Incorporating Patient Preferences Research Articles

Acceptability of intramuscular injection of tranexamic acid in postpartum hemorrhage prevention

Newer research comparing routes of medication administration has extended beyond efficacy as a primary endpoint to incorporate patient preference. However, little is known about the preferences of pregnant women in terms of routes of medication administration, specifically with regards to hemorrhage prevention and control. This study aimed to understand the preferences of pregnant women in terms of medical interventions to prevent hemorrhage at the time of delivery. Surveys were distributed from April 2022 to September 2022 using electronic tablets at a single urban center with an annual delivery volume of 3000 women per year to women >18 years of age who were either currently pregnant or have been pregnant in the past. Subjects were asked to choose their preferred route of administration from the following options: intravenous, intramuscular, or subcutaneous. The primary outcome was patient preference on the route of medication administration during a hemorrhage event. The study cohort included 300 patients, mostly African American (39.8%) followed by White (32.1%), and the majority of the participants ranged from 30 to 34 years of age (31.7%). When asked which method of administration they would prefer to prevent hemorrhage before birth, the results were as follows: 31.1% would prefer intravenous, 23.0% had no preference, 21.2% were unsure, 15.9% preferred subcutaneous, and 8.8% preferred intramuscular administration. In addition, 69.4% of respondents reported that they have never declined or avoided intramuscular administration of medication if recommended by their physician. Although some survey participants preferred an intravenous route of administration, 68.9% of subjects were unsure, had no preference, or preferred nonintravenous routes. This information is helpful particularly in low-resource settings where intravenous treatments are not readily available or in urgent clinical situations in which intravenous administration routes are not easily obtainable in high-risk patients.

Acceptability of patient-centered, multi-disciplinary medication therapy management recommendations: results from the INCREASE randomized study

BackgroundPolypharmacy and inappropriate medications may be a modifiable risk factor for Alzheimer’s Disease and Related Dementias (ADRD). Medication therapy management (MTM) interventions may mitigate medication-induced cognitive dysfunction and delay onset of symptomatic impairment. The objective of the current study is to describe an MTM protocol for a patient-centered team intervention (pharmacist and non-pharmacist clinician) in a randomized controlled trial (RCT) directed at delaying the symptomatic onset of ADRD.MethodsCommunity dwelling adults 65 + years, non-demented, using ≥ 1 potentially inappropriate medications (PIM) were enrolled in an RCT to evaluate the effect of an MTM intervention on improving medication appropriateness and cognition (NCT02849639). The MTM intervention involved a three-step process: (1) pharmacist identified potential medication-related problems (MRPs) and made initial recommendations for prescribed and over-the-counter medications, vitamins, and supplements; (2) study team reviewed all initial recommendations together with the participants, allowing for revisions prior to the finalized recommendations; (3) participant responses to final recommendations were recorded. Here, we describe initial recommendations, changes during team engagement, and participant responses to final recommendations.ResultsAmong the 90 participants, a mean 6.7 ± 3.6 MRPs per participant were reported. Of the 259 initial MTM recommendations made for the treatment group participants (N = 46), 40% percent underwent revisions in the second step. Participants reported willingness to adopt 46% of final recommendations and expressed need for additional primary care input in response to 38% of final recommendations. Willingness to adopt final recommendations was highest when therapeutic switches were offered and/or with anticholinergic medications.ConclusionThe evaluation of modifications to MTM recommendations demonstrated that pharmacists’ initial MTM recommendations often changed following the participation in the multidisciplinary decision-making process that incorporated patient preferences. The team was encouraged to see a correlation between engaging patients and a positive overall response towards participant acceptance of final MTM recommendations.Trial registrationStudy registration number: clinicaltrial.gov NCT02849639 registered on 29/07/2016.

