Nathan D. Shippee

Patient engagement in research: a systematic review

BackgroundA compelling ethical rationale supports patient engagement in healthcare research. It is also assumed that patient engagement will lead to research findings that are more pertinent to patients’ concerns and dilemmas. However; it is unclear how to best conduct this process. In this systematic review we aimed to answer 4 key questions: what are the best ways to identify patient representatives? How to engage them in designing and conducting research? What are the observed benefits of patient engagement? What are the harms and barriers of patient engagement?MethodsWe searched MEDLINE, EMBASE, PsycInfo, Cochrane, EBSCO, CINAHL, SCOPUS, Web of Science, Business Search Premier, Academic Search Premier and Google Scholar. Included studies were published in English, of any size or design that described engaging patients or their surrogates in research design. We conducted an environmental scan of the grey literature and consulted with experts and patients. Data were analyzed using a non-quantitative, meta-narrative approach.ResultsWe included 142 studies that described a spectrum of engagement. In general, engagement was feasible in most settings and most commonly done in the beginning of research (agenda setting and protocol development) and less commonly during the execution and translation of research. We found no comparative analytic studies to recommend a particular method. Patient engagement increased study enrollment rates and aided researchers in securing funding, designing study protocols and choosing relevant outcomes. The most commonly cited challenges were related to logistics (extra time and funding needed for engagement) and to an overarching worry of a tokenistic engagement.ConclusionsPatient engagement in healthcare research is likely feasible in many settings. However, this engagement comes at a cost and can become tokenistic. Research dedicated to identifying the best methods to achieve engagement is lacking and clearly needed.

Comparative effectiveness for survival and renal function of partial and radical nephrectomy for localized renal tumors: A systematic review and meta-analysis

PURPOSE The relative effectiveness of partial vs radical nephrectomy remains unclear in light of the recent phase 3 European Organization for the Research and Treatment of Cancer trial. We performed a systematic review and meta-analysis of partial vs radical nephrectomy for localized renal tumors, considering all cause and cancer specific mortality, and severe chronic kidney disease. MATERIALS AND METHODS Cochrane Central Register of Controlled Trials, MEDLINE®, EMBASE®, Scopus and Web of Science® were searched for sporadic renal tumors that were surgically treated with partial or radical nephrectomy. Generic inverse variance with fixed effects models were used to determine the pooled HR for each outcome. RESULTS Data from 21, 21 and 9 studies were pooled for all cause and cancer specific mortality, and severe chronic kidney disease, respectively. Overall 31,729 (77%) and 9,281 patients (23%) underwent radical and partial nephrectomy, respectively. According to pooled estimates partial nephrectomy correlated with a 19% risk reduction in all cause mortality (HR 0.81, p < 0.0001), a 29% risk reduction in cancer specific mortality (HR 0.71, p = 0.0002) and a 61% risk reduction in severe chronic kidney disease (HR 0.39, p < 0.0001). However, the pooled estimate of cancer specific mortality for partial nephrectomy was limited by the lack of robustness in consistent findings on sensitivity and subgroup analyses. CONCLUSIONS Our findings suggest that partial nephrectomy confers a survival advantage and a lower risk of severe chronic kidney disease after surgery for localized renal tumors. However, the results should be evaluated in the context of the low quality of the existing evidence and the significant heterogeneity across studies. Future research should use higher quality evidence to clearly demonstrate that partial nephrectomy confers superior survival and renal function.

