Shared Treatment Decision Research Articles

Should all people starting urate-lowering therapy for gout receive anti-inflammatory prophylaxis?

Co-prescription of anti-inflammatory prophylaxis with colchicine, non-steroidal anti-inflammatory drugs (NSAIDs), or corticosteroids when initiating urate-lowering therapy (ULT) for gout is recommended in clinical guidelines to prevent ULT-induced flares and optimise adherence to ULT. Colchicine prophylaxis is highly clinically and cost-effective and there has been great interest recently in its cardioprotective effects. However, one in four people initiating ULT without prophylaxis in randomised trials do not have a gout flare within six months of initiation, raising the question of whether all people initiating ULT for gout should receive prophylaxis. Uptake of prophylaxis varies and appears to be common in secondary care settings but less commonly used in primary care, where most people with gout are managed and gout may be less severe. Recent clinical guidelines have highlighted that the patient’s perspective is important and that the pros and cons of prophylaxis should be discussed with people with gout initiating ULT. Uptake of prophylaxis seems likely to be influenced by perception of an individual’s risk of ULT-induced flares, as well as concerns about adverse events, polypharmacy, drug interactions, and cost. We advocate a personalised approach between people with gout and clinicians to reach shared treatment decisions when considering co-prescription of prophylaxis when initiating ULT, empowering people with gout to make decisions about their care.

Incorporating Patient Values in Large Language Model Recommendations for Surrogate and Proxy Decisions.

Surrogates, proxies, and clinicians making shared treatment decisions for patients who have lost decision-making capacity often fail to honor patients' wishes, due to stress, time pressures, misunderstanding patient values, and projecting personal biases. Advance directives intend to align care with patient values but are limited by low completion rates and application to only a subset of medical decisions. Here, we investigate the potential of large language models (LLMs) to incorporate patient values in supporting critical care clinical decision-making for incapacitated patients in a proof-of-concept study. We simulated text-based scenarios for 50 decisionally incapacitated patients for whom a medical condition required imminent clinical decisions regarding specific interventions. For each patient, we also simulated five unique value profiles captured using alternative formats: numeric ranking questionnaires, text-based questionnaires, and free-text narratives. We used pre-trained generative LLMs for two tasks: 1) text extraction of the treatments under consideration and 2) prompt-based question-answering to generate a recommendation in response to the scenario information, extracted treatment, and patient value profiles. Model outputs were compared with adjudications by three domain experts who independently evaluated each scenario and decision. Automated extractions of the treatment in question were accurate for 88% (n = 44/50) of scenarios. LLM treatment recommendations received an average Likert score by the adjudicators of 3.92 of 5.00 (five being best) across all patients for being medically plausible and reasonable treatment recommendations, and 3.58 of 5.00 for reflecting the documented values of the patient. Scores were highest when patient values were captured as short, unstructured, and free-text narratives based on simulated patient profiles. This proof-of-concept study demonstrates the potential for LLMs to function as support tools for surrogates, proxies, and clinicians aiming to honor the wishes and values of decisionally incapacitated patients.

Consensus-Based Overarching Principles and Recommendations on the Use of Biosimilars in the Treatment of Inflammatory Arthritis in the Gulf Region.

Though biologic agents have significantly improved the treatment of inflammatory arthritis (rheumatoid arthritis, psoriatic arthritis, and axial spondyloarthritis), high costs, stringent regulations, strict reimbursement criteria, and existing patents have limited patient access to treatments. While being highly similar in quality, safety, and efficacy to biologic reference products, biosimilars can reduce the financial burden and prevent underutilization of medication. The objective of this initiative was to develop an evidence-based consensus of overarching principles and recommendations aimed at standardizing the use of biosimilars in treating inflammatory arthritis in the Gulf region. A task force of practicing rheumatologists, a clinical pharmacist, a health economist, patients, regulators, and payors from across the Gulf region developed recommendations and overarching principles based on the outputs of a systematic literature review conducted to address Patient-Intervention-Comparison-Outcome (PICO) questions specific to key challenges regarding the use of biosimilars for the treatment of inflammatory arthritis in the region. As the data before 2017 have been previously reviewed in another publication, the current review focused on data published between January 2017 and August 2022 (PROSPERO ID CRD42022364002). Consensus on each statement required a level of agreement of 70% or greater. Consensus was reached for five overarching principles and nine recommendations by the task force. The principles emphasize the importance of improving the awareness, understanding, and perception of biosimilars, as well as the need for regulated regional real-world data generation and protocols to make biosimilars a viable and affordable treatment option for all patients. The consensus recommendations advocate the need for shared treatment decisions between rheumatologists and patients when considering biosimilars. They further recommend that confirmation of a biosimilar's efficacy and safety in a single indication is sufficient for extrapolation to other diseases for which the reference product has been approved. Finally, there is a need for pharmacovigilance and national health policies governing the adoption and prescription of biosimilars in clinical practice across the region. These are the first consensus recommendations for the Gulf region based on a systematic literature review and Preferred Reporting Items for Systematic Review and Meta-analysis (PRISMA) guidelines, integrating clinical evidence with clinical expertise to optimize decision making for the use of biosimilars in patients with inflammatory arthritis. They were formulated based on predominantly international data because of the limited regional data and therefore can be generalized to serve as recommendations for healthcare professionals in other parts of the world.