Use of Bayesian decision analysis to maximize value in patient-centered randomized clinical trials in Parkinson’s disease

ABSTRACT A fixed one-sided significance level of 5% is commonly used to interpret the statistical significance of randomized clinical trial (RCT) outcomes. While it is necessary to reduce the false positive rate, the threshold used could be chosen quantitatively and transparently to specifically reflect patient preferences regarding benefit–risk tradeoffs as well as other considerations. How can patient preferences be explicitly incorporated into RCTs in Parkinson’s disease (PD), and what is the impact on statistical thresholds for device approval? In this analysis, we apply Bayesian decision analysis (BDA) to PD patient preference scores elicited from survey data. BDA allows us to choose a sample size () and significance level () that maximizes the overall expected value to patients of a balanced two-arm fixed-sample RCT, where the expected value is computed under both null and alternative hypotheses. For PD patients who had previously received deep brain stimulation (DBS) treatment, the BDA-optimal significance levels fell between 4.0% and 10.0%, similar to or greater than the traditional value of 5%. Conversely, for patients who had never received DBS, the optimal significance level ranged from 0.2% to 4.4%. In both of these populations, the optimal significance level increased with the severity of the patients’ cognitive and motor function symptoms. By explicitly incorporating patient preferences into clinical trial designs and the regulatory decision-making process, BDA provides a quantitative and transparent approach to combine clinical and statistical significance. For PD patients who have never received DBS treatment, a 5% significance threshold may not be conservative enough to reflect their risk-aversion level. However, this study shows that patients who previously received DBS treatment present a higher tolerance to accept therapeutic risks in exchange for improved efficacy which is reflected in a higher statistical threshold.

Abstract P4-07-22: Clinical decision support systems for multidisciplinary team decision-making in patients with solid cancer: an implementation model based on a scoping review

Abstract Background Clinical decision-making by multidisciplinary teams (MDTs) is getting more complex as treatment advice for the individual patient must be based on an increasing amount of patient and tumor characteristics, and scientific evidence on treatment efficacy. Clinical decision support systems (CDSSs) can make an important contribution to assist but also optimize MDT decision-making. However, implementation of CDSSs in clinical practice is challenging. Aim & methods The aim of our study is to set up a CDSSs implementation model for multidisciplinary decision-making in solid cancer. It is based on a scoping review of the currently reported CDSSs for MDT decision-making in solid cancers with identification of reported barriers and facilitators for implementation of these CDSSs. For this we systematically searched the Cochrane Library, MEDLINE (accessed through PubMed) and Scopus up to September 1st 2021. Results Of the 710 screened abstracts, 38 papers met the inclusion criteria (table 1). Sixteen different CDSSs were identified. For implementation of CDSSs, 87 barriers and 73 facilitators were reported. The reported barriers could be categorized in the same categories as those of the facilitators (a factor can be reported as a barrier if the factor is not addressed well, and as a facilitator if the factor is properly addressed). The most frequently reported barriers for CDSS implementation for MDT decision making mainly concerned CDSS maintenance (e.g. not incorporating guideline updates), loco-regional feasibility of the CDDS recommendation (e.g. no access to diagnostics or treatment), validity, not incorporating patient preference in decision making, data accuracy, noncoverage of certain patient subpopulations, lack of an information standard, usability, data availability and no interoperability of the CDSS with the electronic health record. The most frequently reported facilitators included, besides the categories as mentioned above, the category shared decision making (reporting of alternative treatment options) and technical skills (involvement of a computer scientist). Table 2 shows the most frequently reported categories of barriers and facilitators, and scores for each included study the number of reported barriers (B) and facilitators (F) in each category. Conclusion Based on the identified barriers and facilitators, we developed a CDSS implementation model to guide more successful CDSS integration in the clinical workflow to support MDTs (the model will be shown at the congress). The usability of this theoretical model should be explored in future studies. Table 1. Characteristics of 38 included articles Table 2. Overview of the most frequently reported categories of barriers and facilitators. For each included study the number of reported barriers (B) and facilitatiors (F) are scored for each category. Citation Format: Mathijs P. Hendriks, Kees C. Ebben, Janine A. Van Til, Agnes Jager, Sabine Siesling. Clinical decision support systems for multidisciplinary team decision-making in patients with solid cancer: an implementation model based on a scoping review [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 P4-07-22.

Implementation Science in Surgery: Translating Outcomes to Action.

Implementation Science in Surgery: Translating Outcomes to Action.

P600 Democratic deliberation to assess Veteran preferences for biosimilar medication switching for the treatment of Inflammatory Bowel Disease in a resource-conscious setting.

