Executive summary of the KDIGO 2024 Clinical Practice Guideline for the Evaluation and Management of Chronic Kidney Disease: known knowns and known unknowns.

The Kidney Disease: Improving Global Outcomes (KDIGO) Clinical Practice Guideline for the Evaluation and Management of Chronic Kidney Disease (CKD) updates the KDIGO 2012 guideline and has been developed with patient partners, clinicians, and researchers around the world, using robust methodology. This update, based on a substantially broader base of evidence than has previously been available, reflects an exciting time in nephrology. New therapies and strategies have been tested in large and diverse populations that help to inform care; however, this guideline is not intended for people receiving dialysis nor those who have a kidney transplant. The document is sensitive to international considerations, CKD across the lifespan, and discusses special considerations in implementation. The scope includes chapters dedicated to the evaluation and risk assessment of people with CKD, management to delay CKD progression and its complications, medication management and drug stewardship in CKD, and optimal models of CKD care. Treatment approaches and actionable guideline recommendations are based on systematic reviews of relevant studies and appraisal of the quality of the evidence and the strength of recommendations which followed the "Grading of Recommendations Assessment, Development, and Evaluation" (GRADE) approach. The limitations of the evidence are discussed. The guideline also provides practice points, which serve to direct clinical care or activities for which a systematic review was not conducted, and it includes useful infographics and describes an important research agenda for the future. It targets a broad audience of people with CKD and their healthcare, while being mindful of implications for policy and payment.

Learning challenges of healthcare professionals supporting open-source automated insulin delivery.

BACKGROUND: Open-source automated insulin delivery (AID) is a user-driven treatment modality used by thousands globally. Healthcare professionals' (HCPs) ability to support users of this technology is limited by a lack of knowledge of these systems. AIMS: To describe the challenges experienced by HCPs supporting participants' use of open-source automated insulin delivery in the Community deRivEd AuTomatEd insulin delivery (CREATE) study. METHODS: Data were collected prospectively from the study team's fortnightly meetings and Slack Workspace (Slack Technologies, Ltd. 2018) during the first 4 months of the trial. Key topics were identified from minutes of meetings. Slack conversations were categorised by topic, with the number of posts per conversation, number of sites per conversation and involvement of experts in open-source AID being recorded. RESULTS: In the first 4 months of the trial, there were 254 conversations in Slack with a mean of 5.2 (±4.25) posts per conversation. The most frequent learning challenge was insulin pump and cannula problems relating to the DANA-iTM insulin pump, which totalled 24.0% of all conversations. Experts on open-source AID use were involved in 83.3% of conversations. CONCLUSIONS: A significant proportion of challenges related to specific devices, rather than AID. Challenges relating to the functioning of open-source AID were more likely to involve input from experts in open-source AID. This is the first report of challenges experienced by a multidisciplinary team in a supported open-source environment that may inform expectations in routine clinical care.

Implementation and Effectiveness of Integrating Palliative Care Into Ambulatory Care of Noncancer Serious Chronic Illness: Mixed Methods Review and Meta-Analysis.

PURPOSE: To perform a mixed methods review to evaluate the effectiveness and implementation of models for integrating palliative care into ambulatory care for US adults with noncancer serious chronic illness. METHODS: We searched 3 electronic databases from January 2000 to May 2020 and included qualitative, mixed methods studies and randomized and nonrandomized controlled trials. For each study, 2 reviewers abstracted data and independently assessed for quality. We conducted meta-analyses as appropriate and graded strength of evidence (SOE) for quantitative outcomes. RESULTS: Quantitative analysis included 14 studies of 2,934 patients. Compared to usual care, models evaluated were not more effective for improving patient health-related quality of life (HRQOL) (standardized mean difference [SMD] of 4 of 8 studies, 0.19; 95% CI, ‒0.03 to 0.41) (SOE: moderate) or for patient depressive symptom scores (SMD of 3 of 9 studies, ‒0.09; 95% CI, ‒0.35 to 0.16) (SOE: moderate). Models might have little to no effect on patient satisfaction (SOE: low) but were more effective for increasing advance directive (AD) documentation (relative risk, 1.62; 95% CI, 1.35 to 1.94) (SOE: moderate). Qualitative analysis included 5 studies of 146 patients. Patient preferences for appropriate timing of palliative care varied; costs, additional visits, and travel were considered barriers to implementation. CONCLUSION: Models might have little to no effect on decreasing overall symptom burden and were not more effective than usual care for improving HRQOL or depressive symptom scores but were more effective for increasing AD documentation. Additional research should focus on identifying and addressing characteristics and implementation factors critical to integrating models to improve ambulatory, patient-centered outcomes.