Cumulative complexity: a functional, patient-centered model of patient complexity can improve research and practice

OBJECTIVE To design a functional, patient-centered model of patient complexity with practical applicability to analytic design and clinical practice. Existing literature on patient complexity has mainly identified its components descriptively and in isolation, lacking clarity as to their combined functions in disrupting care or to how complexity changes over time. STUDY DESIGN AND SETTING The authors developed a cumulative complexity model, which integrates existing literature and emphasizes how clinical and social factors accumulate and interact to complicate patient care. A narrative literature review is used to explicate the model. RESULTS The model emphasizes a core, patient-level mechanism whereby complicating factors impact care and outcomes: the balance between patient workload of demands and patient capacity to address demands. Workload encompasses the demands on the patients time and energy, including demands of treatment, self-care, and life in general. Capacity concerns ability to handle work (e.g., functional morbidity, financial/social resources, literacy). Workload-capacity imbalances comprise the mechanism driving patient complexity. Treatment and illness burdens serve as feedback loops, linking negative outcomes to further imbalances, such that complexity may accumulate over time. CONCLUSION With its components largely supported by existing literature, the model has implications for analytic design, clinical epidemiology, and clinical practice.

Patient and service user engagement in research: a systematic review and synthesized framework

There is growing attention towards increasing patient and service user engagement (PSUE) in biomedical and health services research. Existing variations in language and design inhibit reporting and indexing, which are crucial to comparative effectiveness in determining best practices.

Rethinking the patient: Using Burden of Treatment Theory to understand the changing dynamics of illness

BackgroundIn this article we outline Burden of Treatment Theory, a new model of the relationship between sick people, their social networks, and healthcare services. Health services face the challenge of growing populations with long-term and life-limiting conditions, they have responded to this by delegating to sick people and their networks routine work aimed at managing symptoms, and at retarding – and sometimes preventing – disease progression. This is the new proactive work of patient-hood for which patients are increasingly accountable: founded on ideas about self-care, self-empowerment, and self-actualization, and on new technologies and treatment modalities which can be shifted from the clinic into the community. These place new demands on sick people, which they may experience as burdens of treatment.DiscussionAs the burdens accumulate some patients are overwhelmed, and the consequences are likely to be poor healthcare outcomes for individual patients, increasing strain on caregivers, and rising demand and costs of healthcare services. In the face of these challenges we need to better understand the resources that patients draw upon as they respond to the demands of both burdens of illness and burdens of treatment, and the ways that resources interact with healthcare utilization.SummaryBurden of Treatment Theory is oriented to understanding how capacity for action interacts with the work that stems from healthcare. Burden of Treatment Theory is a structural model that focuses on the work that patients and their networks do. It thus helps us understand variations in healthcare utilization and adherence in different healthcare settings and clinical contexts.

Contemporary Trends in Nephrectomy for Renal Cell Carcinoma in the United States: Results From a Population Based Cohort

PURPOSE Despite benefits in functional renal outcome and the similar oncological efficacy of partial nephrectomy for renal cell carcinoma, previous studies show marked underuse of partial nephrectomy. We describe national trends in partial and radical nephrectomy using a contemporary, population based cohort. MATERIALS AND METHODS Using the 2003 to 2008 Nationwide Inpatient Sample we identified 188,702 patients treated with partial or radical nephrectomy for renal cell carcinoma at a total of 1,755 hospitals. Multivariate logistic regression was used to assess the independent associations of patient and hospital characteristics with partial nephrectomy. Post-estimations from multivariate logistic regression were done to ascertain the annual predicted probability of partial nephrectomy by hospital feature. RESULTS Overall 149,636 (79.3%) and 39,066 patients (20.7%) underwent radical and partial nephrectomy for renal cell carcinoma, respectively. Partial nephrectomy use increased each year from 16.8% in 2003 to 25.1% in 2008 (p for trend <0.001). On multivariate analysis patients were more likely to undergo partial nephrectomy at teaching (OR 1.31, p <0.001) and urban (OR 1.13, p = 0.05) hospitals compared to nonteaching and rural hospitals, respectively. Each quartile of higher nephrectomy annual volume was associated with higher odds of partial nephrectomy compared to the lowest quartile (OR 1.21, p <0.001). Although annual predicted partial nephrectomy use increased across all hospitals, differences in annual partial nephrectomy use by teaching status, site (urban vs rural) and case volume persisted with time. CONCLUSIONS Although the use of partial nephrectomy for renal cell carcinoma is increasing nationally across all hospitals, academic and urban hospitals as well as those with higher nephrectomy volume continue to show higher partial nephrectomy use for renal cell carcinoma.