Incorporating a polygenic risk score into cardiovascular disease examinations to identify subclinical coronary artery disease: rationale and design of the ESCALATE trial

Abstract Background Identifying and targeting established modifiable risk factors such as hyperlipidaemia, blood pressure, smoking, and diabetes have led to a successful reduction in poor outcomes from coronary artery disease (CAD) at the population-level. However, up to one-in-four patients who present with ST elevation myocardial infarction do so in the absence of such risk factors. There is a significant unmet need for new diagnostic strategies independent of these factors to detect CAD in its subclinical phase. Polygenic risk scores (PRS) have demonstrated an ability in cohort studies to improve risk prediction models independent of traditional risk factors and self-reported family history. However, there is a lack of data from implementation and interventional studies demonstrating efficacy, participant and provider experience, and cost effectiveness of utilising PRS. Aim The aim of this study is to examine the incorporation of a CAD PRS during primary care-based cardiovascular disease (CVD) risk assessments to identify low or intermediate risk individuals with newly diagnosed CAD and subsequent impact on shared treatment decisions, participant experience, and provider experience. Our hypothesis is that incorporating a CAD PRS-based decision tool for a coronary calcium scan into CVD risk assessments will identify new, potentially missed individuals with subclinical CAD. Methods The ESCALATE study is a 12-month, prospective, multi-centre implementation study incorporating CAD PRS into otherwise standard primary care CVD risk assessments, identifying patients at increased lifetime CAD risk, for non-invasive CT coronary imaging. 1,000 eligible participants aged 45-65 years old will enter the PPP-CAD Diagnostic Pathway, which applies CAD PRS to those considered low or moderate 5-year absolute CVD risk based on traditional risk factors and triages those with CAD PRS ≥80% for a coronary calcium scan (Figure 1). The primary outcome will be the identification of subclinical CAD, defined as a coronary artery calcium score (CACS) >0 Agatston units (AU). Multiple secondary outcomes will be assessed, including baseline CACS ≥100 AU or ≥75th age-/sex-matched percentile, medical management strategies, cholesterol and blood pressure levels, health-related quality of life (HRQOL), and patient experience. Results Study recruitment was initiated recruitment in August 2022 and is actively recruiting across Australia. 298 participants have been enrolled as of 27th February 2022. Conclusion The ESCALATE study is the first prospectively registered clinical trial testing the ability of a CAD PRS to: a) identify individuals with subclinical CAD by non-invasive coronary imaging; and b) measure the impact of this diagnostic strategy on personalised diagnosis and shared decision-making. The top-line study results are expected in mid-2025.

Comparison of the Efficacy in Clinical Trials Versus Effectiveness in the Real-World of Treatments for Multiple Myeloma: A Population-Based Cohort Study