Selecting Patient-Reported Outcome Measures to Contribute to Primary Care Performance Measurement: a Mixed Methods Approach.

New models of primary care include patient-reported outcome measures (PROMs) to promote patient-centered care. PROMs provide information on patient functional status and well-being, can be used to enhance care quality, and are proposed for use in assessing performance. Our objective was to identify a short list of candidate PROMs for use in primary care practice and to serve as a basis for performance measures (PMs). We used qualitative and quantitative methods to identify relevant patient-reported outcome (PRO) domains for use in performance measurement (PRO-PM) and their associated PROMs. We collected data from key informant groups: patients (n = 13; one-on-one and group interviews; concept saturation analysis), clinical thought leaders (n = 9; group discussions; thematic analysis), primary care practices representatives (n = 37; six focus groups; thematic analysis), and primary care payer representatives (n = 10; 12-question survey; frequencies of responses). We merged the key informant group information with findings from environmental literature scans. We conducted a targeted evidence review of measurement properties for candidate PROMs. We used a scoping review and key informant groups to identify PROM evaluation criteria, which were linked to the National Quality Forum measure evaluation criteria. We developed a de novo schema to score candidate PROMs against our criteria. We identified four PRO domains and 10 candidate PROMs: 3 for depressive symptoms, 2 for physical function, 3 for self-efficacy, 2 for ability to participate. Five PROMs met ≥ 70% of the evidence criteria for three PRO domains: PHQ-9 or PROMIS Depression (depression), PF-10 or PROMIS-PF (physical functioning), and PROMIS Self-Efficacy for Managing Treatments and Medications (self-efficacy). The PROMIS Ability to Participate in Social Roles and Activities met 68% of our criteria and might be considered for inclusion. Existing evidence and key informant data identified 5 candidate PROMs to use in primary care. These instruments can be used to develop PRO-PMs.

Informing Patient-Centered Care Through Stakeholder Engagement and Highly Stratified Quantitative Benefit-Harm Assessments.

OBJECTIVES: In a previous project aimed at informing patient-centered care for people with multiple chronic conditions, we performed highly stratified quantitative benefit-harm assessments for 2 top priority questions. In this current work, our goal was to describe the process and approaches we developed and to qualitatively glean important elements from it that address patient-centered care. METHODS: We engaged patients, caregivers, clinicians, and guideline developers as stakeholder representatives throughout the process of the quantitative benefit-harm assessment and investigated whether the benefit-harm balance differed based on patient preferences and characteristics (stratification). We refined strategies to select the most applicable, valid, and precise evidence. RESULTS: Two processes were important when assessing the balance of benefits and harms of interventions: (1) engaging stakeholders and (2) stratification by patient preferences and characteristics. Engaging patients and caregivers through focus groups, preference surveys, and as co-investigators provided value in prioritizing research questions, identifying relevant clinical outcomes, and clarifying the relative importance of these outcomes. Our strategies to select evidence for stratified benefit-harm assessments considered consistency across outcomes and subgroups. By quantitatively estimating the range in the benefit-harm balance resulting from true variation in preferences, we clarified whether the benefit-harm balance is preference sensitive. CONCLUSIONS: Our approaches for engaging patients and caregivers at all phases of the stratified quantitative benefit-harm assessments were feasible and revealed how sensitive the benefit-harm balance is to patient characteristics and individual preferences. Accordingly, this sensitivity can suggest to guideline developers when to tailor recommendations for specific patient subgroups or when to explicitly leave decision making to individual patients and their providers.

Outcome preferences of older people with multiple chronic conditions and hypertension: a cross-sectional survey using best-worst scaling.