Decision aids for advance care planning: An overview of the state of the science

Advance care planning is a way to inform care choices for a patient who cannot express a preference and a planning tool that helps patients begin to prioritize their treatment goals. The preferences of seriously ill patients for life-sustaining interventions depend on their care goals. Some prioritize living longer to achieve life goals, whereas others may not wish to be kept alive when meaningful recovery or a particular quality of life is no longer possible (13). Religious and spiritual values and beliefs also affect goals of care (4, 5). Advance care planning helps to honor patient preferences and goals if incapacitating illness or injury prevents adequate communication (6). Decision aids help patients consider health care options. Such aids for advance care planning support the 3 key components of the process: learning about anticipated conditions and options for care; considering these options; and communicating preferences for future care, either orally or in writing. The most important information a decision aid can provide to a decision maker depends on the patients current health status and the predictability of illness trajectories (Figure). A healthy person may benefit most from general decision aids focused on choice of health care proxies and goals of care for hypothetical catastrophic situations, such as after loss of function or cognition or terminal illness. For patients with a life-threatening illness, appropriate aids focus on decisions to accept, withhold, or terminate specific treatments. Advance care planning with decision aids takes place in various settings; it is often done outside clinical settings, particularly among healthy older adults. Nonclinical partners in shared decision making may include family members, caregivers, or attorneys or other professionals. Figure. Continuum of health states during which advance care planning may be considered. Opportunity exists for expansion and improvement of advance care planning. A 2003 Agency for Healthcare Research and Quality (AHRQ) literature summary (7) found that fewer than 50% of the severely or terminally ill patients who were studied had an advance directive (a common outcome of the advance care planning process) in their medical records (811). Furthermore, only 12% of patients with an advance directive had received input from their physician in its development (9), and physicians were accurate only about 65% of the time when predicting patient preferences; they tended to assume that patients would want less life-prolonging treatment than they actually desired, even after reviewing the patients advance directive (12). Decision aids may improve participation in advance care planning and the effectiveness of communication by facilitating clear documentation across platforms and providers and by offering insights into why patients make the decisions they do. This review, commissioned as a technical brief by the AHRQ Effective Health Care Program, provides an overview of advance care planning decision aids for adults. It describes available tools, identifies a framework for future research, and summarizes published studies that used a decision aid as an intervention. Methods Key Informants In November 2013, we conducted semistructured telephone interviews (Appendix Table 1) with 7 key informants, including practicing clinicians and attorneys involved in advance care planning, experts in medical law and medical ethics, consumer advocates, and decision aid researchers and developers. We identified these informants via frequently listed and cited authors of relevant literature, Internet searches for persons with potentially relevant viewpoints, and nominations by other key informants. They contributed information about decision aids, the context in which they are used, and important issues to consider. Appendix Table 1. Interview Probes for Key Informants Literature Search We searched MEDLINE (via Ovid), the Cochrane Library, PsycINFO, and CINAHL from January 1990 to May 2014 using a search strategy based on relevant Medical Subject Headings terms and text words (Appendix Table 2). We also conducted a gray literature search of federal and state government Web sites, the Ottawa Hospital Research Institutes Decision Aid Library Inventory, Web sites of professional organizations, and leads from key informants for decision aids available to the public and in use. Appendix Table 2. MEDLINE Search Strategy We screened abstracts and full-text articles to identify English-language studies of any sample size and design that assessed the effect of a decision aid on outcomes relevant to advance care planning. We excluded studies that involved children or advance planning for psychiatric care. We also excluded studies of decision aids for current (not future or hypothetical) end-of-life decisions; studies of forms for completing advance directives, living wills, or provider orders for life-sustaining treatment