Introduction: Improvements in outcomes of patients with multiple myeloma (MM) depend on the use of regimens approved based on results from large phase III randomized controlled trials (RCTs) demonstrating their efficacy. However, many real-world (RW) patients would not have met the stringent RCTs inclusion criteria. Therefore, the effectiveness of these drugs in the RW setting is unknown. Understanding this efficacy-effectiveness gap is important to contextualize the expected outcomes in the current RW setting. Therefore, we conducted a retrospective population-based study to compare the efficacy versus effectiveness of registration RCTs with RW patients using standard of care (SoC) MM regimens for the primary outcomes of 1) progression free survival [PFS]; 2) overall survival [OS] and 3) serious adverse events (AEs). Methods: RW data was obtained from the Institute for Clinical Evaluative Sciences, an administrative database capturing all health records in the publicly funded health care system in Ontario, Canada. Adult patients treated between Jan 2007 to Dec 2020 with SoC regimens were included. Only regimens with corresponding registrational phase III RCTs which led to the public reimbursement in Ontario were included (lenalidomide/dex [Rd] and bortezomib/Rd [VRd] for newly diagnosed transplant ineligible patients, and relapsed MM (RRMM) regimens included carfilzomib/Rd [KRd], carfilzomib/dex [Kd], daratumumab/Rd [DRd], daratumumab/bortezomib/dex [DVd], pomalidomide/dex [Pd]). In the RW cohort, PFS was defined as the time from initiation of index regimen to death, initiation of subsequent MM treatment, or last follow-up, and patients remaining on the index regimen as last follow up were censored. Kaplan-Meier curves from pivotal phase 3 RCTs were manually digitized to provide individual patient-level estimates of PFS and OS. Meta-analyses were performed to compare the gap of PFS and OS outcomes of RW versus RCT patients, and effect estimates were summarized using hazard ratios (HR). The frequency of serious AE data was abstracted from published RCTs. Given that serious AEs in RCTs would have resulted in hospitalization, hospital admission during treatment with the index regimen was used as a surrogate for serious AEs in the RW cohort. Differences in RW and RCT safety outcomes were reported descriptively. Results & Discussion: A total of 3951 RW MM patients, treated with 7 standard of care MM regimens, were included. Baseline characteristics of patients in the RW and RCT cohorts are shown in table 1. Overall, patients in the RW cohort were older than in the RCTs. For relapsed regimens, there was a longer time between MM diagnosis and start of the regimen in the real-world versus RCT. With regards to the efficacy-effectiveness gap, MM patients treated in routine practise in the RW had a worse PFS despite overestimated of RW PFS compared to highly selected RCT patients for 6 of the 7 MM regimens evaluated, with a pooled HR of 1.44 (95% CI 1.34-1.54) in the meta-analysis (Figure 1A). Similarly, RW patients had a worse OS compared to RCT patients treated with 6 of the 7 regimens, with a pooled HR of 1.75 (95% ci 1.63-1.88) in the meta-analysis (Figure 1B). RRMM patients in the RW had higher rates of prior lenalidomide exposure compared to RCT patients. The only regimen which showed a trend towards performing better in the RW as compared to RCTs was Pd. The reason for this is likely multifactorial but perhaps patients included in the MM-003 RCT may have had more refractory MM (given the higher prior immunomodulatory drug exposure and longer time from diagnosis to treatment among Pd RCT patient) compared to RW patients in this study. With regards to safety, the percentage of patients with inpatient hospitalization during treatment in the real-world cohort and reported serious AEs in RCT were comparable (VRD 57% vs not reported [NR]; Rd 64% vs NR; Kd 57% vs 59%; KRd 53% vs 60%; DVd 36% vs NR; DRd 46% vs 49%; Pd 59% vs 61%). Conclusion: This is one the largest population-level studies highlighting the significant efficacy-effectiveness gap between registrational RCTs and RW usage of these regimens, with RW patients experiencing 44% worse PFS and 75% worse OS compared to RCT patients. Our data emphasize the importance of ongoing evaluation of RW data to contextualize effectiveness and toxicity of selected regimens in the clinic, and better inform both clinicians and patients for shared treatment decision making.

What Is Most Important to Family Caregivers When Helping Patients Make Treatment-Related Decisions: Findings from a National Survey.

Prioritizing patient values-who/what matters most-is central to palliative care and critical to treatment decision making. Yet which factors are most important to family caregivers in these decisions remains understudied. Using data from a U.S. national survey of cancer caregivers (N = 1661), we examined differences in factors considered very important by caregivers when partnering with patients in cancer treatment decision making by cancer stage and caregiver sociodemographics. Fifteen factors were rated on a 4-point Likert-scale from 'very unimportant' to 'very important.' Descriptive statistics were used to characterize caregiver factors and tabulate proportions of importance for each. Generalized linear mixed effect modeling was used to examine the importance of factors by cancer stage, and chi-square analyses were performed to determine associations between caregiver sociodemographics and the five most commonly endorsed factors: quality of life (69%), physical well-being (68%), length of life (66%), emotional well-being (63%), and opinions/feelings of oncology team (59%). Significant associations (all p's < 0.05) of small magnitude were found between the most endorsed factors and caregiver age, race, gender, and ethnicity, most especially 'opinions/feelings of the oncology team'. Future work is needed to determine the best timing and approach for eliciting and effectively incorporating caregiver values and preferences into shared treatment decision making.

The role of low health literacy in shared treatment decision-making in patients with kidney failure.