BACKGROUND: Older people with hypertension and multiple chronic conditions (MCC) receive complex treatments and face challenging trade-offs. Patients' preferences for different health outcomes can impact multiple treatment decisions. Since evidence about outcome preferences is especially scarce among people with MCC our aim was to elicit preferences of people with MCC for outcomes related to hypertension, and to determine how these outcomes should be weighed when benefits and harms are assessed for patient-centered clinical practice guidelines and health economic assessments. METHODS: We sent a best-worst scaling preference survey to a random sample identified from a primary care network of Kaiser Permanente (Colorado, USA). The sample included individuals age 60 or greater with hypertension and at least two other chronic conditions. We assessed average ranking of patient-important outcomes using conditional logit regression (stroke, heart attack, heart failure, dialysis, cognitive impairment, chronic kidney disease, acute kidney injury, fainting, injurious falls, low blood pressure with dizziness, treatment burden) and studied variation across individuals. RESULTS: Of 450 invited participants, 217 (48%) completed the survey, and we excluded 10 respondents who had more than two missing choices, resulting in a final sample of 207 respondents. Participants ranked stroke as the most worrisome outcome and treatment burden as the least worrisome outcome (conditional logit parameters: 3.19 (standard error 0.09) for stroke, 0 for treatment burden). None of the outcomes were always chosen as the most or least worrisome by more than 25% of respondents, indicating that all outcomes were somewhat worrisome to respondents. Predefined subgroup analyses according to age, self-reported life-expectancy, degree of comorbidity, number of medications and antihypertensive treatment did not reveal meaningful differences. CONCLUSIONS: Although some outcomes were more worrisome to patients than others, our results indicate that none of the outcomes should be disregarded for clinical practice guidelines and health economic assessments.

Methods for Evaluating Natural Experiments in Obesity: A Systematic Review.

Background: Given the obesity pandemic, rigorous methodological approaches, including natural experiments, are needed. Purpose: To identify studies that report effects of programs, policies, or built environment changes on obesity prevention and control and to describe their methods. Data Sources: PubMed, CINAHL, PsycINFO, and EconLit (January 2000 to August 2017). Study Selection: Natural experiments and experimental studies evaluating a program, policy, or built environment change in U.S. or non-U.S. populations by using measures of obesity or obesity-related health behaviors. Data Extraction: 2 reviewers serially extracted data on study design, population characteristics, data sources and linkages, measures, and analytic methods and independently evaluated risk of bias. Data Synthesis: 294 studies (188 U.S., 106 non-U.S.) were identified, including 156 natural experiments (53%), 118 experimental studies (40%), and 20 (7%) with unclear study design. Studies used 106 (71 U.S., 35 non-U.S.) data systems; 37% of the U.S. data systems were linked to another data source. For outcomes, 112 studies reported childhood weight and 32 adult weight; 152 had physical activity and 148 had dietary measures. For analysis, natural experiments most commonly used cross-sectional comparisons of exposed and unexposed groups (n = 55 [35%]). Most natural experiments had a high risk of bias, and 63% had weak handling of withdrawals and dropouts. Limitation: Outcomes restricted to obesity measures and health behaviors; inconsistent or unclear descriptions of natural experiment designs; and imperfect methods for assessing risk of bias in natural experiments. Conclusion: Many methodologically diverse natural experiments and experimental studies were identified that reported effects of U.S. and non-U.S. programs, policies, or built environment changes on obesity prevention and control. The findings reinforce the need for methodological and analytic advances that would strengthen evaluations of obesity prevention and control initiatives. Primary Funding Source: National Institutes of Health, Office of Disease Prevention, and Agency for Healthcare Research and Quality. (PROSPERO: CRD42017055750).

Effectiveness of Policies and Programs to Combat Adult Obesity: a Systematic Review.

