of this service did not improve anticoagulation care compared with usual care. The effect of the anticoagulation service was limited by the utilization of the service, the degree to which the referring physician supports strict adherence to recommended target ranges for the INR, and the ability of the anticoagulation service to identify and to respond to out-of-range values promptly. [Copyright &y& Elsevier]

This paper describes a recent randomized controlled trial in which 42% of patients receiving ancrod attained a favourable outcome in comparison with 34% of controls. Although the above effect size corresponds to a number needed to treat (to achieve a favourable outcome) of approximately 13, intuition does not necessarily suggest what would be the overall impact of a treatment with this level of efficacy. The objective was to evaluate the cost-effectiveness of ancrod. Cost-effectiveness analysis of data from the Stroke Treatment with Ancrod Trial (STAT) trial was carried out. The participants were 495 patients with data on functional status at the conclusion of follow-up. Short-term results were based upon utilization and quality of life observed during the trial; these were merged with expected long-term results obtained through simulation using the Stroke Policy Model. The main outcome measure was incremental cost-effectiveness ratio. Ancrod treatment resulted in both better quality-adjusted life expectancy and lower medical costs than placebo as supported by sensitivity analysis. The cost differential was primarily attributable to the long-term implications of ancrod's role in reducing disability. If ancrod is even modestly effective, it will probably be cost-effective (and, indeed, cost-saving) as well. The net population-level impact of even modestly effective stroke treatments can be substantial. [ABSTRACT FROM AUTHOR]

Describes a general framework by which information from nonrandomized studies might be integrated into evidence reports. Details of a report supported by the Agency for Healthcare Research and Quality on preventing pulmonary complications among patients with spinal cord injury; Information on randomized controlled trials; Discussion of the efficacy of interventions pertaining to treatment of disease.

Evaluates the effectiveness of anticoagulation management programs among atrial fibrillation patients. Importance of access to the medication for such programs to become more effective; Identification of deficiencies in the practice of anticoagulant management; Alternatives recommended to medical practitioners.

With increased access to biomedical and electronic health records data and the complexity of research questions, individuals in residency programmes who aim to conduct research require specialized educational programmes and biostatistics support. Biostatistics collaboration units in academic health centres often work with residents to conduct data‐intensive research. These units face numerous challenges related to providing training in statistical literacy and collaborating on resident‐led research within very restricted timelines. Since 2019, the Duke Biostatistics, Epidemiology, and Research Design (BERD) Methods Core has supported over 247 resident‐led projects by developing tools and resources to address these challenges. This manuscript presents novel processes and training materials that other institutions can use to help biostatistics collaboration units effectively support resident training programmes. We provide a framework to support the development of collaborative teams, along with specialized training materials for residents who collaborate with these teams. [ABSTRACT FROM AUTHOR]

Simulation models that support decision and cost-effectiveness analysis can further the goals of evidence-based medicine by facilitating the synthesis of information from several sources into a single comprehensive structure. The Stroke Prevention Policy Model (SPPM) performs this function for the clinical and policy questions that surround stroke prevention. This paper first describes the basic structure and functions of the SPPM, concentrating on the role of large databases (broadly defined as any database that contains many patients, regardless of study design) in providing the SPPM inputs. Next, recognizing that the use of modeling continues to be a source of some controversy in the medical community, it discusses the philosophical underpinnings of the SPPM. Finally, it discusses conclusions in the context of both stroke prevention and other complex medical decisions. We conclude that despite the difficulties in developing comprehensive models (for example, the length and complexity of model development and validation processes, the proprietary nature of data sources, and the necessity for developing new software), the benefits of such models exceed the costs of continuing to rely on more conventional methods. Although they should not replace the clinician in decision making, comprehensive models based on the best available evidence from large databases can support decision making in medicine. [ABSTRACT FROM AUTHOR]

The CONSORT guideline defines a pilot trial as a small-scale version of a desired future efficacy trial that is intended to answer the key questions of whether and how a larger study should be done. For example, a pilot trial might evaluate different approaches to data collection or outcome measurement. However, pilot trials are unreliable for assessing treatment efficacy due to the statistical phenomenon called sampling variability. In this tutorial we use computer simulation to demonstrate the influence of sampling variability on efficacy estimates from pilot trials, illustrating why pilot trial designs should not be used to evaluate whether a treatment is promising or not. We simulate a 2-arm parallel group trial (N=20 per group) with a survival outcome as an example. Simulations are done under two scenarios: 1) the treatment is efficacious at the level of a hypothetical minimum clinically important difference (hazard ratio [HR] = 0.75); and 2) the treatment is not efficacious (HR=1). As expected, in both simulated scenarios the range of observed results is distributed around the true treatment effect, HR=0.75 or HR=1. Importantly, ∼20% of trials simulated under scenario 1 incorrectly suggest the treatment may be harmful (HR > 1). Under scenario 2, half of the simulated studies incorrectly suggest the treatment is beneficial. Treatment effect estimates from pilot trials should not be used to make future development decisions regarding a novel therapy because of the high risk of misleading conclusions. [ABSTRACT FROM AUTHOR]

