Add-On Deep Brain Stimulation versus Continued Vagus Nerve Stimulation for Childhood Epilepsy (ADVANCE): A Partially Randomized Patient Preference Trial.

Outcomes following vagus nerve stimulation (VNS) improve over years after implantation in children with drug-resistant epilepsy. The added value of deep brain stimulation (DBS) instead of continued VNS optimization is unknown. In a prospective, non-blinded, randomized patient preference trial of 18 children (aged 8-17 years) who did not respond to VNS after at least 1 year, add-on DBS resulted in greater seizure reduction compared with an additional year of VNS optimization (51.9% vs. 12.3%, p = 0.047). Add-on DBS also resulted in less bothersome seizures (p = 0.03), but no change in quality of life. DBS may be considered earlier for childhood epilepsy after non-response to VNS. ANN NEUROL 2024;96:405-411.

Merging machine learning and patient preference: a novel tool for risk prediction of percutaneous coronary interventions.

BACKGROUND AND AIMS: Predicting personalized risk for adverse events following percutaneous coronary intervention (PCI) remains critical in weighing treatment options, employing risk mitigation strategies, and enhancing shared decision-making. This study aimed to employ machine learning models using pre-procedural variables to accurately predict common post-PCI complications. METHODS: A group of 66 adults underwent a semiquantitative survey assessing a preferred list of outcomes and model display. The machine learning cohort included 107 793 patients undergoing PCI procedures performed at 48 hospitals in Michigan between 1 April 2018 and 31 December 2021 in the Blue Cross Blue Shield of Michigan Cardiovascular Consortium (BMC2) registry separated into training and validation cohorts. External validation was conducted in the Cardiac Care Outcomes Assessment Program database of 56 583 procedures in 33 hospitals in Washington. RESULTS: Overall rate of in-hospital mortality was 1.85% (n = 1999), acute kidney injury 2.51% (n = 2519), new-onset dialysis 0.44% (n = 462), stroke 0.41% (n = 447), major bleeding 0.89% (n = 942), and transfusion 2.41% (n = 2592). The model demonstrated robust discrimination and calibration for mortality {area under the receiver-operating characteristic curve [AUC]: 0.930 [95% confidence interval (CI) 0.920-0.940]}, acute kidney injury [AUC: 0.893 (95% CI 0.883-0.903)], dialysis [AUC: 0.951 (95% CI 0.939-0.964)], stroke [AUC: 0.751 (95%CI 0.714-0.787)], transfusion [AUC: 0.917 (95% CI 0.907-0.925)], and major bleeding [AUC: 0.887 (95% CI 0.870-0.905)]. Similar discrimination was noted in the external validation population. Survey subjects preferred a comprehensive list of individually reported post-procedure outcomes. CONCLUSIONS: Using common pre-procedural risk factors, the BMC2 machine learning models accurately predict post-PCI outcomes. Utilizing patient feedback, the BMC2 models employ a patient-centred tool to clearly display risks to patients and providers (https://shiny.bmc2.org/pci-prediction/). Enhanced risk prediction prior to PCI could help inform treatment selection and shared decision-making discussions.

Patient preferences in the treatment of stage III/IV classic Hodgkin lymphoma: Results from the CONNECT cross-sectional survey.

We explored patient front-line treatment preferences in newly diagnosed stage III/IV classic Hodgkin lymphoma (cHL). The CONNECT patient survey, administered online from 30 December 2020 to 1 March 2021, examined preferences overall and by age at diagnosis in 182 adult patients diagnosed with stage III/IV cHL within the past 10 years in the United States. At diagnosis, patients' median age was 36 years; 66% of patients were younger (aged 16-41 years) and 34% older (aged 42-85 years). When asked about initial treatment goals, 74% of patients ranked cure as their first or second goal (86% younger vs. 52% older patients; p < 0.001). At diagnosis, 72% of patients preferred aggressive treatment, and 85% were willing to accept more short-term risks in exchange for a better-working therapy long term. For long-term risks, younger versus older patients were significantly more concerned about second cancers (p < 0.001) and fertility issues (p = 0.007), whereas older patients were more concerned about lung damage (p = 0.028) and infections (p < 0.001). Most patients (94%) reported having a caregiver at some point, but 99% of these patients retained some control of treatment decisions. Collectively, these survey results highlight patient treatment preferences and differences in treatment goals and long-term side effect concerns based on patient age.

Patient Priorities Concerning Treatment Decisions for Advanced Neuroendocrine Tumors Identified by Discrete Choice Experiments.