Abstract Background Optimizing care while being resource-conscious, the Veterans Health Administration (VHA) must develop equitable policies incorporating Veterans’ values and beliefs. Switching patients with Inflammatory Bowel Disease (IBD) from biologic medications (originators) to less expensive biosimilars offers opportunities to explore potential policies. Unlike originators, the FDA did not require disease-specific randomized controlled trials for approval of biosimilars, raising provider and patient concerns about the safety of switching to these medications. Deliberative democratic (DD) methods allow us to assess Veteran views of switching programs, a necessary step in creating equitable policies. Methods We carried out two 2-day sessions with Veterans from the Ann Arbor VA (n=17) and Houston VA (n=12). Deliberation began with education about IBD, the use of biologics (including biosimilars), and related implementation challenges. Participants engaged in facilitated small group discussions to 1) identify Veterans views about biosimilar switching and Veteran preferences about switching, and 2) evaluate five approaches for switching: (a) “status quo”–switching policies vary by VHA facility, (b) “sickest last”–Veterans with severe IBD are switched last, (c) “opt-out”–Veterans may opt-out of switching, (d) “next appointment”–Veterans are switched at follow-up, and (e) “lottery”–patients are switched based on random selection. Participants ranked options (1=most preferred, 5=least preferred) and proposed modifications to these approaches. Participants completed pre- and post-DD session surveys. Transcripts were analysed using qualitative methods. Results Participant demographics are in Table 1. Most Veterans sessions preferred the “sickest last” (mean rank: 1.8, standard deviation: 0.9) or “opt-out” (2.4,1.4) approaches over the “status quo” (3.1,1.0) or “lottery” (4.6,0.9); preferences did not significantly change after deliberation (Figure 1). Preferences reflected Veterans’ concerns about the risk of switching to biosimilars for the most vulnerable Veterans and their strong preference for transparency around biosimilar switching. Participants emphasized the importance of informed choice, prioritizing based on health status, personalization, and shared decision-making. Conclusion DD methods are effective for soliciting Veterans' opinions and preferences for health policies. Veterans favoured policies that provided information and control, promoted shared decision-making, and reduced risk to vulnerable patients. Incorporating patient preferences and insights is critical when implementing health policies in the VHA and other resource-limited settings.

DEMOCRATIC DELIBERATION TO ASSESS VETERAN PREFERENCES FOR BIOSIMILAR MEDICATION SWITCHING FOR THE TREATMENT OF INFLAMMATORY BOWEL DISEASE IN A RESOURCE CONSCIOUS SETTING

Abstract BACKGROUND To optimize care while being resource conscious, the Veterans Health Administration (VHA) must develop equitable policies that incorporate Veterans’ values and beliefs. Switching patients with Inflammatory Bowel Disease (IBD) from biologic medications (originators) to less expensive biosimilars offers the opportunity to explore potential policies. Unlike originators, the FDA did not require disease-specific randomized controlled trials for approval of biosimilars, creating provider and patient concerns about the safety of switching to these medications. Deliberative democratic (DD) methods allow us to assess Veteran views of switching programs, a necessary step in creating equitable policy approaches. METHODS We carried out two 2-day sessions with Veterans from the Ann Arbor VA (n=17) and Houston VA (n=12). Deliberation began with education about IBD, use of biologics (including biosimilars), and related implementation challenges. Participants engaged in facilitated small group discussions designed to 1) identify Veterans views about biosimilar switching and their preferences for informing Veterans about switching, and 2) evaluate responses to five approaches for switching: (a) “status quo”–switching policies vary by VHA facility, (b) “sickest last”–Veterans with the most severe IBD are switched last, (c) “opt-out”–Veterans may opt-out of switching, (d) “next appointment”–Veterans are switched at their next appointment, and (e) “lottery”–patients are switched based on random selection. Participants ranked options (1=most preferred, 5=least preferred) and proposed modifications to these approaches. Participants completed pre- and post-DD session surveys. Discussion transcripts were analyzed using qualitative methods. RESULTS Participant demographics are in Table 1. Most Veterans sessions preferred the “sickest last” (mean rank: 1.8, standard deviation: 0.9) or “opt-out” (2.4,1.4) approaches over the “status quo” (3.1,1.0) or “lottery” (4.6,0.9); preferences did not significantly change after deliberation (Figure 1). Preferences reflected Veterans’ concerns about the risk of switching to biosimilars for the most vulnerable Veterans and their strong preference for information and transparency around biosimilar switching. Participants emphasized the importance of informed choice, prioritizing based on health status, personalization, and shared decision-making (e.g., treatment should be geared to individual patient in discussion with their provider). CONCLUSION DD methods are effective for soliciting Veterans' opinions and preferences for health policies. Veterans favored policies that provided information and control, promoted shared decision-making, and reduced risk to vulnerable patients. Incorporating patient preferences and insights is critical when implementing health policies in the VHA and other resource-limited settings.