Context: The Patient Care Monitor (PCM), version 2.0, is an electronic patient-reported outcomes instrument designed to be embedded into oncology practices. One key psychometric component of an instrument is its factor structure.Objectives: To validate the factor structure of the PCM.Methods: The PCM was administered within various oncology clinics at our institution from 2006 to 2011 as part of standard of care, yielding a large (n = 5624) and diverse data set. An exploratory factor analysis was performed.Results: The PCM performed well in terms of missing values and floor and ceiling effects. The three scales postulated by the PCM developers exhibited high internal consistency (Cronbach alpha 0.94-0.95); the six subscales exhibited good internal consistency (Cronbach alpha 0.80-0.95). A three-factor model approximated simple structure and was consistent with the constructs of emotional function, physical function, and physical symptoms suggested by the PCM developers. However, a six-factor model did not support the division of these three constructs into subscales of despair, distress, ambulation, impaired performance, treatment side effects, and general physical symptoms. Instead, we observed an emotional factor, a physical functioning factor, a factor including many of the treatment side effects, and three factors consisting of various clusters of physical symptoms.Conclusion: Although six subscales postulated by its developers perform reasonably, allocation of the PCM items to three constructs is more accurate and likely more consistent with how symptoms and concerns are conceptualized by patients. [ABSTRACT FROM AUTHOR]

Background: Little is known about quality of life (QOL), depression, and end-of-life (EOL) outcomes among hospitalized patients with advanced cancer. Objective: To assess whether the surprise question identifies inpatients with advanced cancer likely to have unmet palliative care needs. Design: Prospective cohort study and long-term follow-up. Setting/Subjects: From 2008 to 2010, we enrolled 150 inpatients at Duke University with stage III/IV solid tumors or lymphoma/acute leukemia and whose physician would not be surprised if they died in less than one year. Measurements: We assessed QOL (FACT-G), mood (brief CES-D), and EOL outcomes. Results: Mean FACT-G score was quite low (66.9; SD 11). Forty-five patients (30%) had a brief CES-D score of ≥4 indicating a high likelihood of depression. In multivariate analyses, better QOL was associated with less depression (OR 0.91, p < 0.0001), controlling for tumor type, education, and spiritual well-being. Physicians correctly estimated death within one year in 101 (69%) cases, yet only 37 patients (25%) used hospice, and 4 (2.7%) received a palliative care consult; 89 (60.5%) had a do-not-resuscitate order, and 63 (43%) died in the hospital. Conclusions: The surprise question identifies inpatients with advanced solid or hematologic cancers having poor QOL and frequent depressive symptoms. Although physicians expected death within a year, EOL quality outcomes were poor. Hospitalized patients with advanced cancer may benefit from palliative care interventions to improve mood, QOL, and EOL care, and the surprise question is a practical method to identify those with unmet needs. [ABSTRACT FROM AUTHOR]

This is a tutorial on designing a persuasive pilot study of a psychosocial intervention (e.g., behavioral symptom management) in the palliative care setting. This tutorial is most relevant for early stages of intervention research that aims to progress toward a randomized controlled trial with a high degree of internal validity. Broadly, a pilot study aims to address multiple elements of feasibility and acceptability so that investigators are well positioned for the next study in their program of research. To assist investigators in writing compelling grant applications we designed this tutorial as an annotated checklist of goals that a pilot study within the palliative care domain should seek to accomplish. These goals include the following: 1) begin with the end in mind, 2) use a formal conceptual model, 3) use measures with strong psychometric properties, 4) justify the timing of study sessions and assessments, 5) test recruitment methods, 6) estimate retention, 7) assess interventionist fidelity, 8) assess acceptability of the intervention, 9) assess feasibility, and 10) identify barriers to the next study. We elaborate on these goals by describing an ongoing pilot study testing the feasibility and acceptability of a psychosocial pain management intervention for patients with advanced cancer. Pilot studies are crucial for building a successful program of research, but they are also limited in terms of their sample size and overall objectives. A persuasive pilot study is one that is limited yet useful rather than limited and trivial. [ABSTRACT FROM AUTHOR]

Purpose: We sought to determine the prevalence, reliability, and predictors of conflict of interest (COI) and funding disclosure statements for studies of anticancer targeted therapies conducted in the off-label prescribing setting. Methods: As a part of a federally funded systematic review, manuscripts were included in the analysis if they were used to support one of 19 indications for cancer targeted therapies that were off-label but reimbursable according to compendia published in 2006 or before. Studies were categorized according to trial design, trial results, average impact factor of journals, and presence of COI and funding disclosure statements. Results: Among the 69 included studies, prevalence of COI and funding disclosures was low, at 33% and 58% respectively; time trends showed some improvement between 2002 to 2007, but only 60% of studies had disclosures by 2007. Predictors of COI disclosure were publication in high-impact-factor journals (P = .001), large study sample size (P = .001), enrollment exclusively in the United States (P = .04), and study of the targeted therapy in combination with other agents as opposed to the study drug alone (P = .03). Conclusion: Disclosure of potential sources of bias in COI and funding statements in studies of off-label indications for anticancer targeted therapies was low and did not increase substantially over time. [ABSTRACT FROM AUTHOR]

The authors sought to examine the relationship between physician characteristics and patient blood pressure (BP) in participants enrolled in the Hypertension Improvement Project (HIP). In this cross-sectional study using baseline data of HIP participants, the authors used multiple linear regression to examine how patient BP was related to physician characteristics, including experience, practice patterns, and clinic load. Patients had significantly lower systolic BP (SBP) (−0.2 mm Hg for every 1% increase, P=.008) and diastolic BP (DBP) (−0.1 mm Hg for every 1% increase, P=.0007) when seen by physicians with a higher percentage of patients with hypertension. Patients had significantly higher SBP (0.8 mm Hg for every 1% increase, P=.002) when seen by physicians with a higher number of total clinic visits per day. Patients had significantly lower DBP (−4.4 mm Hg decrease, P=.0002) when seen by physicians with inpatient duties. Physician's volume of patients with hypertension was related to better BP control. However, two indicators of a busy practice had conflicting relationships with BP control. Given the increasing time demands on physicians, future research should examine how physicians with a busy practice are able to successfully address BP in their patients. J Clin Hypertens (Greenwich). 2011;13:106-111. © 2010 Wiley Periodicals, Inc. [ABSTRACT FROM AUTHOR]