The classic paternalist medicine in nephrology has been modified to a shared decision-making model that clearly offers a benefit in patients with kidney disease. One of the cornerstones of shared treatment decision in patients with kidney failure is the understanding of kidney disease. As kidney disease is silent until advanced stages and is also an entity with a complex pathophysiology with little knowledge in the general population, its presence and understanding are difficult for most people. Health literacy (HL) plays a crucial role in the care of patients with kidney disease and the shared treatment decision. Limited HL has been associated with inefficient use of health services, non-compliance of medications, worse quality of life and increased mortality. In this review, we will address the importance of low HL in nephrology in terms of diagnosis, measurement, its effect on shared decision-making and how to increase it in people with kidney disease.

Understanding how shared decision-making approaches and patient aids influence patients with advanced cancer when deciding on palliative treatments and care: A realist review.

Patients with advanced incurable cancer face difficult decisions about palliative treatment options towards their end of life. However, they are often not provided with the appropriate information and support that is needed to make informed decisions. This review aimed to identify contexts and mechanisms associated with communication tools, patient decision-aids and shared decision-making (SDM) approaches that influence patient outcomes. We used a realist review method to search for published studies of patients (adults > 18) with advanced cancer who were expected to make a decision about palliative treatment and/or supportive care in consultationwith healthcare practitioners. We appraised and synthesised literature describing the contexts of (when and how) decision aids and SDM approaches are used, and how these contexts interact with mechanisms (resources and reasoning) which impact patient outcomes. Stakeholders including academics, palliative healthcare professionals(HCPs) and people with lived experience of supporting people with advanced incurable cancer contributed to identifying explanatory accounts. These accounts were documented, analysed and consolidated to contribute to the development of a programme theory. From the 33 included papers, we consolidated findings into 20 explanatory accounts to develop a programme theory that explains key contexts and mechanisms that influence patient and SDM. Contexts include underlying patients' and HCPs'attitudes and approaches. These need to be understood in relation to key mechanisms, including presenting information in multiple formats and providing adequate time and opportunities to prepare for and revisit decisions. Contexts influenced mechanisms which then influence the levels of patient decisional satisfaction, conflict and regret. Our programme theory highlights mechanisms that are important in supporting shared treatment decisions for advanced noncurative cancer. The findings are informative for developing and evaluating interventions to improve understanding and involvement in SDM for patients with advanced incurable cancer. We included patient and public involvement (PPI) representatives in four stakeholder meetings. PPI helped to define the scope of the review, identify their unique experiences and perspectives, synthesise their perspectives with our review findings, make decisions about which theories we included in our programme theory and develop recommendations for policy and practice and future research.

Incorporating a polygenic risk score-triaged coronary calcium score into cardiovascular disease examinations to identify subclinical coronary artery disease (ESCALATE): Protocol for a prospective, nonrandomized implementation trial

Identifying and targeting established modifiable risk factors has been a successful strategy for reducing the burden of coronary artery disease (CAD) at the population-level. However, up to 1-in-4 patients who present with ST elevation myocardial infarction do so in the absence of such risk factors. Polygenic risk scores (PRS) have demonstrated an ability to improve risk prediction models independent of traditional risk factors and self-reported family history, but a pathway for implementation has yet to be clearly identified. The aim of this study is to examine the utility of a CAD PRS to identify individuals with subclinical CAD via a novel clinical pathway, triaging low or intermediate absolute risk individuals for noninvasive coronary imaging, and examining the impact on shared treatment decisions and participant experience. The ESCALATE study is a 12-month, prospective, multicenter implementation study incorporating PRS into otherwise standard primary care CVD risk assessments, to identify patients at increased lifetime CAD risk for noninvasive coronary imaging. One-thousand eligible participants aged 45 to 65 years old will enter the study, which applies PRS to those considered low or moderate 5-year absolute CVD risk and triages those with CAD PRS ≥80% for a coronary calcium scan. The primary outcome will be the identification of subclinical CAD, defined as a coronary artery calcium score (CACS) >0 Agatston units (AU). Multiple secondary outcomes will be assessed, including baseline CACS ≥100 AU or ≥75th age-/sex-matched percentile, the use and intensity of lipid- and blood pressure-lowering therapeutics, cholesterol and blood pressure levels, and health-related quality of life (HRQOL). This novel trial will generate evidence on the ability of a PRS-triaged CACS to identify subclinical CAD, as well as subsequent differences in traditional risk factor medical management, pharmacotherapy utilization, and participant experience. Australian New Zealand Clinical Trials Registry, ACTRN12622000436774. Trial was prospectively registered on March 18, 2022. https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id=383134.