BACKGROUND: This systematic review identifies programs, policies, and built-environment changes targeting prevention and control of adult obesity and evaluates their effectiveness. METHODS: We searched PubMed, CINAHL, PsycINFO, and EconLit from January 2000 to March 2018. We included natural experiment studies evaluating a program, policy, or built-environment change targeting adult obesity and reporting weight/body mass index (BMI). Studies were categorized by primary intervention target: physical activity/built environment, food/beverage, messaging, or multiple. Two reviewers independently assessed the risk of bias for each study using the Effective Public Health Practice Project tool. RESULTS: Of 158 natural experiments targeting obesity, 17 reported adult weight/BMI outcomes. Four of 9 studies reporting on physical activity/built environment demonstrated reduced weight/BMI, although effect sizes were small with low strength of evidence and high risk of bias. None of the 5 studies targeting the food/beverage environment decreased weight/BMI; strength of evidence was low, and 2 studies were rated high risk of bias. DISCUSSION: We identified few natural experiments reporting on the effectiveness of programs, policies, and built-environment changes on adult obesity. Overall, we found no evidence that policies intending to promote physical activity and healthy eating had beneficial effects on weight/BMI and most studies had a high risk of bias. Limitations include few studies met our inclusion criteria; excluded studies in children and those not reporting on weight/BMI outcomes; weight/BMI reporting was very heterogeneous. More high-quality research, including natural experiments studies, is critical for informing the population-level effectiveness of obesity prevention and control initiatives in adults.

The study of effect moderation in youth suicide-prevention studies.

PURPOSE: Suicide is now the second leading cause of death among persons between the ages of adolescents and emerging adults and rates have increased despite more funding and broader implementation of youth suicide-prevention programs. A systematic review was conducted focusing on identifying youth suicide-prevention studies within the United States. This paper reports on the methods utilized for understanding possible moderators of suicide-prevention program outcomes. METHODS: We searched six databases from 1990 through August 2017 to identify studies of suicide-preventive interventions among those under age 26 years. Two independent team members screened search results and sequentially extracted information related to statistical methods of moderation analyses. RESULTS: 69 articles were included in the systematic review of which only 17 (24.6%) explored treatment effect heterogeneity using moderation analysis. The most commonly used analytic tool was regression with an interaction term. The moderators studied included demographic characteristics such as gender and ethnicity as well as individual characteristics such as traumatic stress exposure and multiple prior suicide attempts. CONCLUSIONS: With a greater emphasis from the federal government and funding agencies on precision prevention, understanding which prevention programs work for specific subgroups is essential. Only a small percentage of the reviewed articles assessed moderation effects. This is a substantial research gap driven by sample size or other limitations which have impeded the identification of intervention effect heterogeneity.

Engaging Stakeholders to Inform Clinical Practice Guidelines That Address Multiple Chronic Conditions.

BACKGROUND: Having more than one chronic condition is common and is associated with greater health care utilization, higher medication burden and complexity of treatment. However, clinical practice guidelines (CPGs) do not routinely address the balance between harms and benefits of treatments for people with multiple chronic conditions (MCCs). OBJECTIVE: To partner with the Kaiser Permanente Integrated Cardiovascular Health (ICVH) program to engage multiple stakeholders in a mixed-methods approach in order to: 1) identify two high-priority clinical questions related to MCCs, and 2) understand patients' and family caregivers' perceptions of meaningful outcomes to inform benefit/harm assessments for these two high-priority questions. These clinical questions and outcomes will be used to inform CPG recommendations for people with MCCs. DESIGN AND PARTICIPANTS: The ICVH program provided 130 topics rank-ordered by the potential for finding evidence that would change clinical recommendations regarding the topic. We used a modified Delphi method to identify and reword topics into questions relevant to people with MCCs. We used two sets of focus groups (n = 27) to elicit patient and caregiver perspectives on two important research questions and relevant patient-important outcomes on benefit/harm balance for people with MCCs. KEY RESULTS: Co-investigators, patients and caregivers identified "optimal blood pressure goals" and "diabetes medication management" as important clinical topics for CPGs related to people with MCCs. Stakeholders identified a list of relevant outcomes to be addressed in future CPG development including 1) physical function and energy, 2) emotional health and well-being, 3) avoidance of treatment burden, side effects and risks, 4) interaction with providers and health care system, and 5) prevention of adverse long-term health outcomes. CONCLUSIONS: Through the application of a mixed-methods process, we identified the questions regarding optimal blood pressure goals and diabetes medication management, along with related patient-centered outcomes, to inform novel evidence syntheses for those with MCCs. This study provides the lessons learned and a generalizable process for CPG developers to engage patient and caregivers in priority-setting for the translation of evidence into future CPGs. Ultimately, engaging patient and stakeholders around MCCs could improve the relevance of CPGs for the care of people with MCCs.

Comparative Effect of Contrast Media Type on the Incidence of Contrast-Induced Nephropathy: A Systematic Review and Meta-analysis.