Context.— Headache is a common, disabling disorder that is frequently not well managed in general clinical practice. Objective.— To determine if patients cared for in a coordinated headache management program would achieve reduced headache disability compared with patients in usual care. Design.— A randomized controlled trial of headache management vs usual care. Setting.— Three distinctly different practice sites: an academic internal medicine practice located in a major east coast city, a staff-model managed care organization located in a major west coast city, and a community practice in a medium-sized city in the southeast. Patients.— Individuals 21 years of age or older with chronic tension-type, migraine, or mixed etiology headache and a Migraine Disability Assessment (MIDAS) score greater than 5, not receiving treatment from a neurologist or headache clinic currently or within the previous 6 months and with an intention to continue general medical care at their current location and to continue their present health insurance coverage for the next 12 months. Interventions.— Active intervention is a headache management program consisting of: (1) a class specifically designed to inform patients about headache types, triggers, and treatment options; (2) diagnosis and treatment by a professional especially trained in headache care (based on US Headache Consortium guidelines); and (3) proactive follow-up by a case manager. Participation lasted 6 months. Control patients received usual care from their primary care providers. Main Outcome Measures.— The primary efficacy measure reported in this article is a comparison of MIDAS scores of headache disability between the intervention group and the control group at 6 months. Secondary measures were response at 12 months, general health and quality of life, and satisfaction with headache care. Results.— The intervention improved (ie, decreased) MIDAS scores by 7.0 points (95% confidence interval 2.9 to 11.1) more than the control ( P = .008) at 6 months. The difference was not affected by site ( P = .59 for clinic by intervention interaction), and a trend toward persistent benefit at 12 months (mean difference in improvement 6.8 points, 95% confidence interval −.3 to 13.9, P = .06) was observed. Quality of life and satisfaction with headache treatment were similarly improved. Conclusions.— Coordinated headache management significantly improved outcomes for patients who, despite contact with the healthcare system for headache, had substantial unmet needs. The intervention in this trial can be implemented practically in a wide range of settings with the expectation that meaningful improvements will accrue. [ABSTRACT FROM AUTHOR]

Objective: To develop a model to predict stroke-free survival and mortality over a multiyear time frame for a trial-excluded population of medically managed asymptomatic patients with significant carotid artery stenosis. Methods: We calibrated, validated, and applied a Monte Carlo microsimulation model. For calibration we adjusted general-population mortality and stroke risks to capture these risks specific to asymptomatic carotid stenosis patients. For validation, we compared model-predicted and actual stroke-free survival curves and stroke counts from a population of comparable patients. For application, the validated model predicted stroke-free survival for a hypothetical medically managed arm of a recent single-arm carotid revascularization trial. Results: For each month in the 60-month time frame, the model-predicted and actual calibration trial stroke-free survival curves were not statistically different ( P > 0.62). In validation, the calibrated model's stroke-free survival curvematched the actual curve from an independent population; beyond 24 months, the model-predicted and actual curves were not statistically different ( P > 0.32). We also compared model-predicted and actual number of strokes from the independent trial. The model predicted 187.25 strokes (95% confidence interval 161.49–213.01), while the actual number was 171.6, within 1.22 standard deviations of the simulated mean. Conclusions: Given the absence of medically managed populations in recent carotid stenosis trials, our model can estimate stroke-free survival and mortality data for these patients. The model may also estimate the effectiveness of novel medical and procedural therapies for stroke prevention. These effectiveness estimates can inform the development of policies, guidelines, or cost-effectiveness analyses when only single-arm trial data exist. [ABSTRACT FROM AUTHOR]

It is common to assess disability of stroke patients using standardized scales, such as the Rankin Stroke Outcome Scale (RS) and the Barthel Index (BI). The RS, which was designed for applications to stroke, is based on assessing directly the global conditions of a patient. The BI, which was designed for more general applications, is based on a series of questions about the patient's ability to carry out 10 basic activities of daily living. Because both scales are commonly used, but few studies use both, translating between scales is important in gaining an overall understanding of the efficacy of alternative treatments, and in developing prognostic models that combine several datasets. The objective of our analysis is to provide a tool for translating between BI and RS. Specifically, we estimate the conditional probability distributions of each given the other. Subjects consisted of 459 individuals who sustained a stroke and who were recruited for the Kansas City Stroke Study from 1995 to 1998. We assessed patients with BI and RS measures 1, 3, and 6 months after stroke. In addition, we included data from the Framingham study, in the form of a table cross-classifying patients by RS and coarsely aggregated BI. Our statistical estimation approach is motivated by several goals: (a) overcoming the difficulty presented by the fact that our two sources report data at different resolutions; (b) smoothing the empirical counts to provide estimates of probabilities in regions of the table that are sparsely populated; (c) avoiding estimates that would conflict with medical knowledge about the relationship between the two measures; and (d) estimating the relationship between RS and BI at three months after the stroke, while borrowing strength from measurements made at 1 month and 6 months. We address these issues via a Bayesian analysis combining data augmentation and constrained semiparametric inference. Our results provide the basis for comparing and integrating the results of clinical trials using different disability measures, and integrating clinical trials results into a comprehensive decision model for the assessment of long-term implications and cost-effectiveness of stroke prevention and acute treatment interventions. In addition, our results indicate that the degree of agreement between the two measures is less strong than commonly reported, and emphasize the importance of trial designs that include multiple assessments of outcome. [ABSTRACT FROM AUTHOR]