Breaking down barriers for prescribing and using hormone therapy for the treatment of menopausal symptoms: an experts’ perspective

ABSTRACT Introduction Around 80% of women suffer menopause-related symptoms that affect their daily activities and quality of life. Menopausal hormone therapy (MHT) has proven to be beneficial in relieving these symptoms. Nevertheless, only 20/30% of symptomatic women seek treatment. This has resulted in neglect of a generation of healthcare professionals’ (HCPs) education in menopausal medicine and a reduction in the prescription of MHT in menopausal women for over two decades. Areas covered The aim of this article was to identify the main barriers that HCPs face for prescribing MHT and menopausal women for using it. Six European experts in menopause agreed on the profiles of women that benefit from MHT and proposed strategies to break down these barriers. Expert opinion The most important barrier for HCPs was deficient knowledge of the true evidence-based information, with inadequate training regarding the efficacy and safety of personalized MHT and the real benefit/risk ratio in the treatment of symptomatic women. For patients, fear of developing breast cancer was identified as the single most important barrier. Breaking down barriers is possible, by providing appropriate training and education to HCPs and women. This should result in fully informed, evidence based, shared treatment decisions by women and their physicians.

Baclofen and the Risk of Encephalopathy: A Real-World, Active-Comparator Cohort Study

ObjectiveTo quantify the risk of encephalopathy associated with oral baclofen compared with other muscle relaxants—tizanidine or cyclobenzaprine. Patients and MethodsWe conducted a new-user, active-comparator study of 2 pairwise cohorts using tertiary health system data from Geisinger Health in Pennsylvania (January 1, 2005, through December 31, 2018). Adults (aged ≥18 years) newly treated with baclofen or tizanidine were included in cohort 1. Adults newly treated with baclofen or cyclobenzaprine were included in cohort 2. Propensity score–based inverse probability of treatment weighting (IPTW) was used to balance the respective cohorts on 45 patient characteristics. Fine-Gray competing risk regression was used to estimate the risk of encephalopathy. ResultsCohort 1 included 16,192 new baclofen users and 9782 new tizanidine users. The 30-day risk of encephalopathy was higher in patients treated with baclofen vs tizanidine (IPTW incidence rate, 64.7 vs 28.3 per 1000 person-years) with an IPTW subdistribution hazard ratio (SHR) of 2.29 (95% CI, 1.43 to 3.67). This risk persisted through 1 year (SHR, 1.32 [95% CI, 1.07 to 1.64]). Similarly in cohort 2, baclofen vs cyclobenzaprine was associated with a greater risk of encephalopathy at 30 days (SHR, 2.35 [95% CI, 1.59 to 3.48]) that persisted through the first year of treatment (SHR, 1.94 [95% CI, 1.56 to 2.40]). ConclusionThe risk of encephalopathy was greater with baclofen vs tizanidine or cyclobenzaprine use. The elevated risk was apparent as early as 30 days and persisted through the first year of treatment. Our findings from routine care settings may inform shared treatment decisions between patients and prescribers.

National Knowledge-Driven Management of Obstructive Sleep Apnea—The Swedish Approach

This paper describes the development of "Swedish Guidelines for OSA treatment" and the underlying managed care process. The Apnea Hypopnea Index (AHI) is traditionally used as a single parameter for obstructive sleep apnea (OSA) severity classification, although poorly associated with symptomatology and outcome. We instead implement a novel matrix for shared treatment decisions based on available evidence. A national expert group including medical and dental specialists, nurses, and patient representatives developed the knowledge-driven management model. A Delphi round was performed amongst experts from all Swedish regions (N = 24). Evidence reflecting treatment effects was extracted from systematic reviews, meta-analyses, and randomized clinical trials. The treatment decision in the process includes a matrix with five categories from a "very weak"" to "very strong" indication to treat, and it includes factors with potential influence on outcome, including (A) OSA-related symptoms, (B) cardiometabolic comorbidities, (C) frequency of respiratory events, and (D) age. OSA-related symptoms indicate a strong incitement to treat, whereas the absence of symptoms, age above 65 years, and no or well-controlled comorbidities indicate a weak treatment indication, irrespective of AHI. The novel treatment matrix is based on the effects of treatments rather than the actual frequency of respiratory events during sleep. A nationwide implementation of this matrix is ongoing, and the outcome is monitored in a prospective evaluation by means of the Swedish Sleep Apnea Registry (SESAR).