Individualized Net Benefit estimation and meta-analysis using generalized pairwise comparisons in N-of-1 trials.

The Net Benefit (Δ) is a measure of the benefit-risk balance in clinical trials, based on generalized pairwise comparisons (GPC) using several prioritized outcomes and thresholds of clinical relevance. We extended Δ to N-of-1 trials, with a focus on patient-level and population-level Δ. We developed a Δ estimator at the individual level as an extension of the stratum-specific Δ, and at the population-level as an extension of the stratified Δ. We performed a simulation study mimicking PROFIL, a series of 38 N-of-1 trials testing sildenafil in Raynaud's phenomenon, to assess the power for such an analysis with realistic data. We then reanalyzed PROFIL using GPC. This reanalysis was finally interpreted in the context of the main analysis of PROFIL which used Bayesian individual probabilities of efficacy. Simulations under the null showed good size of the test for both individual and population levels. The test lacked power when being simulated from the true PROFIL data, even when increasing the number of repetitions up to 140 days per patient. PROFIL individual-level estimated Δ were well correlated with the probabilities of efficacy from the Bayesian analysis while showing similarly wide confidence intervals. Population-level estimated Δ was not significantly different from zero, consistently with the previous Bayesian analysis. GPC can be used to estimate individual Δ which can then be aggregated in a meta-analytic way in N-of-1 trials. GPC ability to easily incorporate patient preferences allow for more personalized treatment evaluation, while needing much less computing time than Bayesian modeling.

Effect of standardised surgical assessment and shared decision-making on morbidity and patient satisfaction after breast conserving therapy: A cross-sectional study.

The role of oncoplastic breast conserving therapy (OPBCT) on physical function, morbidity and patient satisfaction has yet to be defined. Additionally, technique selection should be individualised and incorporate patient preference. The study aim was to investigate differences between "standard" (sBCT) and oncoplastic breast conservation (OPBCT) in patient-reported outcomes (PROs) when patients have been assessed in a standardised manner and technique selection has been reached through shared decision-making (SDM). This is a cross-sectional study of 215 women treated at a tertiary referral centre. Standardised surgical assessment included breast and lesion volumetry, definition of resection ratio, patient-related risk factors and patient preference. Postoperative morbidity and patient satisfaction were assessed by validated PROs tools (Diseases of the Arm, Shoulder and Hand-DASH and Breast-Q). Patient experience was assessed by semi-structured interviews. There was no difference of the median values between OPBCT and sBCT in postoperative morbidity of the upper extremity (DASH 3.3 vs 5, p=0.656) or the function of the chest wall (Breast-Q 82 vs 82, p=0.758). Postoperative satisfaction with breasts did not differ either (Breast-Q 65 vs 61, p=0.702). On the individual level, women that opted for OPBCT after SDM had improved satisfaction when compared to baseline (+3 vs-1, p=0.001). Shared decision-making changed patient attitude in 69.8% of patients, leading most often to de-escalation from mastectomy. These findings support that a combination of standardised surgical assessment and SDM allows for tailored treatment and de-escalation of oncoplastic surgery without negatively affecting patient satisfaction and morbidity.

993. “I feel like they’re actually listening to me”: A Pilot Study of Hospital Discharge-Decision Making for Patients with Injection Drug Use-Associated Infections