▪ BACKGROUND Even organizations with differing mammography recommendations agree that regular repeat screening is required for mortality reduction. However, most studies have focused on one-time screening rather than repeat adherence. We compare trends in beliefs and health-related behaviors among women screened and adherent to the National Cancer Institute's screening mammography recommendations (on schedule), those screened at least once and nonadherent (off schedule), and those never screened. METHODS Our data are from a baseline telephone interview conducted among 1287 female members of Blue Cross Blue Shield of North Carolina who were aged either 40 to 44 years or 50 to 54 years. ▪ RESULTS The 3 groups differed significantly on beliefs and health-related behaviors, with the off-schedule group almost consistently falling between the on-schedule and never screened groups. Off-schedule women were more likely than on-schedule women, but less likely than those never screened, to not have a clinical breast examination within 12 months, to be ambivalent about screening mammography, to be confused about screening guidelines, and to not be advised by a physician to get a mammogram in the past 2 years. Off-schedule women perceived their breast cancer risk as lower and were less likely to be up to date with other cancer screening. tests. ▪ CONCLUSIONS Our findings suggest that women who are off schedule are in need of mammography-promoting interventions, including recommendations from and discussion with their health care providers. Because they are more positive and knowledgeable about mammography than women who have never been screened, they may benefit from brief interventions from health care providers that highlight the importance of repeat screening. [ABSTRACT FROM AUTHOR]

Purpose: Acute myeloid leukemia (AML) is a hematologic malignancy characterized by a poor prognosis but also a paradoxical possibility of cure. This renders decision-making complex and imminent. Unfortunately, many patients with AML misestimate their prognosis and treatment risk. While decision aids can improve illness understanding and reduce decisional conflict, there are no validated decision aids for AML. We developed and tested a novel AML decision aid (NCT03442452).Methods: Patients (n = 20) were recruited at Duke University from May 2018 to February 2019. Participants completed assessments of AML knowledge and decisional conflict, before and after using the electronic decision aid. The primary endpoint was feasibility (endpoint met if > 80% of study participants completed all study components). Secondary analyses of efficacy were conducted using paired t tests for dependent pre-/post-samples.Results: The primary endpoint of feasibility was met (100% of participants completed all study components). Secondary analyses showed improved knowledge and reduced decisional conflict after using the decision aid. Knowledge scores improved from a mean of 11.8 (out of 18) correct items at baseline to 15.1 correct items after using the decision aid (mean difference 3.35; p < 0.0001). Decisional conflict scores reduced significantly from baseline to post-test as well (mean difference - 6.5; p = 0.02).Conclusion: These findings suggest that our AML decision aid is a useful tool to improve the patient experience and promote shared decision-making in AML. A randomized efficacy trial is planned. [ABSTRACT FROM AUTHOR]

Examines the accuracy of blood pressure (BP) measurements in the nursing home, which a trained observer (physician) and nursing home staff (NHS) measured for 146 nursing home residents on two separate occasions. Methods; Results; Discussion.

Purpose: Utilization of electronic patient-reported outcomes (ePROs) in the clinic can improve quality of life and prolong survival in cancer care. However, there remain unanswered questions regarding trends in missing data and the potential effect on real-time patient care. Methods: This study utilized a prospectively collected dataset of ePROs from oncology clinics that administered the Patient Care Monitor 2.0 (PCM), a validated symptoms survey assessing 78 items for men, and 86 for women. We tabulated the frequency of missing items, by item and domain (emotional, functional and physical symptom-related), and examined these by age, gender, education, race and marital status. Results: Within 20,986 encounters, there were responses to at least 1 PCM item from 6933 unique patients. The highest frequency of missing answers occurred for: "attend a paid job" (10.7%), "reduced sexual enjoyment" (3.8%), and "run" (3.7%). By domain, 12.3% of functional, 8.4% of physical symptom-related, and 1.6% of emotional constructs contained at least one missing item. For functional and physical symptom-related items, missingness was most common in patients >60 years old. Conclusion: The frequency of missingness was highest for functional items, like attending a paid job, suggesting that some respondents (e.g., retirees without a paid job) skipped questions that were less applicable to them. More universal issues for cancer patients, such as emotional well-being, had much lower frequencies of missingness. This suggests differential item completion that warrants further study to understand the inherent drivers. Identifying causes of missingness could improve the clinical utility of ePROs and highlight opportunities to personalize care. [ABSTRACT FROM AUTHOR]

Purpose: Pruritus is a common symptom in cutaneous malignancies, but its impact on patients with solid tumors is unclear. We explored the impact and management of pruritus in patients with solid tumors, using patient-reported outcomes (PRO) data from a real-world registry.Methods: From 2006 to 2011, patients seen in the Duke Cancer Institute reported their symptoms via the Patient Care Monitor v2.0, a validated PRO tool that includes a 0-10-point question about pruritus severity. From > 25,000 encounters, 203 patients reported severe pruritus (> 6/10) on at least one visit and 506 total visits were abstracted where patients reported either moderate or severe pruritus (> 3/10). From this cohort, we abstracted demographics, diagnosis, stage, cancer therapy, anti-pruritic therapy, and clinicians' responses.Results: Mean age was 59.8 (SD 13.3), 134 (66%) were female, 125 (62%) were Caucasian, and 65 (32%) were African American. Breast cancer was the most common tumor (36.5%), followed by lung cancer (23.2%). Mean pruritus severity score was 6.8 (SD 1.8) for patients on chemotherapy, 6.9 (SD 1.8) for patients on targeted therapy alone or in combination, and 7.1(SD 1.8) for patients off treatment. Overall, 67% of patients reported at least two episodes of moderate-severe pruritus (mean # of visits 4.2 (SD 2.7)). Despite frequent report of severe and persistent pruritus, this was mentioned in just 28% of clinician notes and an intervention was recommended/prescribed in only 7% of visits.Conclusions: Pruritus is an under-addressed symptom in patients with solid tumors. Additional research is needed to understand the burden of pruritus in affected populations. [ABSTRACT FROM AUTHOR]