Abstract OT2-05-01: FLEX: 30K Full Transcriptome, Real-World Evidence Database for Early-Stage Breast Cancer, and Investigator-Initiated Protocols

Abstract FLEX: 30K Full Transcriptome, Real-World Evidence Database for Early-Stage Breast Cancer, and Investigator-Initiated Protocols BACKGROUND: Genomic signatures, such as the 70-gene MammaPrint, provide additional prognostic information for early-stage breast cancer (EBC), such as tumor metastatic potential, and expand the available information clinicians and patients require for shared treatment decision making beyond the standard clinicopathologic factors. EBC tumors are further stratified into clinically actionable subtypes by molecular assays such as the 80-gene molecular profiling assay BluePrint, improving pathological complete response rates (pCR) and outcomes as evidenced by a number of recent trials. The ongoing FLEX Study (NCT03053193) is designed to expand the genomic information available for EBC cases, and to increase the speed of data generation for rare and underserved research areas. To date, FLEX is the largest international multicenter real-world evidence (RWE) EBC registry, with more than 10,000 patients enrolled in fewer than five years since opening. FLEX pairs full genome data with more than 800 clinical data points collected over 10 years of follow up to provide the most comprehensive big data database available for early-stage breast cancer. The FLEX enrollment has a goal of a minimum of 30,000 patients within 10 years. METHODS: The FLEX study is a multicenter, prospective, observational trial for patients ≥18 years old with histologically proven stage I-III invasive breast cancer that is node negative or positive (up to three nodes) who receive MammaPrint, with or without BluePrint as standard of care management. Patients consent to the collection of clinically annotated full transcriptome data. Additionally, this study protocol allows physicians to investigate targeted populations or clinical trial investigator-initiated studies (IIS) upon approval by a peer-driven Scientific Review Committee. As patients enrolled in the FLEX study meet all eligibility criteria for inclusion no additional consent is required. The FLEX enrollment has already surpassed 1/3 of set target goal. As of April 2022, the trial has surpassed 10,000 patients enrolled at nearly 100 trial sites across the US and Europe, Middle East and Africa (EMEA). To date, there have been 36 publications in international scientific congresses with 39 FLEX IIS and in progress. With over 250 active and collaborating physicians leveraging the shared infrastructure, the IIS have enabled the ability to address disparities in treatment to underrepresented populations, rare subtypes, age, and patient centered specific topics. Current and future questions investigated via this platform will continue to strive to improve outcomes for early-stage breast cancer patients. Citation Format: Alejandra Perez, Hannah Linden, Nathalie Johnson, Sami Diab, Chirag Jani, Chelsea D. Gawryletz, Richard Fine, Laura Lawson, Megan Baker, Victoria Poillucci, Lisa E. Blumencranz, William Audeh. FLEX: 30K Full Transcriptome, Real-World Evidence Database for Early-Stage Breast Cancer, and Investigator-Initiated Protocols [abstract]. In: Proceedings of the 2022 San Antonio Breast Cancer Symposium; 2022 Dec 6-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2023;83(5 Suppl):Abstract nr OT2-05-01.

Clinician and Patient Factors Influencing Treatment Decisions in Front-Line Chronic Lymphocytic Leukemia: A Qualitative Research Study

Background. Emerging data on novel targeted agents have been advancing the treatment landscape of front-line (1L) chronic lymphocytic leukemia (CLL) in the past decade. Despite the advances, lack of mature overall survival data for novel agents presents a challenge to treatment selection. In the absence of demonstrated long-term survival advantages, treatment selection may be subject to other factors. We hypothesize that these include physician preferences and patients’ unique evaluation of potential risks, perceived benefits, and characteristics of individual treatment modalities, such as location, time-limited (TL) vs. treat-to-progression (TPP) duration and need for monitoring. As such, there is value in understanding physician and patient preferences and the key factors that influence their goals of therapy and subsequent treatment selection. Methods. Patients and treating physicians (hematologist oncologists) were recruited across three countries to participate in semi-structured telephone interviews lasting approximately 45 minutes in April 2022. The sample size was based on thematic data saturation principle, setting a threshold for when no new key themes emerge with each subsequent additional interview. Our sample size of 23 patients (France, n=8; Germany, n=7; UK, n=8) and 12 physicians (France, n=3; Germany, n=3; UK, n=3; Australia, n=3) considered the range where saturation is usually achieved, between 6 and 12 interviews (Guest, 2006) and the desire for diverse patient voices. The semi-structured interview guide elicited feedback on treatment experiences, allowed spontaneous mention of attributes, and probed on a set of prespecified attributes. The key areas of focus were patient-physician interactions, treatment attributes (including efficacy, side effects, and duration of therapy), and overall treatment experiences. Thematic analysis of interview data was done using NVivo transcription coding to see the connection between emerging themes and to identify key insights. Results. Among patients, 30% (7/23) were treatment naive, 30% (7/23) had received 1L treatment and 39% (9/23) had received 2L treatment or later. Study participants demographics are summarized in Table 1. Efficacy was consistently reported as the top priority in selecting a new treatment for both patients (43%, 10/23) and physicians (91%, 11/12). However, only slightly over half of the patients and physicians reported discussing efficacy with each other at the point of treatment decision (patients, 56%, 13/23; physicians, 58%, 7/12). When asked about attributes most important in treatment selection, 56% (13/23) of patients unaided mentioned a desire to avoid side effects that would impact their quality of life. When presented with a list of treatment attributes (Table 2), patients’ top side effects to avoid were heart rhythm problems and diarrhea. Physicians similarly chose avoiding atrial fibrillation as their top concern. In contrast, physicians also prioritized avoiding infection and side effects leading to treatment discontinuation. Patients varied in the importance they placed on TL vs. TPP (explained as "taken until the cancer worsens") duration. When selecting treatment, 22% (5/23) of patients reported treatment duration as a consideration, while 48% (11/23) reported duration as "less important” than side effects and administration mode (infusion vs. oral); and 26% (6/23) reported it "not important". Nine patients stated a preference for TL treatment, of which two preferred TL therapy as they were looking forward to a treatment-free period. One patient preferred a TPP treatment they perceived as safer and offering better QoL. Most physicians (n=8, 75%) reported deferring to patients on preferences for TL versus TTP assuming eligibility; however, 15 out of 18 (83%) patients reported that they were offered only one treatment option. Conclusions: This research highlights the similarities and variation in patient perspectives on treatment selection, the importance of covering key topics in shared treatment decision making and the remaining gaps in communication around treatment options. Regularly providing and discussing information on efficacy, and accounting for patient preferences, may improve the treatment selection experience for patients, particularly as novel treatment regimens become available. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