Abstract Background Conversation guides have the potential to transform care for patients hospitalized with injection drug use (IDU)-associated infections. This study’s objectives were to 1) pilot a structured conversation guide for hospital discharge decision making in patients with IDU-associated infections 2) investigate the guide’s feasibility and acceptability and 3) examine patient and provider experiences, patient outcomes, and lessons learned. Methods We developed a conversation guide and conducted semi-structured interviews with physicians and patients at a tertiary care center in Maine. We interviewed physicians after each piloted the guide with two patients. We interviewed patients less than one week after the conversation and again after 4-6 weeks. Two analysts indexed transcriptions and used the framework method to identify and organize relevant information. We conducted retrospective chart review to corroborate and contextualize qualitative data. Results Eight patients and four infectious disease physicians piloted the conversation guide. All patients (N=8) completed antimicrobial treatment and 88% were discharged on medication for opioid use disorder (Table 1). All providers and most patients stated that the conversation guide was important for incorporating patient preferences and antimicrobial treatment options. Patients appreciated more autonomy and their voices being included in their care. Providers felt the guide facilitated their understanding of patient values. Values and preferences between patients and providers were aligned. Participants identified the length of the guide, discussion of pain management, and addressing post-discharge needs such as housing as areas for improvement (Table 2). Conclusion The use of a conversation guide to inform hospital discharge decision making for patients with IDU-associated infections incorporates patient preferences and values into treatment decisions. While we identified areas for improvement, overall patients and providers believed that this novel conversation guide helped to improve patient care and autonomy. Disclosures Kinna Thakarar, DO, MPH, Maine Medical Center: Board Member|NIH: Grant/Research Support|University of New England: Board Member Monica K. Sikka, MD, F2G: Site research investigator Ellen Eaton, MD, MSPH, Gilead: Grant/Research Support.

What is the role of up-front autologous stem cell transplantation in mantle cell lymphoma?

Up-front autologous stem cell transplantation (ASCT) is the established standard of care for younger, transplant-eligible MCL patients and is associated with a prolonged progression-free survival (PFS) benefit. However, there is no randomized controlled trial data, with therapy including rituximab and cytarabine, that has established a PFS and overall survival (OS) benefit with ASCT in the modern era. Multiple retrospective studies have failed to identify an OS benefit associated with ASCT in younger MCL patients. The high-risk patient subgroup with evidence of baseline TP53 mutation has a dismal outcome with intensive chemoimmunotherapy followed by ASCT, thus up-front ASCT is not optimal for this patient subset. Ongoing randomized clinical trials will help to clarify the role of up-front ASCT in the future. For example, the ongoing European MCL Network Triangle study incorporating ibrutinib into chemoimmunotherapy induction and maintenance with and without ASCT will help define the role of ASCT in the era of novel biologically targeted agents (ClinicalTrials.gov identifier: NCT02858258). Additionally, minimal residual disease (MRD) assessment is a powerful prognostic tool in MCL, and the ongoing Eastern Cooperative Oncology Group-American College of Radiology Imaging Network E4151 study is comparing maintenance rituximab alone vs ASCT consolidation in MCL patients who achieve remission and MRD-undetectable status post induction (ClinicalTrials.gov identifier: NCT03267433). ASCT remains a highly efficacious initial therapy for younger MCL patients; however, ultimately the decision to pursue ASCT requires discussion of risks vs benefits, incorporating patient preferences and values.

Incorporating patient preferences and burden-of-disease in evaluating ALS drug candidate AMX0035: a Bayesian decision analysis perspective

Objective Provide US FDA and amyotrophic lateral sclerosis (ALS) society with a systematic, transparent, and quantitative framework to evaluate the efficacy of the ALS therapeutic candidate AMX0035 in its phase 2 trial, which showed statistically significant effects (p-value 3%) in slowing the rate of ALS progression on a relatively small sample size of 137 patients. Methods We apply Bayesian decision analysis (BDA) to determine the optimal type I error rate (p-value) under which the clinical evidence of AMX0035 supports FDA approval. Using rigorous estimates of ALS disease burden, our BDA framework strikes the optimal balance between FDA’s need to limit adverse effects (type I error) and patients’ need for expedited access to a potentially effective therapy (type II error). We apply BDA to evaluate long-term patient survival based on clinical evidence from AMX0035 and Riluzole. Results The BDA-optimal type I error for approving AMX0035 is higher than the 3% p-value reported in the phase 2 trial if the probability of the therapy being effective is at least 30%. Assuming a 50% probability of efficacy and a signal-to-noise ratio of treatment effect between 25% and 50% (benchmark: 33%), the optimal type I error rate ranges from 2.6% to 26.3% (benchmark: 15.4%). The BDA-optimal type I error rate is robust to perturbations in most assumptions except for a probability of efficacy below 5%. Conclusion BDA provides a useful framework to incorporate subjective perspectives of ALS patients and objective burden-of-disease metrics to evaluate the therapeutic effects of AMX0035 in its phase 2 trial.