Purpose: Shared decision-making in acute myeloid leukemia (AML) requires understanding patients' longitudinal experiences of illness, but little is known about the impact of remission status on patient-reported outcomes (PROs). We aimed to explore the association between remission status and PROs 6-12 months following induction chemotherapy.Methods: Forty-two patients completed three validated instruments characterizing symptom burden (Patient Care Monitor v2.0), distress (NCCN Distress Thermometer), and QOL (FACT-Leu), as part of a longitudinal observational study. We used regression models to explore the relationship between remission status and PROs, and explore differences by initial disease type (de novo versus secondary/relapsed AML).Results: Those with secondary or relapsed AML at study onset had marked impairments in all measures compared to de novo AML patients. After 6 months, their mean distress score was 4.8 (> 4.0 warrants intervention), they reported a mean of 14.1 moderate/severe symptoms and had a mean QOL score of 113.6, compared to 1.0, 1.7, and 155.2, respectively, for those with de novo AML (p < .0001). Similarly, patients in relapse had a mean distress score of 5.3, a mean of 12.8 moderate/severe symptoms, and a mean QOL score of 113.4, compared to 1.8, 5.7, and 143.8, respectively, among those in remission (p < .005). These patterns persisted after adjusting for baseline differences (p < .0001).Conclusion: Remission is associated with markedly better patient well-being in AML. Patients with secondary or relapsed AML face more severe symptom burden, distress, and QOL issues after induction. Interventions are needed to improve AML patients' experiences of illness. [ABSTRACT FROM AUTHOR]

Context: Palliative care services are growing at an unprecedented pace. Yet, the characteristics of the clinician population who deliver these services are not known. Information on the roles, motivations, and future plans of the clinician workforce would allow for planning to sustain and grow the field.Objectives: To better understand the characteristics of clinicians within the field of hospice and palliative care.Methods: From June through December 2013, we conducted an electronic survey of American Academy of Hospice and Palliative Medicine members. We queried information on demographics, professional roles and responsibilities, motivations for entering the field, and future plans. We compared palliative care and hospice populations alongside clinician roles using chi-square analyses. Multivariable logistic regression was used to identify predictors of leaving the field early.Results: A total of 1365 persons, representing a 30% response rate, participated. Our survey findings revealed a current palliative care clinician workforce that is older, predominantly female, and generally with less than 10 years clinical experience in the field. Most clinicians have both clinical hospice and palliative care responsibilities. Many cite personal or professional growth or influential experiences during training or practice as motivations to enter the field.Conclusion: Palliative care clinicians are a heterogeneous group. We identified motivations for entering the field that can be leveraged to sustain and grow the workforce. [ABSTRACT FROM AUTHOR]

Context: In the emerging Learning Health System (LHS), the application and generation of medical knowledge are a natural outgrowth of patient care. Achieving this ideal requires a physician workforce adept in information systems, quality improvement methods, and systems-based practice to be able to use existing data to inform future care. These skills are not currently taught in medical school or graduate medical education. Case Description: We initiated a first-ever Learning Health Systems Training Program (LHSTP) for resident physicians. The curriculum builds analytical, informatics and systems engineering skills through an active-learning project utilizing health system data that culminates in a final presentation to health system leadership. Findings: LHSTP has been in place for two years, with 14 participants from multiple medical disciplines. Challenges included scheduling, mentoring, data standardization, and iterative optimization of the curriculum for real-time instruction. Satisfaction surveys and feedback were solicited mid-year in year 2. Most respondents were satisfied with the program, and several participants wished to continue in the program in various capacities after their official completion. Major Themes: We adapted our curriculum to successes and challenges encountered in the first two years. Modifications include a revised approach to teaching statistics, smaller cohorts, and more intensive mentorship. We continue to explore ways for our graduates to remain involved in the LHSTP and to disseminate this program to other institutions. Conclusion: The LHSTP is a novel curriculum that trains physicians to lead towards the LHS. Successful methods have included diverse multidisciplinary educators, just in time instruction, tailored content, and mentored projects with local health system impact. [ABSTRACT FROM AUTHOR]

Purpose: Patients with advanced non-small cell lung cancer (aNSCLC) face a significant symptom burden. Little is known about the frequency and severity of symptoms over time, so we longitudinally characterized patients' symptoms using the Patient Care Monitor (PCM) version 2.0, an electronic symptom-assessment tool.Methods: Ninety-seven patients with aNSCLC completed the PCM at up to four clinic visits. We analyzed symptom data by incidence, severity, type (functional vs. nonfunctional), proximity to death, and cancer anorexia-cachexia syndrome status (CACS).Results: Functional concerns predominated, even in the non-CACS group. Average severity among the top 5 symptoms was worse for functional than nonfunctional items (mean difference 0.62, 95% CI 0.22-1.01, P = 0.003). Severe dyspnea and fatigue were the most prevalent nonfunctional symptoms; moderate/severe dyspnea was reported by at least 29% of patients, and fatigue by over 50%. Depression was reported infrequently, with over half of patients at each visit reporting "none"; moderate or severe depression was reported in only 2.5-9.3 and 3.4-6.2% of patients, respectively. The average number of moderate/severe symptoms increased with proximity to death; 84% reported moderate/severe fatigue in the last 3 months of life, compared to 48% at ≥ 12 months from death (P = 0.007).Conclusions: Patients with aNSCLC face a significant symptom burden, which increases with proximity to death. Symptom type and severity vary by proximity to death, but even patients without overt CACS report significant functional symptoms throughout. We recommend an individualized approach to palliative symptom intervention in advanced lung cancer, based on detailed symptom assessment and tracking. [ABSTRACT FROM AUTHOR]