BACKGROUND: Iodine contrast media are essential components of many imaging procedures. An important potential side effect is contrast-induced nephropathy (CIN). PURPOSE: To compare CIN risk for contrast media within and between osmolality classes in patients receiving diagnostic or therapeutic imaging procedures. DATA SOURCES: PubMed, EMBASE, Cochrane Library, Clinical Trials.gov, and Scopus through June 2015. STUDY SELECTION: Randomized, controlled trials that reported CIN-related outcomes in patients receiving low-osmolar contrast media (LOCM) or iso-osmolar contrast media for imaging. DATA EXTRACTION: Independent study selection and quality assessment by 2 reviewers and dual extraction of study characteristics and results. DATA SYNTHESIS: None of the 5 studies that compared types of LOCM reported a statistically significant or clinically important difference among study groups, but the strength of evidence was low. Twenty-five randomized, controlled trials found a slight reduction in CIN risk with the iso-osmolar contrast media agent iodixanol compared with a diverse group of LOCM that just reached statistical significance in a meta-analysis (pooled relative risk, 0.80 [95% CI, 0.65 to 0.99]; P = 0.045). This comparison's strength of evidence was moderate. In a meta regression of randomized, controlled trials of iodixanol, no relationship was found between route of administration and comparative CIN risk. LIMITATIONS: Few studies compared LOCM. Procedural details about contrast administration were not uniformly reported. Few studies specified clinical indications or severity of baseline renal impairment. CONCLUSION: No differences were found in CIN risk among types of LOCM. Iodixanol had a slightly lower risk for CIN than LOCM, but the lower risk did not exceed a criterion for clinical importance. PRIMARY FUNDING SOURCE: Agency for Healthcare Research and Quality.

Effectiveness of Prevention Strategies for Contrast-Induced Nephropathy: A Systematic Review and Meta-analysis.

BACKGROUND: N-acetylcysteine, sodium bicarbonate, statins, and ascorbic acid have been studied for reducing contrast-induced nephropathy (CIN). PURPOSE: To evaluate the comparative effectiveness of interventions to reduce CIN in adults receiving contrast media. DATA SOURCES: MEDLINE, EMBASE, Cochrane Library, ClinicalTrials.gov, and Scopus databases through June 2015. Risk of bias and overall strength of evidence (SOE) of studies were assessed. STUDY SELECTION: Randomized, controlled trials of N-acetylcysteine, sodium bicarbonate, statins, or ascorbic acid that used intravenous (IV) or intra-arterial contrast media and defined CIN with enough data for meta-analysis. DATA EXTRACTION: Two reviewers independently extracted data and assessed study quality. DATA SYNTHESIS: Low-dose N-acetylcysteine plus IV saline compared with IV saline (risk ratio [RR], 0.75 [95% CI, 0.63 to 0.89]; low SOE), N-acetylcysteine plus IV saline compared with IV saline in patients receiving low-osmolar contrast media (RR, 0.69 [CI, 0.58 to 0.84]; moderate SOE), and statins plus N-acetylcysteine plus IV saline versus N-acetylcysteine plus IV saline (RR, 0.52 [CI, 0.29 to 0.93]; low SOE) had clinically important and statistically significant benefits. The following 3 comparisons suggested a clinically important difference that was not statistically significant: sodium bicarbonate versus IV saline in patients receiving low-osmolar contrast media (RR, 0.65 [CI, 0.33 to 1.25]; low SOE), statins plus IV saline versus IV saline (RR, 0.68 [CI, 0.39 to 1.20]; low SOE), and ascorbic acid versus IV saline (RR, 0.72 [CI, 0.48 to 1.01]; low SOE). Strength of evidence was generally insufficient for comparisons of the need for renal replacement, cardiac events, and mortality. LIMITATION: Too few studies were done in patients receiving IV contrast media. CONCLUSION: The greatest reduction in CIN was seen with N-acetylcysteine plus IV saline in patients receiving LOCM and with statins plus N-acetylcysteine plus IV saline. PRIMARY FUNDING SOURCE: Agency for Healthcare Research and Quality.

Data Linkage Strategies to Advance Youth Suicide Prevention: A Systematic Review for a National Institutes of Health Pathways to Prevention Workshop.