Supporting Shared Decision-Making and Home Dialysis in End-Stage Kidney Disease.

It has been widely demonstrated that patient education and empowerment, especially involving shared treatment decisions, improve patient outcomes in chronic medical conditions, including chronic kidney disease requiring kidney replacement therapies. Accordingly, regulatory agencies in the US and worldwide recommend shared decision-making for finalizing one’s choice of kidney replacement therapy. It is also recognized that the US needs to substantially increase home dialysis utilization to leverage its positive impacts on patient and healthcare cost-related outcomes. This perspective highlights how the routine clinical use of the recommended practice of shared decision-making can exist in synergy with the system’s goal for increased home dialysis use. It introduces a pragmatic provider checklist, The Nephrologist’s Shared Decision-Making Checklist, grounded in the relevant theories of shared decision-making, and, unlike some research assessments and extant tools, is easy to understand and implement in clinical practice. This qualitative Checklist can help providers ensure that they have co-constructed an SDM experience with the patient and involved caretakers, helping them benefit from the improved outcomes associated with SDM.

Improving adherence to guidelines for spine pain care: what tools could support primary care clinicians in conforming to guidelines?

BackgroundSpine pain is one of the most common conditions seen in primary care and is often treated with ineffective, aggressive interventions, such as prescription pain medications, imagery and referrals to...

Abstract 5866: Racial ethnic variations in trajectory of health-related quality of life in older women with breast cancer

Abstract The purpose of the research study was to explore the ethnic/racial variations in health-related quality of life (HRQoL) scores, and the trajectory of comorbidities in older women with breast cancer. This study analyzed data from the Surveillance, Epidemiology, and End-Results (SEER) – Medicare Health Outcomes Survey (MHOS) linked data resource. The study population consists of 982 women (≥ 65 years old at diagnosis) with primary breast cancer who were diagnosed in 1998-2012 and completed the MHOS survey within 24 months before diagnosis and completed at least one more survey after diagnosis. The study population was grouped into three ethnic/racial groups: Whites (n=791), Black/African American (n=104) and Hispanics (n=87). The mean age of the population was 74.8 ± 6.1. HRQoL was measured using SF-36/VR-12 questionnaire and summarized as Physical Component Summary (PCS) Score and a Mental Component Summary (MCS) Score. Results showed an increase in the PCS score at month 6 after diagnosis, and a decrease at month 12 was observed in Whites and Black/African Americans. A continuous decrease in the PCS score till month 12 and an increase at month 24 was observed in Hispanics. All three groups showed similar trends in MCS scores over time - the MCS score dropped at month 6 but was back at baseline by month 12. The Whites had the highest MCS score out of the three, and Hispanics had the lowest. The MCS score for Hispanics decreased by month 36, whereas it increased for Black/African Americans. The MCS score for Whites remained fairly the same. The results also showed an increase in the total number of comorbidities at month six after diagnosis, along with a decrease to the baseline comorbidities by month 12. Hispanics had the highest increase in comorbidities at month 6 and Whites had the lowest and most stable. The number of comorbidities remained fairly the same in Whites and Black/African Americans, whereas the number of comorbidities increased slightly at month 24 for Hispanics and drastically increased at month 36. Overall, changes in the total number of comorbidities had a negative correlation with the changes in MCS scores, therefore it can be concluded that the number of comorbidities have a negative effect on a patient’s mental health. Whites were observed to have less comorbidities and better MCS scores than Black/African Americans or Hispanics. The information gained from this study will help clinicians and patients in the shared treatment decision process and guide individualized survivorship care plans among older women with breast cancer. Citation Format: Sushantti Rupesh, Eunkyung Lee. Racial ethnic variations in trajectory of health-related quality of life in older women with breast cancer [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2022; 2022 Apr 8-13. Philadelphia (PA): AACR; Cancer Res 2022;82(12_Suppl):Abstract nr 5866.