BACKGROUND: Patients with advanced neuroendocrine tumors (NETs) have multiple treatment options. Ideally, treatment decisions are shared between physician and patient; however, previous studies suggest that oncologists and patients place different value on treatment attributes such as adverse event (AE) rates. High-quality information on NET patient treatment preferences may facilitate patient-centered decision making by helping clinicians understand patient priorities. METHODS: This study used 2 discrete choice experiments (DCE) to elicit preferences of NET patients regarding advanced midgut and pancreatic NET (pNET) treatments. The DCEs used the "potentially all pairwise rankings of all possible alternatives" (PAPRIKA) method. The primary objective was to determine relative utility rankings for treatment attributes, including progression-free survival (PFS), treatment modality, and AE rates. Ranking of attribute profiles matching specific treatments was also determined. Levels for treatment attributes were obtained from randomized clinical trial data of NET treatments. RESULTS: One hundred and 10 participants completed the midgut NET DCE, and 132 completed the pNET DCE. Longer PFS was the highest ranked treatment attribute in 64.5% of participants in the midgut NET DCE, and in 59% in the pNET DCE. Approximately, 40% of participants in both scenarios prioritized lower AE rates or less invasive treatment modalities over PFS. Ranking of treatment profiles in the midgut NET scenario identified 60.9% of participants favoring peptide receptor radionuclide therapy (PRRT), and 30.0% somatostatin analogue dose escalation. CONCLUSION: NET patients have heterogeneous priorities when choosing between treatment options based on the results of 2 independent DCEs. These results highlight the importance of shared decision making for NET patients.

A Feasibility Study Investigating an Exercise Program in Metastatic Cancer Based on the Patient-Preferred Delivery Mode.

BACKGROUND: Feasibility of exercise in patients with metastatic cancer is still a challenge. This study aimed to determine the feasibility and preliminary efficacy of an exercise intervention based on a patient-preferred delivery mode in patients affected by metastatic cancer. MATERIALS AND METHODS: Forty-four patients with a confirmed diagnosis of metastatic cancer were recruited in a 3-month exercise program. Whereas the exercise program consisted of aerobic and resistance activities performed twice a week, the participants may choose the mode of delivery: home based, personal training, or group based. The primary endpoint was the feasibility, defined by recruitment rate, attendance, adherence, dropout rate, tolerability (comparing the session RPE with the target RPE), and safety (using the Common Terminology Criteria for Adverse Events, version 5.0). Secondary endpoints included cardiorespiratory fitness (six minutes walking test), muscle strength (handgrip strength test and isometric leg press test), flexibility (the back scratch and chair sit and reach tests), anthropometric parameters (body mass index and waist-hip ratio), quality of life (EORTC QLQ C-30 questionnaire), and amount of physical exercise (Godin's Shepard Leisure Time Exercise Questionnaire). Descriptive statistics, Student t test, and Wilcoxon signed rank test were used to analyze data. RESULTS: The study recruitment rate was 81%. Out of 44 recruited patients, 28 chose the personal training program, 16 chose the home-based program, and none chose the group-based program. Nine dropouts occurred (20%), 6 in the personal training program, and 3 in the home-based intervention. The median attendance rate was 92%, adherence was 88%, tolerability was 100%, and 9 nonsevere adverse events were registered during the exercise sessions. An increase in cardiorespiratory fitness (P < .001) and flexibility (P = .011 for chair sit and reach; P = .040 for back scratch) was observed at the end of the intervention, while no changes in anthropometric values and muscle strength were detected. Different quality-of-life domains were improved following the intervention, including physical (P = .002), emotional (P < .001), and role functioning (P = .018), fatigue (P = .030), and appetite loss (P = .005). CONCLUSION: A 3-month exercise program based on a patient-preferred delivery mode is feasible in patients with metastatic cancer and may improve physical function and quality of life. TRIAL REGISTRATION: NCT04226508.

A randomized controlled trial of shared decision-making treatment planning process to enhance shared decision-making in patients with MBC.

PURPOSE: Opportunities exist for patients with metastatic breast cancer (MBC) to engage in shared decision-making (SDM). Presenting patient-reported data, including patient treatment preferences, to oncologists before or during a treatment plan decision may improve patient engagement in treatment decisions. METHODS: This randomized controlled trial evaluated the standard-of-care treatment planning process vs. a novel treatment planning process focused on SDM, which included oncologist review of patient-reported treatment preferences, prior to or during treatment decisions among women with MBC. The primary outcome was patient perception of shared decision-making. Secondary outcomes included patient activation, treatment satisfaction, physician perception of treatment decision-making, and use of treatment plans. RESULTS: Among the 109 evaluable patients from December 2018 to June 2022, 28% were Black and 12% lived in a highly disadvantaged neighborhood. Although not reaching statistical significance, patients in the intervention arm perceived SDM more often than patients in the control arm (63% vs. 59%; Cramer's V = 0.05; OR 1.19; 95% CI 0.55-2.57). Among patients in the intervention arm, 31% were at the highest level of patient activation compared to 19% of those in the control arm (V = 0.18). In 82% of decisions, the oncologist agreed that the patient-reported data helped them engage in SDM. In 45% of decision, they reported changing management due to patient-reported data. CONCLUSIONS: Oncologist engagement in the treatment planning process, with oncologist review of patient-reported data, is a promising approach to improve patient participation in treatment decisions which should be tested in larger studies. TRIAL REGISTRATION: NCT03806738.