that did not include an educational component, help clarify values, or prompt action; and studies that focused on implementation science questions. The reviewers read the full text of selected articles and used a standardized data extraction form to collect reported information about study populations, decision aids, and outcomes. One reviewer abstracted data by using standardized abstraction tables, and a second reviewer provided a quality check. We used the criteria developed by the International Patient Decision Aids Standards (IPDAS) Collaboration to provide a structure for describing and comparing decision aids. These criteria have been used formally to judge quality and effectiveness in existing systematic literature reviews (13, 14). Because we followed technical brief methods, we did not synthesize outcomes, rate risk of bias, or grade the strength of evidence of the literature. Role of the Funding Source The Minnesota Evidence-based Practice Center (EPC) prepared this technical brief with funding from AHRQ. The EPC collaborated with AHRQ to develop the research protocol. Staff at AHRQ helped formulate questions and reviewed the draft report but were not involved in the study selection, data extraction, or drafting of the manuscript for publication. The full report is available at www.effectivehealthcare.ahrq.gov. Results Existing Advance Care Planning Decision Aids and Context for Use In shared clinical decision making, patients and clinicians use evidence-based knowledge, weigh options against treatment goals, and consensually arrive at a clinically prudent decision concordant with patient preferences (15, 16). Although advance care planning lies within the bounds of clinical decision making, it differs from many well-studied decision processes for medical procedures (such as surgical or nonsurgical options for cancer) because patients can make decisions with no health care provider involvement by using readily available, do-it-yourself decision aids. These aids tend to target persons with only general risks for life-threatening conditions, for whom advance care planning may involve considering a wide range of possible future scenarios, eliciting preferred goals of care, or choosing a health care proxy. Although not exhaustive, Table 1 summarizes advance care planning decision aids that target a general, predominantly healthy, older adult audience. These aids, identified through the gray literature search and by key informants, are relatively easy to find online by using common search engines. The most popular issues they address include designation of a health care proxy, clarification of values and desire for comfort care at the end of life, information on living wills or other advance directives, conversation prompts for talking to loved ones or physicians about wishes, and general preferences for various life-sustaining treatments. These aids vary in the degree to which they include the 3 components of our working definition of advance care planning decision aids, which is based on the IPDAS criteria (13, 14): an education component, a structured approach to thinking about the choices a patient faces, and a way for those choices to be communicated. Table 1. Examples of General Advance Care Planning Decision Aids Publicly Available on the Internet General decision aids for advance care planning are often used in conjunction with tools to document the decisions. Health care preferences can be documented in an advance directive and stored at a Web site, such as MyDirectives (www.MyDirectives.com). One or more proxies and their powers can be documented in a durable power of attorney for health care or as part of a more comprehensive advance directive. Health care providers can record advance care planning results (from oral discussions or an advance directive) in health care records; a specific order (such as a do-not-resuscitate order); or a template most commonly called a Physician Orders for Life-Sustaining Treatment form (found at www.polst.org), which has the advantage of serving as standing orders. Most patients gain clarity about what information can best support specific advance care planning treatment decisions as they move from hypothetical to actual clinical decisions and their familiarity with health states increases or when the health state for which a decision is needed becomes more certain. For patients with predictable progressive disease (such as amyotrophic lateral sclerosis), chronic critical illness, or frailty, a structured approach to decisions in advance care planning often requires information on prognosis. Table 2, which is not exhaustive, summarizes decision aids for advance care planning that target patients with a life-limiting illness, for which the decision trajectory is often more clearly defined. These tools are distinct from the general population tools in Table 1 because they are more likely to focus on a single advance care planning topic. They also are more likely to be designed by decision-making