BACKGROUND: Linking national, state, and community data systems to data from prevention programs could allow for longer-term assessment of outcomes and evaluation of interventions to prevent suicide. PURPOSE: To identify and describe data systems that can be linked to data from prevention studies to advance youth suicide prevention research. DATA SOURCES: A systematic review, an environmental scan, and a targeted search were conducted to identify prevention studies and potentially linkable external data systems with suicide outcomes from January 1990 through December 2015. STUDY SELECTION: Studies and data systems had to be U.S.-based and include persons aged 25 years or younger. Data systems also had to include data on suicide, suicide attempt, or suicidal ideation. DATA EXTRACTION: Information about participants, intervention type, suicide outcomes, primary analytic method used for linkage, statistical approach, analyses performed, and characteristics of data systems was abstracted by 2 reviewers. DATA SYNTHESIS: Of 47 studies (described in 59 articles) identified in the systematic review, only 6 were already linked to data systems. A total of 153 unique and potentially linkable data systems were identified, but only 66 were classified as "fairly accessible" and had data dictionaries available. Of the data systems identified, 19% were established primarily for research, 11% for clinical care or operations, 29% for administrative services (such as billing), and 52% for surveillance. About one third (37%) provided national data, 12% provided regional data, 63% provided state data, and 41% provided data below the state level (some provided coverage for >1 geographic unit). LIMITATION: Only U.S.-based studies published in English were included. CONCLUSION: There is untapped potential to evaluate and enhance suicide prevention efforts by linking suicide prevention data with existing data systems. However, sparse availability of data dictionaries and lack of adherence to standard data elements limit this potential. PRIMARY FUNDING SOURCE: Agency for Healthcare Research and Quality.

Validity of the Agency for Health Care Research and Quality Patient Safety Indicators and the Centers for Medicare and Medicaid Hospital-acquired Conditions: A Systematic Review and Meta-Analysis.

BACKGROUND: The Agency for Health Care Research and Quality Patient Safety Indicators (PSIs) and Centers for Medicare and Medicaid Services Hospital-acquired Conditions (HACs) are increasingly being used for pay-for-performance and public reporting despite concerns over their validity. Given the potential for these measures to misinform patients, misclassify hospitals, and misapply financial and reputational harm to hospitals, these need to be rigorously evaluated. We performed a systematic review and meta-analysis to assess PSI and HAC measure validity. METHODS: We searched MEDLINE and the gray literature from January 1, 1990 through January 14, 2015 for studies that addressed the validity of the HAC measures and PSIs. Secondary outcomes included the effects of present on admission (POA) modifiers, and the most common reasons for discrepancies. We developed pooled results for measures evaluated by ≥3 studies. We propose a threshold of 80% for positive predictive value or sensitivity for pay-for-performance and public reporting suitability. RESULTS: Only 5 measures, Iatrogenic Pneumothorax (PSI 6/HAC 17), Central Line-associated Bloodstream Infections (PSI 7), Postoperative hemorrhage/hematoma (PSI 9), Postoperative deep vein thrombosis/pulmonary embolus (PSI 12), and Accidental Puncture/Laceration (PSI 15), had sufficient data for pooled meta-analysis. Only PSI 15 (Accidental Puncture and Laceration) met our proposed threshold for validity (positive predictive value only) but this result was weakened by considerable heterogeneity. Coding errors were the most common reasons for discrepancies between medical record review and administrative databases. POA modifiers may improve the validity of some measures. CONCLUSION: This systematic review finds that there is limited validity for the PSI and HAC measures when measured against the reference standard of a medical chart review. Their use, as they currently exist, for public reporting and pay-for-performance, should be publicly reevaluated in light of these findings.

Are patients with amyotrophic lateral sclerosis at risk of a therapeutic misconception?