Making space for patients’ preferences in precision medicine: a qualitative study exploring perspectives of patients with rheumatoid arthritis

ObjectivePrecision medicine in rheumatoid arthritis (RA) creates new opportunities to involve patients in early identification of accurate indicators of health trajectories. The aim of this study was to explore patient...

The Role of Multimodality Imaging for Percutaneous Coronary Intervention in Patients With Chronic Total Occlusions.

BackgroundPercutaneous coronary intervention (PCI) of Chronic total occlusions (CTOs) has been traditionally considered a challenging procedure, with a lower success rate and a higher incidence of complications compared to non-CTO-PCI. An accurate and comprehensive evaluation of potential candidates for CTO-PCI is of great importance. Indeed, assessment of myocardial viability, left ventricular function, individual risk profile and coronary lesion complexity as well as detection of inducible ischemia are key information that should be integrated for a shared treatment decision and interventional strategy planning. In this regard, multimodality imaging can provide combined data that can be very useful for the decision-making algorithm and for planning percutaneous CTO recanalization.AimsThe purpose of this article is to appraise the value and limitations of several non-invasive imaging tools to provide relevant information about the anatomical characteristics and functional impact of CTOs that may be useful for the pre-procedural assessment and follow-up of candidates for CTO-PCI. They include echocardiography, coronary computed tomography angiography (CCTA), nuclear imaging, and cardiac magnetic resonance (CMR). As an example, CCTA can accurately delineate CTO location and length, distal coronary bed, vessel tortuosity and calcifications that can predict PCI success, whereas stress CMR, nuclear imaging and stress-CT can provide functional evaluation in terms of myocardial ischemia and viability and perfusion defect extension.

Explicating Integrative and Declarative Shared Treatment Decision Making in Rehabilitation Encounters When Patients are Without a Voice: A Grounded Theory Analysis

<h3>Research Objectives</h3> To describe the extent of collaboration in everyday treatment decisions between practitioners (e.g., rehabilitation therapists, physiatrists) and family caregivers of a patient with disorders of consciousness (DoC). <h3>Design</h3> Ethnographic approach used rehabilitation observations triangulated with narrative interviews. <h3>Setting</h3> Observations of rehabilitation encounters occurred in a level I trauma hospital. Interviews occurred either virtually or in-person at a mutually agreed upon location. <h3>Participants</h3> 14 encounters among rehabilitation practitioners (n=8), family caregivers (n=2), and adult patients with disorders of consciousness (n=2). Interviews with family caregivers (n=17) and rehabilitation practitioners (n=7). <h3>Interventions</h3> Not applicable. <h3>Main Outcome Measures</h3> Not applicable. <h3>Results</h3> Data were analyzed using open coding and constant comparison techniques based in a grounded theory approach. 88 interactions were identified across 14 rehabilitation encounters. For analysis, we defined micro-decisions as small-scale treatment decisions that often occur multiple times within a rehabilitation encounter. Each rehabilitation encounter had multiple treatment activities and micro-decisions at the patient's bedside. Two types of shared treatment decisions were identified—integrative and declarative. Integrative decision-making occurred when family were included in the treatment session and their unsolicited contributions provided new information for in-the-moment decision-making. Declarative decision making occurred when the practitioner elicited the patient's values, preferences, and/or priorities from the family and then the practitioner executed these preferences within the session. Identifying and describing two ways in which sharing treatment decisions occurs makes the tacit explicit. <h3>Conclusions</h3> Because individuals in DoC cannot advocate for themselves, partnership between the rehabilitation practitioners and family caregivers is the hallmark of rehabilitation treatments with these individuals. Explicating two types of shared treatment decision-making informs the rehabilitation field so that we can communicate the process of engaging families as partners in rehabilitation treatment decisions. <h3>Author(s) Disclosures</h3> No known conflicts.