Decision Aids for Advance Care Planning

Advance care planning is a way to inform care choices for a patient who cannot express a preference and a planning tool that helps patients begin to prioritize their treatment goals. The preferences of seriously ill patients for life-sustaining interventions depend on their care goals. Some prioritize living longer to achieve life goals, whereas others may not wish to be kept alive when meaningful recovery or a particular quality of life is no longer possible (13). Religious and spiritual values and beliefs also affect goals of care (4, 5). Advance care planning helps to honor patient preferences and goals if incapacitating illness or injury prevents adequate communication (6). Decision aids help patients consider health care options. Such aids for advance care planning support the 3 key components of the process: learning about anticipated conditions and options for care; considering these options; and communicating preferences for future care, either orally or in writing. The most important information a decision aid can provide to a decision maker depends on the patients current health status and the predictability of illness trajectories (Figure). A healthy person may benefit most from general decision aids focused on choice of health care proxies and goals of care for hypothetical catastrophic situations, such as after loss of function or cognition or terminal illness. For patients with a life-threatening illness, appropriate aids focus on decisions to accept, withhold, or terminate specific treatments. Advance care planning with decision aids takes place in various settings; it is often done outside clinical settings, particularly among healthy older adults. Nonclinical partners in shared decision making may include family members, caregivers, or attorneys or other professionals. Figure. Continuum of health states during which advance care planning may be considered. Opportunity exists for expansion and improvement of advance care planning. A 2003 Agency for Healthcare Research and Quality (AHRQ) literature summary (7) found that fewer than 50% of the severely or terminally ill patients who were studied had an advance directive (a common outcome of the advance care planning process) in their medical records (811). Furthermore, only 12% of patients with an advance directive had received input from their physician in its development (9), and physicians were accurate only about 65% of the time when predicting patient preferences; they tended to assume that patients would want less life-prolonging treatment than they actually desired, even after reviewing the patients advance directive (12). Decision aids may improve participation in advance care planning and the effectiveness of communication by facilitating clear documentation across platforms and providers and by offering insights into why patients make the decisions they do. This review, commissioned as a technical brief by the AHRQ Effective Health Care Program, provides an overview of advance care planning decision aids for adults. It describes available tools, identifies a framework for future research, and summarizes published studies that used a decision aid as an intervention. Methods Key Informants In November 2013, we conducted semistructured telephone interviews (Appendix Table 1) with 7 key informants, including practicing clinicians and attorneys involved in advance care planning, experts in medical law and medical ethics, consumer advocates, and decision aid researchers and developers. We identified these informants via frequently listed and cited authors of relevant literature, Internet searches for persons with potentially relevant viewpoints, and nominations by other key informants. They contributed information about decision aids, the context in which they are used, and important issues to consider. Appendix Table 1. Interview Probes for Key Informants Literature Search We searched MEDLINE (via Ovid), the Cochrane Library, PsycINFO, and CINAHL from January 1990 to May 2014 using a search strategy based on relevant Medical Subject Headings terms and text words (Appendix Table 2). We also conducted a gray literature search of federal and state government Web sites, the Ottawa Hospital Research Institutes Decision Aid Library Inventory, Web sites of professional organizations, and leads from key informants for decision aids available to the public and in use. Appendix Table 2. MEDLINE Search Strategy We screened abstracts and full-text articles to identify English-language studies of any sample size and design that assessed the effect of a decision aid on outcomes relevant to advance care planning. We excluded studies that involved children or advance planning for psychiatric care. We also excluded studies of decision aids for current (not future or hypothetical) end-of-life decisions; studies of forms for completing advance directives, living wills, or provider orders for life-sustaining treatment that did not include an educational component, help clarify values, or prompt action; and studies that focused on implementation science questions. The reviewers read the full text of selected articles and used a standardized data extraction form to collect reported information about study populations, decision aids, and outcomes. One reviewer abstracted data by using standardized abstraction tables, and a second reviewer provided a quality check. We used the criteria developed by the International Patient Decision Aids Standards (IPDAS) Collaboration to provide a structure for describing and comparing decision aids. These criteria have been used formally to judge quality and effectiveness in existing systematic literature