OBJECTIVES: To assess whether persons with amyotrophic lateral sclerosis (ALS) are at risk of a therapeutic misconception (TM) in which they misconceive research as treatment or overestimate the likelihood of its benefit. METHODS: 72 patients with ALS recruited via academic and patient organisations were surveyed using a hypothetical first-in-human intervention study scenario. We elicited their understanding of the purpose of the study ('purpose-of-research question') and then asked how they interpreted the question. We then asked for an estimate of the likelihood that their ALS would improve by participating and asked them to explain the meaning of their estimates. RESULTS: Although 10 of 72 (14%) subjects incorrectly said that the intervention study was 'mostly intending to help [me]' in response to the purpose-of-research question, 7 of those 10 thought that the question was asking them about their own motivations for participating. Overall, only one of 72 respondents (1.4%) both understood the purpose-of-research question as intended and gave the incorrect response. Subjects' mean estimate of likelihood of benefit was 31% (SD 26). This was due to 29 of 72 of respondents providing high estimates (50%-54% likelihood), which they said were expressions of hope and need for a positive attitude; among those who said their estimates meant 'those are the facts' or 'there is a lot of uncertainty', the estimates were much lower (12.6% and 18.5%, respectively). CONCLUSIONS: In this group of patients with ALS considering a hypothetical first-in-human intervention study, apparent TM responses have alternative explanations and the risk of true TM appears low.

Effect of childhood obesity prevention programs on blood pressure: a systematic review and meta-analysis.

BACKGROUND: Childhood overweight and obesity are associated with elevated blood pressure (BP). However, little is known about how childhood obesity lifestyle prevention programs affect BP. We assessed the effects of childhood obesity prevention programs on BP in children in developed countries. METHODS AND RESULTS: We searched databases up to April 22, 2013, for relevant randomized, controlled trials, quasi-experimental studies, and natural experiments. Studies were included if they applied a diet or physical activity intervention(s) and were followed for ≥ 1 year (or ≥ 6 months for school-based intervention studies); they were excluded if they targeted only overweight/obese subjects or those with a medical condition. In our meta-analysis, intervention effects were calculated for systolic BP and diastolic BP with the use of weighted random-effects models. Of the 23 included intervention studies (involving 18 925 participants), 21 involved a school setting. Our meta-analysis included 19 studies reporting on systolic BP and 18 on diastolic BP. The pooled intervention effect was -1.64 mm Hg (95% confidence interval, -2.56 to -0.71; P=0.001) for systolic BP and -1.44 mm Hg (95% confidence interval, -2.28 to -0.60; P=0.001) for diastolic BP. The combined diet and physical activity interventions led to a significantly greater reduction in both systolic BP and diastolic BP than the diet-only or physical activity-only intervention. Thirteen interventions (46%) had a similar effect on both adiposity-related outcomes and BP, whereas 11 interventions (39%) showed a significant desirable effect on BP but not on adiposity-related outcomes. CONCLUSIONS: Obesity prevention programs have a moderate effect on reducing BP, and those targeting both diet and physical activity seem to be more effective.

"It is not guaranteed that you will benefit": True but misleading?

BACKGROUND: Participants of early-phase intervention trials for serious conditions provide high estimates of likelihood of benefit, even when informed consent forms do not promise such benefits. However, some technically correct, negatively stated benefits statements­such as "it is not guaranteed that you will benefit"­could play a role in raising expectations of benefit because in ordinary English usage such statements denote a likely but not a certain-to-occur event. METHODS: An experimental online survey of 584 English-speaking adults recruited online. They were randomized to receive one of two benefit statements ("not guaranteed" vs "some but very small chance"), using a hypothetical scenario of an early-phase clinical trial testing an intervention to treat amyotrophic lateral sclerosis. We assessed respondents' willingness to consider participating in the amyotrophic lateral sclerosis trial, their estimates of likelihood of benefit, and their explanations for those estimates. RESULTS: The two arms did not differ in willingness to consider participation in the amyotrophic lateral sclerosis trial. Those receiving "not guaranteed" benefit statement had higher estimates of benefit than those receiving "some but very small chance" statement (35.7% (standard deviation 20.2) vs 28.3% (standard deviation 22.0), p < 0.0001). A total of 43% of all respondents chose expressions of positive sentiment (hope and need to stay positive) as explanations of their estimates; these respondents' estimates of benefit were higher than others but similar between the two arms. The effect of benefit statements was greatest among those who chose "Those are just the facts" as the explanation for their estimate (31.0% (standard deviation 22.4%) in "not guaranteed" arm vs 18.9% (standard deviation 21.0%) in comparison arm, p = 0.008). CONCLUSION: The use of "not guaranteed" language in benefit statements, when compared to "small but very small chance" language, appeared to increase the perception of likelihood of benefit of entering an early-phase trial, especially among those who view their estimates of benefits as "facts." Such "no guarantee" benefit statements may be misleading and should not be used in informed consent forms.