Interventions aiming to improve advance care planning uptake in oncology: a scoping review of recent randomized controlled trials.

PURPOSE OF REVIEW: The evidence regarding the effectiveness of interventions aiming to improve ACP uptake appears mixed. The purpose of this scoping review is to summarize findings from recent RCTs assessing the efficacy of interventions designed to increase ACP uptake in cancer care. RECENT FINDINGS: The PubMed database was searched for studies published between 2021 and 2023. Ten RCTs were identified that evaluated a wide range of interventions aimed at encouraging or facilitating ACP communication among patients with advanced cancer, caregivers, and HCPs. Three RCTs developed ACP facilitation tools for patients and sometimes caregivers. Six RCTs involved integrating trained ACP facilitators into patient care, and one RCT used nudges to encourage oncologists trained in ACP discussions to initiate discussions with patients at highest risk of short-term mortality. Most of the reviewed interventions were based on previously tested intervention approaches or tools. SUMMARY: The key effects identified in this review are the facilitation of ACP discussions among patients, caregivers, and HCPs, and the documentation of patient preferences in the medical record. The direct involvement of facilitators in ACP document drafting and inclusion in patient files, as well as interventions prompting end-of-life discussions, contributed to this effect.

Patients' Preferences Towards Decision Counseling for Active Surveillance After Neoadjuvant Chemoradiotherapy for Esophageal Cancer.

BACKGROUND: Decision counseling (DC) is offered to enable patients to reflect on their treatment preferences and to think through the consequences of alternative treatment options. However, the timing of DC is debatable. In this study, patients who underwent DC at different times were interviewed about their experiences, specifically focusing on the timing of DC. METHODS: Patients with locally advanced esophageal cancer eligible for participation in a prospective cohort study on active surveillance (SANO-2 study) were offered DC either before or after neoadjuvant chemoradiotherapy (nCRT). Structured interviews were conducted by phone 1 week after DC, and responses were analyzed using frequency counts for the answers to set response categories. The primary outcome was the preferred time to receive DC, while the secondary outcome was the overall experience of patients with DC. RESULTS: Overall, 40 patients were offered DC between 2021 and 2023. Patients who had counseling before the start of nCRT (n = 20) were satisfied with the timing of DC. Of the 20 patients who had DC after nCRT, 6 would have preferred counseling at an earlier time point. Patients who had DC both before or after the completion of nCRT reflected positively on DC. CONCLUSION: It is recommended to introduce the option of DC as early as possible and discuss with the patient at which moment during the decision-making process they prefer to discuss all treatment options more extensively.

Patient Preference for Surgical Methods for Ipsilateral Breast Tumor Recurrence.

BACKGROUND: Mastectomy has been the standard surgical treatment for ipsilateral breast tumor recurrence (IBTR). Recently, there has been growing interest in repeat breast-conserving surgery (rBCS) for IBTR among breast surgeons; however, there is currently little information regarding patient preferences for surgical procedure for IBTR. The purpose of this study was to evaluate preference for surgical procedure (mastectomy vs. rBCS) among breast cancer patients who had undergone salvage surgery for IBTR. METHODS: Overall, 100 breast cancer patients who had undergone salvage surgery for IBTR were asked about their preferred surgical methods for IBTR and the reason. The association of patient preference and the reasons related to various clinical and pathological factors were assessed. RESULTS: Of the 100 respondents, only 11 patients (11%) preferred rBCS. Patients who had undergone rBCS and radiotherapy for IBTR were significantly more likely to prefer to undergo rBCS than other groups (p = 0.030). The most frequent reason for choosing rBCS was the patient's desire to minimize breast deformity and surgical wounds. CONCLUSIONS: Our study revealed that there is a low rate of patients who opt to undergo rBCS among patients who had undergone salvage surgery for IBTR. Discrepancies in perceptions regarding the surgical procedure for IBTR between patients and their surgeons may exist.

Patient decision-making in the era of transcarotid artery revascularization.