There’s no place like home: Home time among older adults with AML by therapy intensity.

376 Background: Older adults with acute myeloid leukemia (AML) have identified home time (HT) as a critical outcome influencing their treatment selection. However, few studies have characterized HT in this disease setting. Methods: We conducted a retrospective, observational study of adults age ≥60 diagnosed with AML from 2015 to 2020 at University of North Carolina Health. First-line therapy was classified by intensity: low (hypomethylating agent [HMA]), intermediate (HMA+Venetoclax), and high (anthracycline-based). The primary outcome was cumulative HT days from time of diagnosis. A day was defined as HT if an individual was not hospitalized and did not utilize emergency department services or ambulatory oncology clinics. Secondary outcomes were proportion of days at home (PDH) and overall survival (OS). Covariates included demographics and disease risk by ELN 2017 criteria. HT was evaluated via summary statistics with comparisons among treatment groups made via linear (for HT / PDH) or Cox proportional hazard (for OS) regression models adjusted for age & ELN risk. PDH was further adjusted for log of follow up time. Median OS was calculated via the Kaplan-Meier method. Results: 197 individuals were identified. Mean age was 71 (range 60-95), 79% were white, and 59% male. 38% received high-intensity therapy, 33% received low, and 29% intermediate. Mean age was lower in the high-intensity group (66 vs 74 in low, 75 in intermediate; Kruskal-Wallis p < 0.0001). Mean HT was 284 days, and median survival was 10.3 months (95% CI 8.4-12.7). Mean HT was greater in the high-intensity group at 409 days (CI 310-508) vs. 243 (158-328) in low and 169 (121-216) in the intermediate group (Table). The high-intensity group had greater median OS at 19.9 months vs. 8.8 (low) or 7.7 (intermediate) but a lower mean adjusted PDH (high: 0.60 vs. low: 0.73 vs. intermediate: 0.75; p < 0.0001). In adjusted models, these differences remained statistically significant. Mean HT, median OS, and mean adjusted PDH did not differ significantly between the low & intermediate groups. Conclusions: Older adults with AML spend a tremendous amount of time - roughly 40% of days - engaged in oncology care. Although receiving high-intensity therapy was associated with longer OS, increases in HT were more modest, indicating that much of the survival gains were spent engaged in care. Shared treatment decision-making should incorporate patient preferences for securing HT versus attempting to prolong survival.[Table: see text]

The development and utility of a multicriteria patient decision aid for people contemplating treatment for osteoarthritis.

There are a range of treatment options for osteoarthritis (OA) of the knee and hip, each with a unique profile of risks and benefits. Patient decision aids can help incorporate patient preferences in treatment decision-making. The aim of this study was to develop and test the utility of a patient decision aid for OA that was developed using a multicriteria decision analytic framework. People contemplating treatment for OA who had accessed the website myjointpain.org.au were invited to participate in the study by using the online patient decision aid. Two forms of the patient decision aid were created: A shorter form and a longer form, which allowed greater customization that was offered to respondents after they had completed the shorter form. Respondents also completed questions asking about their experience using the patient decision aid. A total of 625 self-selected respondents completed the short-form and 180 completed the long-form. Across both forms, serious side effects, pain and function were rated as the most important treatment outcomes. Most respondents (64%) who completed the longer form reported that using the tool was a positive experience, 38% reported that using the tool had changed their mind and 48% said that using the tool would improve the quality of their decision-making. Overall, the findings suggest that this patient decision aid may be of use to a substantial number of people in facilitating appropriate treatment decision-making. Service users of myjointpain.org.au were involved through their participation in the study, and their feedback will guide the development of future iterations of the tool.