Are therapeutic motivation and having one's own doctor as researcher sources of therapeutic misconception?

BACKGROUND: Desire for improvement in one's illness and having one's own doctor functioning as a researcher are thought to promote therapeutic misconception (TM), a phenomenon in which research subjects are said to conflate research with treatment. PURPOSE: To examine whether subjects' therapeutic motivation and own doctor functioning as researcher are associated with TM. METHODS: We interviewed 90 persons with advanced Parkinson's disease (PD) enrolled or intending to enrol in sham surgery controlled neurosurgical trials, using qualitative interviews. Subjects were compared by motivation (primarily therapeutic vs primarily altruistic or dually motivated by altruistic and therapeutic motivation), and by doctor status (own doctor as site investigator vs not) on the following: understanding of purpose of study; understanding of research procedures; perception of chance of direct benefit; and recollection and perceptions concerning the risks. RESULTS: 60% had primarily therapeutic motivation and 44% had their own doctor as the site investigator, but neither were generally associated with increased TM responses. Overall level of understanding of purpose and procedures of research were high. Subjects responded with generally high estimates of probability of direct benefit, but their rationales were personal and complex. The therapeutic-motivation group was more sensitive to risks. Five (5.6%) subjects provided incorrect answers to the question about purpose of research, and yet, showed excellent understanding of research procedures. CONCLUSIONS: In persons with PD involved in sham surgery clinical trials, being primarily motivated by desire for direct benefit to one's illness or having one's own doctor as the site investigator were not associated with greater TM responses.

Promoting a culture of safety as a patient safety strategy: a systematic review.

Developing a culture of safety is a core element of many efforts to improve patient safety and care quality. This systematic review identifies and assesses interventions used to promote safety culture or climate in acute care settings. The authors searched MEDLINE, CINAHL, PsycINFO, Cochrane, and EMBASE to identify relevant English-language studies published from January 2000 to October 2012. They selected studies that targeted health care workers practicing in inpatient settings and included data about change in patient safety culture or climate after a targeted intervention. Two raters independently screened 3679 abstracts (which yielded 33 eligible studies in 35 articles), extracted study data, and rated study quality and strength of evidence. Eight studies included executive walk rounds or interdisciplinary rounds; 8 evaluated multicomponent, unit-based interventions; and 20 included team training or communication initiatives. Twenty-nine studies reported some improvement in safety culture or patient outcomes, but measured outcomes were highly heterogeneous. Strength of evidence was low, and most studies were pre-post evaluations of low to moderate quality. Within these limits, evidence suggests that interventions can improve perceptions of safety culture and potentially reduce patient harm.

Effectiveness of telehealth versus in-person care during the COVID-19 pandemic: a systematic review.

In this systematic review, we compared the effectiveness of telehealth with in-person care during the pandemic using PubMed, CINAHL, PsycINFO, and the Cochrane Central Register of Controlled Trials from March 2020 to April 2023. We included English-language, U.S.-healthcare relevant studies comparing telehealth with in-person care conducted after the onset of the pandemic. Two reviewers independently screened search results, serially extracted data, and independently assessed the risk of bias and strength of evidence. We identified 77 studies, the majority of which (47, 61%) were judged to have a serious or high risk of bias. Differences, if any, in healthcare utilization and clinical outcomes between in-person and telehealth care were generally small and/or not clinically meaningful and varied across the type of outcome and clinical area. For process outcomes, there was a mostly lower rate of missed visits and changes in therapy/medication and higher rates of therapy/medication adherence among patients receiving an initial telehealth visit compared with those receiving in-person care. However, the rates of up-to-date labs/paraclinical assessment were also lower among patients receiving an initial telehealth visit compared with those receiving in-person care. Most studies lacked a standardized approach to assessing outcomes. While we refrain from making an overall conclusion about the performance of telehealth versus in-person visits the use of telehealth is comparable to in-person care across a variety of outcomes and clinical areas. As we transition through the COVID-19 era, models for integrating telehealth with traditional care become increasingly important, and ongoing evaluations of telehealth will be particularly valuable.