Objective: Pillars4Life is an educational program that teaches coping skills to cancer patients in a virtual group setting; it was recently implemented at 17 hospitals across the USA. The cost-effective, scalable, and assessable Pillars4Life curriculum targets psychosocial resources (e.g., self-efficacy and coping skills) as a means to reduce symptoms (e.g., depression, anxiety, and posttraumatic stress) and enhance quality of life.Methods: Cancer patients were recruited from hospitals that received the LIVESTRONG Community Impact Project Award to enroll in a pilot study of Pillars4Life. Consenting participants met with a certified instructor weekly for 10 weeks in a virtual environment; the manualized intervention trained participants in personal coping skills. Longitudinal assessments over 6 months were assessed using validated instruments to determine changes in Pillars4Life targeted resources and outcomes. Multiple linear regression models examined the relationship between changes in targeted resources and changes in outcome from baseline to 3 months post-intervention.Results: Participants (n = 130) had the following characteristics: mean age of 56 ± 11 years, 87% women, 11% non-Caucasian, and 77% with college degree. At 3- and 6-month follow-up, mean scores improved on all key outcome measures such as depression (Patient Health Questionnaire), anxiety (Generalized Anxiety Disorder), posttraumatic stress (Posttraumatic Stress Disorder Checklist), fatigue (Functional Assessment of Chronic Illness Therapy-Fatigue), and well-being (Functional Assessment of Cancer Therapy-General) from baseline (all p < 0.01); results were most pronounced among participants who reported ≥4/10 on the Distress Thermometer at baseline (all p < 0.001). Changes in each targeted resource were associated with 3-month improvements in at least one outcome.Conclusions: Participation in the Pillars4Life program was associated with statistically and clinically significant improvements in scores on pre-specified outcomes and targeted resources. [ABSTRACT FROM AUTHOR]

Context: Measurement of dyspnea is important for clinical care and research.Objectives: To characterize the relationship between the 0-10 Numerical Rating Scale (NRS) and four-level categorical Verbal Descriptor Scale (VDS) for dyspnea assessment.Methods: This was a substudy of a double-blind randomized controlled trial comparing palliative oxygen to room air for relief of refractory breathlessness in patients with life-limiting illness. Dyspnea was assessed with both a 0-10 NRS and a four-level categorical VDS over the one-week trial. NRS and VDS responses were analyzed in cross section and longitudinally. Relationships between NRS and VDS responses were portrayed using descriptive statistics and visual representations.Results: Two hundred twenty-six participants contributed responses. At baseline, mild and moderate levels of breathlessness were reported by 41.9% and 44.6% of participants, respectively. NRS scores demonstrated increasing mean and median levels for increasing VDS intensity, from a mean (SD) of 0.6 (±1.04) for VDS none category to 8.2 (1.4) for VDS severe category. The Spearman correlation coefficient was strong at 0.78 (P < 0.0001). Based on the distribution of NRS scores within VDS categories, we calculated test characteristics of two different cutpoint models. Both models yielded 75% correct translations from NRS to VDS; however, Model A was more sensitive for moderate or greater dyspnea, with fewer misses downcoded.Conclusion: There is strong correlation between VDS and NRS measures for dyspnea. Proposed practical cutpoints for the relationship between the dyspnea VDS and NRS are 0 for none, 1-4 for mild, 5-8 for moderate, and 9-10 for severe. [ABSTRACT FROM AUTHOR]

Background: Concerns about unsustainable costs in the US Medicare program loom as the number of retirees increase and experiences serious and costly illnesses like cancer. Engagement of stakeholders, particularly cancer patients and their families, in prioritizing insured services offers a valuable strategy for informing Medicare coverage policy. We designed and evaluated a decision exercise that allowed cancer patients and family members to choose Medicare benefits for advanced cancer patients. Methods: The decision tool, Choosing Health plans All Together (CHAT) was modified to select services for advanced cancer patients. Patients with a cancer history (N = 246) and their family members (N = 194) from North Carolina participated in 70 CHAT sessions. Variables including participants’ socio-demographic characteristics, health status, assessments of the exercise and results of group benefit selections were collected. Routine descriptive statistics summarized participant characteristics and Fisher’s exact test compared group differences. Qualitative analysis of group discussions were used to ascertain reasons for or against selecting benefits. Results: Patients and family members (N = 440) participated in 70 CHAT exercises. Many groups opted for such services as palliative care, nursing facilities, and services not currently covered by the Medicare program. In choosing among four levels of cancer treatment coverage, no groups chose basic coverage, 27 groups (39%) selected intermediate coverage, 39 groups (56%) selected high coverage, and 4 groups (6%) chose the most comprehensive cancer coverage. Reasons for or against benefit selection included fairness, necessity, need for prioritizing, personal experience, attention to family needs, holistic health outlook, preference for comfort, freedom of choice, and beliefs about the proper role of government. Participants found the exercise very easy (59%) or fairly easy (39%) to understand and very informative (66%) or fairly informative (31%). The majority agreed that the CHAT exercise led to fair decisions about priorities for coverage by which they could abide. Conclusions: It is possible to involve cancer patients and families in explicit discussions of their priorities for affordable advanced cancer care through the use of decision tools designed for this purpose. A key question is whether such a conversation is possible on a broader, national level. [ABSTRACT FROM AUTHOR]