Long-term oncological outcomes after local excision of T1 rectal cancer

BackgroundA growing proportion of patients with early rectal cancer is treated by local excision only. The aim of this study was to evaluate long-term oncological outcomes and the impact of local recurrence on overall survival for surgical local excision in pT1 rectal cancer.MethodsPatients who only underwent local excision for pT1 rectal cancer between 1997 and 2014 in two Dutch tertiary referral hospitals were included in this retrospective cohort study. The primary outcome was the local recurrence rate. Secondary outcomes were distant recurrence, overall survival and the impact of local recurrence on overall survival.ResultsA total of 150 patients (mean age 68.5 ± 10.7 years, 57.3% males) were included in the study. Median length of follow-up was 58.9 months (range 6–176 months). Local recurrence occurred in 22.7% (n = 34) of the patients, with a median time to local recurrence of 11.1 months (range 2.3–82.6 months). The vast majority of local recurrences were located in the lumen. Five-year overall survival was 82.0%, and landmark analyses showed that local recurrence significantly impacted overall survival at 6 and 36 months of follow-up (6 months, p = 0.034, 36 months, p = 0.036).ConclusionsLocal recurrence rates after local excision of early rectal cancer can be substantial and may impact overall survival. Therefore, clinical decision-making should be based on patient- and tumour characteristics and should incorporate patient preferences.

Patient-Preference Diagnostics: Adapting Stated-Preference Methods to Inform Effective Shared Decision Making.

While clinical practice guidelines underscore the need to incorporate patient preferences in clinical decision making, incorporating meaningful assessment of patient preferences in clinical encounters is challenging. Structured approaches that combine quantitative patient preferences and clinical evidence could facilitate effective patient-provider communication and more patient-centric health care decisions. Adaptive conjoint or stated-preference approaches can identify individual preference parameters, but they can require a relatively large number of choice questions or simplifying assumptions about the error with which preferences are elicited. We propose an approach to efficiently diagnose preferences of patients for outcomes of treatment alternatives by leveraging prior information on patient preferences to generate adaptive choice questions to identify a patient's proximity to known preference phenotypes. This information can be used for measuring sensitivity and specificity, much like any other diagnostic procedure. We simulated responses with varying levels of choice errors for hypothetical patients with specific preference profiles to measure sensitivity and specificity of a 2-question preference diagnostic. We identified 4 classes representing distinct preference profiles for patients who participated in a previous first-time anterior shoulder dislocation (FTASD) survey. Posterior probabilities of class membership at the end of a 2-question sequence ranged from 87% to 89%. We found that specificity and sensitivity of the 2-question sequences were robust to respondent errors. The questions appeared to have better specificity than sensitivity. Our results suggest that this approach could help diagnose patient preferences for treatments for a condition such as FTASD with acceptable precision using as few as 2 choice questions. Such preference-diagnostic tools could be used to improve and document alignment of treatment choices and patient preferences. Approaches that combine patient preferences and clinical evidence can facilitate effective patient-provider communication and more patient-centric healthcare decisions. However, diagnosing individual-level preferences is challenging, and no formal diagnostic tools exist.We propose a structured approach to efficiently diagnose patient preferences based on prior information on the distribution of patient preferences in a population.We generated a 2-question test of preferences for the outcomes associated with the treatment of first-time anterior shoulder dislocation.The diagnosis of preferences can help physicians discuss relevant aspects of the treatment options and proactively address patient concerns during the clinical encounter.

An updated evaluation of avatrombopag for the treatment of chronic immune thrombocytopenia

ABSTRACT Introduction Multiple agents are available for the management of chronic immune thrombocytopenia (ITP), including thrombopoietin-receptor agonists (TPO-RAs), rituximab, and fostamatinib. Although TPO-RAs are often selected as treatments for chronic ITP, when choosing between the TPO-RAs, clinicians must balance safety profile, dosing restrictions, and method of administration incorporating patient preference. Areas covered We provide an overview of the thrombopoietin receptor agonists with a particular focus on avatrombopag, the newest agent in this class. In phase II and III clinical trials, avatrombopag was shown to offer durable improvement in platelet counts. We also include recent real-world evidence describing avatrombopag effectiveness in patients with poor response to prior treatments (including other TPO-RAs). Expert opinion Compared with other TPO-RAs used to treat ITP, avatrombopag offers practical oral dosing with a single pill strength, does not require long-term dietary restrictions, and has no warning for hepatotoxicity. It is frequently effective after use of other TPO-RAs in ITP. The primary downside with avatrombopag use at present is the lack of longer-term safety data in ITP that presently exists for romiplostim and eltrombopag.