reviews (13, 14). Because we followed technical brief methods, we did not synthesize outcomes, rate risk of bias, or grade the strength of evidence of the literature. Role of the Funding Source The Minnesota Evidence-based Practice Center (EPC) prepared this technical brief with funding from AHRQ. The EPC collaborated with AHRQ to develop the research protocol. Staff at AHRQ helped formulate questions and reviewed the draft report but were not involved in the study selection, data extraction, or drafting of the manuscript for publication. The full report is available at www.effectivehealthcare.ahrq.gov. Results Existing Advance Care Planning Decision Aids and Context for Use In shared clinical decision making, patients and clinicians use evidence-based knowledge, weigh options against treatment goals, and consensually arrive at a clinically prudent decision concordant with patient preferences (15, 16). Although advance care planning lies within the bounds of clinical decision making, it differs from many well-studied decision processes for medical procedures (such as surgical or nonsurgical options for cancer) because patients can make decisions with no health care provider involvement by using readily available, do-it-yourself decision aids. These aids tend to target persons with only general risks for life-threatening conditions, for whom advance care planning may involve considering a wide range of possible future scenarios, eliciting preferred goals of care, or choosing a health care proxy. Although not exhaustive, Table 1 summarizes advance care planning decision aids that target a general, predominantly healthy, older adult audience. These aids, identified through the gray literature search and by key informants, are relatively easy to find online by using common search engines. The most popular issues they address include designation of a health care proxy, clarification of values and desire for comfort care at the end of life, information on living wills or other advance directives, conversation prompts for talking to loved ones or physicians about wishes, and general preferences for various life-sustaining treatments. These aids vary in the degree to which they include the 3 components of our working definition of advance care planning decision aids, which is based on the IPDAS criteria (13, 14): an education component, a structured approach to thinking about the choices a patient faces, and a way for those choices to be communicated. Table 1. Examples of General Advance Care Planning Decision Aids Publicly Available on the Internet General decision aids for advance care planning are often used in conjunction with tools to document the decisions. Health care preferences can be documented in an advance directive and stored at a Web site, such as MyDirectives (www.MyDirectives.com). One or more proxies and their powers can be documented in a durable power of attorney for health care or as part of a more comprehensive advance directive. Health care providers can record advance care planning results (from oral discussions or an advance directive) in health care records; a specific order (such as a do-not-resuscitate order); or a template most commonly called a Physician Orders for Life-Sustaining Treatment form (found at www.polst.org), which has the advantage of serving as standing orders. Most patients gain clarity about what information can best support specific advance care planning treatment decisions as they move from hypothetical to actual clinical decisions and their familiarity with health states increases or when the health state for which a decision is needed becomes more certain. For patients with predictable progressive disease (such as amyotrophic lateral sclerosis), chronic critical illness, or frailty, a structured approach to decisions in advance care planning often requires information on prognosis. Table 2, which is not exhaustive, summarizes decision aids for advance care planning that target patients with a life-limiting illness, for which the decision trajectory is often more clearly defined. These tools are distinct from the general population tools in Table 1 because they are more likely to focus on a single advance care planning topic. They also are more likely to be designed by decision-making

Overwhelmed Patients A videographic analysis of how patients with type 2 diabetes and clinicians articulate and address treatment burden during clinical encounters

OBJECTIVE Patients with diabetes may experience high burden of treatment (BOT), including treatment-related effects and self-care demands. We examined whether patients with type 2 diabetes and their clinicians discuss BOT, the characteristics of their discussions, and their attempts to address BOT during visits. RESEARCH DESIGN AND METHODS Two coders independently reviewed videos of 46 primary care visits obtained during a practice-based trial and identified utterances concerning BOT, classifying them by topic and by whether BOT was addressed (i.e., whether statements emerged aimed at alleviating BOT). RESULTS Of the 46 visits, 43 (93.5%) contained BOT discussions. Both coders identified 83 discussions: 12 involving monitoring, 28 treatment administration, 19 access, and 24 treatment effects. BOT was unambiguously addressed only 30% of the time. CONCLUSIONS BOT discussions usually arise during visits but rarely beget problem-solving efforts. These discussions represent missed opportunities for reducing treatment-related disruptions in the lives of patients with diabetes, which may affect adherence and well-being.

Contemporary trends of in-hospital complications and mortality for radical cystectomy.

Study Type – Therapy (trend analysis)