Background: Little is known about the association between patient-oncologist discussion of cancer treatment out-of-pocket (OOP) cost and medication adherence, a critical component of quality cancer care. Methods: We surveyed insured adults receiving anticancer therapy. Patients were asked if they had discussed OOP cost with their oncologist. Medication nonadherence was defined as skipping doses or taking less medication than prescribed to make prescriptions last longer, or not filling prescriptions because of cost. Multivariable analysis assessed the association between nonadherence and cost discussions. Results: Among 300 respondents (86% response), 16% (n = 49) reported high or overwhelming financial distress. Nineteen percent (n = 56) reported talking to their oncologist about cost. Twenty-seven percent (n = 77) reported medication nonadherence. To make a prescription last longer, 14% (n = 42) skipped medication doses, and 11% (n = 33) took less medication than prescribed; 22% (n = 66) did not fill a prescription because of cost. Five percent (n = 14) reported chemotherapy nonadherence. To make a prescription last longer, 1% (n = 3) skipped chemotherapy doses, and 2% (n = 5) took less chemotherapy; 3% (n = 10) did not fill a chemotherapy prescription because of cost. In adjusted analyses, cost discussion (odds ratio [OR] = 2.58; 95% CI, 1.14 to 5.85; P = .02), financial distress (OR = 1.64, 95% CI, 1.38 to 1.96; P < .001) and higher financial burden than expected (OR = 2.89; 95% CI, 1.41 to 5.89; P < .01) were associated with increased odds of nonadherence. Conclusion: Patient-oncologist cost communication and financial distress were associated with medication nonadherence, suggesting that cost discussions are important for patients forced to make cost-related behavior alterations. Future research should examine the timing, content, and quality of cost-discussions. [ABSTRACT FROM AUTHOR]

Abstract: Background: Efficient dietary interventions for patients with hypertension in clinical settings are needed. Objective: To assess the separate and combined influence of a physician intervention (MD-I) and a patient intervention (PT-I) on dietary intakes of patients with hypertension. Design: A nested 2×2 design, randomized controlled trial conducted over 18 months. Participants: A total of 32 physicians and 574 outpatients with hypertension. Intervention: MD-I included training modules addressing the Seventh Report of the Joint National Committee on Prevention, Detection, Evaluation, and Treatment of High Blood Pressure hypertension management guidelines and lifestyle modification. PT-I included lifestyle coaching to adopt the Dietary Approaches to Stop Hypertension (DASH) eating pattern, reduce sodium intake, manage weight, increase exercise, and moderate alcohol intake. Main outcome measures: Dietary intakes were measured by the Block Food Frequency Questionnaire. Concordance with the DASH dietary pattern was estimated by a DASH score. Statistical analyses: The main effects of MD-I and PT-I, and their interaction, were evaluated using analysis of covariance. Results: After 6 months of intervention, MD-I participants significantly increased intakes of potassium, fruits, juices, and carbohydrate; decreased intake of fat; and improved overall dietary quality as measured by the Healthy Eating Index. PT-I intervention resulted in increased intakes of carbohydrate, protein, fiber, calcium, potassium, fruits and fruit juices, vegetables, dairy and Healthy Eating Index score, and decreased intakes in fat, saturated fat, cholesterol, sodium, sweets, and added fats/oils/sweets. In addition, PT-I improved overall DASH concordance score. The change in DASH score was significantly associated with the changes in blood pressure and weight at 6 months. At 18 months, most changes reversed back toward baseline levels, including the DASH score. Conclusions: Both MD-I and PT-I improved eating patterns at 6 months with some sustained effects at 18 months. Even though all dietary changes observed were consistent with the DASH nutrient targets or food group guidelines, only the PT-I intervention was effective in improving the overall DASH concordance score. This finding affirms the role of medical nutrition therapy in long-term intensive interventions for hypertension risk reduction and weight management and underlines the need for development of maintenance strategies. Furthermore, this study emphasizes the importance of collaborations among physicians, registered dietitians and other dietetics practitioners, and lay health advisors while assisting patients to make healthy behavior changes. [Copyright &y& Elsevier]

CONTEXT: Evidence-based approaches are needed to improve the delivery of specialized palliative care. OBJECTIVES: The aim of this trial was to improve on current models of service provision. METHODS: This 2x2x2 factorial cluster randomized controlled trial was conducted at an Australian community-based palliative care service, allowing three simultaneous comparative effectiveness studies. Participating patients were newly referred adults, experiencing pain, and who were expected to live >48 hours. Patients enrolled with their general practitioners (GPs) and were randomized three times: 1) individualized interdisciplinary case conference including their GP vs. control, 2) educational outreach visiting for GPs about pain management vs. control, and 3) structured educational visiting for patients/caregivers about pain management vs. control. The control condition was current palliative care. Outcomes included Australia-modified Karnofsky Performance Status (AKPS) and pain from 60 days after randomization and hospitalizations. RESULTS: There were 461 participants: mean age 71 years, 50% male, 91% with cancer, median survival 179 days, and median baseline AKPS 60. Only 47% of individuals randomized to the case conferencing intervention received it; based on a priori-defined analyses, 32% of participants were included in final analyses. Case conferencing reduced hospitalizations by 26% (least squares means hospitalizations per patient: case conference 1.26 [SE 0.10] vs. control 1.70 [SE 0.13], P=0.0069) and better maintained performance status (AKPS case conferences 57.3 [SE 1.5] vs. control 51.7 [SE 2.3], P=0.0368). Among patients with declining function (AKPS <70), case conferencing and patient/caregiver education better maintained performance status (AKPS case conferences 55.0 [SE 2.1] vs. control 46.5 [SE 2.9], P=0.0143; patient/caregiver education 54.7 [SE 2.8] vs. control 46.8 [SE 2.1], P=0.0206). Pain was unchanged. GP education did not change outcomes. CONCLUSION: A single case conference added to current specialized community-based palliative care reduced hospitalizations and better maintained performance status. Comparatively, patient/caregiver education was less effective; GP education was not effective. [ABSTRACT FROM AUTHOR]