Patient Medication Preferences for Managing Dry Eye Disease: The Importance of Medication Side Effects.

ObjectivesThe side effects of dry eye medications can lead to medication non-adherence and, eventually, to poor outcomes. This study aimed to quantify to what extent the side effects of dry eye disease (DED) medications (burning/stinging sensation and blurring) are important to patients compared to medication benefits or costs.MethodsPatients diagnosed with DED were recruited at a referral eye center in Singapore (n = 139). This study utilized a Discrete Choice Experiment where patients were presented with 10 choice tasks where they were asked to choose between their current medication (or no medication), and two hypothetical medications that varied based on five attributes: duration of burning/stinging, duration of blurring, time to medication effectiveness, medication frequency, and out-of-pocket cost. The main outcomes were relative attribute importance and predicted uptake.ResultsLatent class logistic regressions found two groups with distinct preferences. For both classes, duration of burning/stinging (Class 1 = 23%, Class 2 = 29%) and cost (Class 1 = 24%, Class 2 = 27%) were the most important attributes while duration of blurring (Class 1 = 15%, Class 2 = 9%) was the least important. The predicted uptake of a medication increased 18 percentage-points when burning/stinging duration decreased from 2 h to a few minutes. The predicted uptake for new medications was lowest for those on medication with well-controlled symptoms and highest for those who were not on medication and could not control their symptoms effectively.ConclusionThis study showed that duration of burning/stinging was an important factor when choosing medications. Incorporating patient preferences in medication decisions can potentially improve patient acceptance of a treatment regimen.Supplementary InformationThe online version contains supplementary material available at 10.1007/s40271-022-00586-8.

Waldenstrom Macroglobulinemia: Tailoring Therapy for the Individual.

With the introduction of multiple new effective therapeutic options for the treatment of macroglobulinemia, a structured approach to management of this rare lymphoma is currently needed. A review of phase II and III treatment trials over the past 20 years was performed, and high-quality trials are summarized in this manuscript. Because of the lack of large prospective trials comparing different classes of therapy, a uniform recommendation applicable to all patients cannot be made, and the approach must be individualized incorporating patient preferences, comorbidities, and the range of therapeutic toxicities. Therapeutic options for patients with newly diagnosed and previously treated macroglobulinemia are presented on the basis of the best available evidence in the literature.

A patient perspective on cure in multiple myeloma: A survey of over 1,500 patients.

8046 Background: As treatments advance in multiple myeloma (MM) and increasingly deeper and longer responses are achieved, the definition of what a cure is becomes increasingly relevant. While many physicians quote that patients may achieve a “functional cure”, understanding the patient perspective on the concept of cure has yet to be explored. Methods: HealthTree Cure Hub by the HealthTree Foundation represents an online portal for patients with plasma cell dyscrasias to help navigate their disease. It is the largest single database of patients with multiple myeloma with over 10,200 patients as of January 2022. Using this platform, we surveyed patients online from November 11th 2021 to Feb 7th 2022. Varying scenarios incorporating toxicity, disease status, and being on/off treatment were presented, and participants were asked to rate them from 1-5, with 5 being an ideal cure. Patient awareness of the term functional or operational cure was also. Results: A total of 1525 participants completed the survey. Table lists characteristics of the patients who completed this survey. The majority of the patients were female (55.5%), college educated (88.5%) and non-Hispanic White (70.7%). Most patients rated being off treatment permanently and having no evidence of disease as a cure (1116/1469, 75.5%), with a median score of 5 amongst respondents, indicating an ideal version of cure. Continuing to take the same pill or injection with significant toxicity and no evidence of disease had a median score of 1, a score lower than either “continuing to take same pill or injection without significant toxicity even in the evidence of disease” (median score 2) or “stopping treatment permanently even with some detectable disease” (median score 2). The majority of patients (76.3%) reported being unfamiliar with the term functional or operational cure. Conclusions: In the first study of patients with plasma cell dyscrasias in which patients were asked about their perceptions of what a cure is, our results highlight that drug toxicity and being on treatment profoundly impacts patient's perception of cure. Furthermore, most patients are not familiar with the term functional or operational cure. Future efforts should recruit more diverse patient populations and incorporate patient preferences in approaches to defining cure in myeloma, as well as explaining cure in easily comprehensible terms to patients. [Table: see text]