Abstract: Context: Evidence-based approaches are needed to improve the delivery of specialized palliative care. Objectives: The aim of this trial was to improve on current models of service provision. Methods: This 2×2×2 factorial cluster randomized controlled trial was conducted at an Australian community-based palliative care service, allowing three simultaneous comparative effectiveness studies. Participating patients were newly referred adults, experiencing pain, and who were expected to live >48 hours. Patients enrolled with their general practitioners (GPs) and were randomized three times: 1) individualized interdisciplinary case conference including their GP vs. control, 2) educational outreach visiting for GPs about pain management vs. control, and 3) structured educational visiting for patients/caregivers about pain management vs. control. The control condition was current palliative care. Outcomes included Australia-modified Karnofsky Performance Status (AKPS) and pain from 60 days after randomization and hospitalizations. Results: There were 461 participants: mean age 71 years, 50% male, 91% with cancer, median survival 179 days, and median baseline AKPS 60. Only 47% of individuals randomized to the case conferencing intervention received it; based on a priori-defined analyses, 32% of participants were included in final analyses. Case conferencing reduced hospitalizations by 26% (least squares means hospitalizations per patient: case conference 1.26 [SE 0.10] vs. control 1.70 [SE 0.13], P =0.0069) and better maintained performance status (AKPS case conferences 57.3 [SE 1.5] vs. control 51.7 [SE 2.3], P =0.0368). Among patients with declining function (AKPS <70), case conferencing and patient/caregiver education better maintained performance status (AKPS case conferences 55.0 [SE 2.1] vs. control 46.5 [SE 2.9], P =0.0143; patient/caregiver education 54.7 [SE 2.8] vs. control 46.8 [SE 2.1], P =0.0206). Pain was unchanged. GP education did not change outcomes. Conclusion: A single case conference added to current specialized community-based palliative care reduced hospitalizations and better maintained performance status. Comparatively, patient/caregiver education was less effective; GP education was not effective. [Copyright &y& Elsevier]

Patterns of medical resource use near the end of life may differ across modes of death. The aim of this study was to characterize patterns of inpatient resource use and direct costs for patients with heart failure (HF) who died of sudden cardiac death (SCD), HF, other cardiovascular causes, or noncardiovascular causes during the last year of life. Data were from a randomized trial of exercise training in patients with HF. Mode of death was adjudicated by an end point committee. Generalized estimating equations were used to compare hospitalizations, inpatient days, and inpatient costs incurred during the final year of life in patients who died of different causes, adjusting for clinical and treatment characteristics. Of 2,331 patients enrolled in the trial, 231 died after ≥1 year of follow-up with an adjudicated mode of death, including 72 of SCD, 80 of HF, 34 of other cardiovascular causes, and 45 of noncardiovascular causes. Patients who died of SCD were younger, had less severe HF, and incurred fewer hospitalizations, fewer inpatient days, and lower inpatient costs than patients who died of other causes. After adjustment for patient characteristics, inpatient resource use varied by 2 to 4 times across modes of death, suggesting that cost-effectiveness analyses of interventions that reduce mortality from SCD compared to other causes should incorporate mode-specific end-of-life costs. In conclusion, resource use and associated medical costs in the last year of life differed markedly in patients with HF who experienced SCD and patients who died of other causes. [ABSTRACT FROM AUTHOR]

Purpose: Performance status is prognostic in oncology and palliative care settings. Traditionally clinician rated, it is often inconsistently collected, recorded, and measured, thereby limiting its utility. Patient-reported strategies are increasingly used for routine symptom and quality of life assessment in the clinic, and may be useful for tracking performance status. Methods: Tablet personal computers were used to collect patient- reported reviews of systems via the Patient Care Monitor (PCM) v2.0 for 86 patients with advanced lung cancer. Relevant subscales included the PCM Impaired Performance and Impaired Ambulation scales. Trained nurse clinicians measured performance status using traditional Karnofsky and Eastern Cooperative Oncology Group (ECOG) instruments. Correlation coefficients were used to compare performance status scales, and survival analysis was performed by Cox proportional hazards modeling. Results: All four performance status scales demonstrated excellent internal consistency and convergent validity. Initial KPS and ECOG scores were statistically correlated with survival, whereas PCM scores showed a nonsignificant trend in this direction. Change in PCM Impaired Performance over time was statistically correlated with survival (hazard ratio = 1.62, P = .046), whereas the other three performance status measures were not statistically prognostic. Conclusion: Patient-reported performance status as measured by PCM v2.0 is at least as reliable as KPS or ECOG. The enhanced resolution provided by this patient-reported method allows for the detection of clinically meaningful changes in trajectory over time, potentially serving as an early-warning system to trigger clinical interventions. Further study is needed to test these findings on a larger scale. [ABSTRACT FROM AUTHOR]