OBJECTIVE: The National Coverage Determination on carotid stenting by Medicare in October 2023 stipulates that patients participate in a shared decision-making (SDM) conversation with their proceduralist before an intervention. However, to date, there is no validated SDM tool that incorporates transcarotid artery revascularization (TCAR) into its decision platform. Our objective was to elicit patient and surgeon experiences and preferences through a qualitative approach to better inform the SDM process surrounding carotid revascularization. METHODS: We performed longitudinal perioperative semistructured interviews of 20 participants using purposive maximum variation sampling, a qualitative technique designed for identification and selection of information-rich cases, to define domains important to participants undergoing carotid endarterectomy or TCAR and impressions of SDM. We also performed interviews with nine vascular surgeons to elicit their input on the SDM process surrounding carotid revascularization. Interview data were coded and analyzed using inductive content analysis coding. RESULTS: We identified three important domains that contribute to the participants' ultimate decision on which procedure to choose: their individual values, their understanding of the disease and each procedure, and how they prefer to make medical decisions. Participant values included themes such as success rates, "wanting to feel better," and the proceduralist's experience. Participants varied in their desired degree of understanding of carotid disease, but all individuals wished to discuss each option with their proceduralist. Participants' desired medical decision-making style varied on a spectrum from complete autonomy to wanting the proceduralist to make the decision for them. Participants who preferred carotid endarterectomy felt outcomes were superior to TCAR and often expressed a desire to eliminate the carotid plaque. Those selecting TCAR felt it was a newer, less invasive option with the shortest procedural and recovery times. Surgeons frequently noted patient factors such as age and anatomy, as well as the availability of long-term data, as reasons to preferentially select one procedure. For most participants, their surgeon was viewed as the most important source of information surrounding their disease and procedure. CONCLUSIONS: SDM surrounding carotid revascularization is nuanced and marked by variation in patient preferences surrounding autonomy when choosing treatment. Given the mandate by Medicare to participate in a SDM interaction before carotid stenting, this analysis offers critical insights that can help to guide an efficient and effective dialog between patients and providers to arrive at a shared decision surrounding therapeutic intervention for patients with carotid disease.

Determining patient preferences for the medical management of osteoporosis using conjoint analysis.

We used conjoint analysis-a method that assesses complex decision making-to quantify patients' choices when selecting an osteoporosis therapy. While 60% of people prioritized medication efficacy when deciding among treatments, the remaining 40% highly valued factors other than efficacy, suggesting the need for personalized shared decision-making tools. INTRODUCTION: In this study, we aimed to examine patient decision-making surrounding osteoporosis medications using conjoint analysis. METHODS: We enrolled osteoporosis patients at an academic medical center to complete an online conjoint exercise which calculated each patient's relative importance score of 6 osteoporosis medication attributes (higher = greater relative importance in decision-making). We used latent class analysis to identify distinct segments of patients with similar choice patterns and then used logistic regression to determine if demographics and osteoporosis disease features were associated with latent class assignment. RESULTS: Overall, 304 participants completed the survey. The rank order of medication attributes by importance score was the following: efficacy at preventing hip fractures (accounted for 31.0% of decision making), mode of administration (17.5%); risk of serious side effects (16.6%); dose frequency (13.9%); efficacy at preventing spine fractures (12.5%); risk of non-serious side effects (8.4%). We found that 60.9% of the cohort prioritized medication efficacy as their top factor when selecting among the therapies. Being a college graduate, having stronger beliefs on the necessity of using medications for osteoporosis, and never having used osteoporosis medicines were the only factors associated with prioritizing medication efficacy for fracture prevention over the other factors in the decision-making process. CONCLUSIONS: While about 60% of patients prioritized efficacy when selecting an osteoporosis therapy, the remaining 40% valued other factors more highly. Furthermore, individual patient characteristics and clinical factors did not reliably predict patient decision making, suggesting that development and implementation of shared decision-making tools is warranted.

Patients' preferences for fracture risk communication: the Risk Communication in Osteoporosis (RICO) study.

The RICO study indicated that most patients would like to receive information regarding their fracture risk but that only a small majority have actually received it. Patients globally preferred a visual presentation of fracture risk and were interested in an online tool showing the risk. PURPOSE: The aim of the Risk Communication in Osteoporosis (RICO) study was to assess patients' preferences regarding fracture risk communication. METHODS: To assess patients' preferences for fracture risk communication, structured interviews with women with osteoporosis or who were at risk for fracture were conducted in 11 sites around the world, namely in Argentina, Belgium, Canada at Hamilton and with participants from the Osteoporosis Canada Canadian Osteoporosis Patient Network (COPN), Japan, Mexico, Spain, the Netherlands, the UK, and the USA in California and Washington state. The interviews used to collect data were designed on the basis of a systematic review and a qualitative pilot study involving 26 participants at risk of fracture. RESULTS: A total of 332 women (mean age 67.5 ± 8.0 years, 48% with a history of fracture) were included in the study. Although the participants considered it important to receive information about their fracture risk (mean importance of 6.2 ± 1.4 on a 7-point Likert scale), only 56% (i.e. 185/332) had already received such information. Globally, participants preferred a visual presentation with a traffic-light type of coloured graph of their FRAX® fracture risk probability, compared to a verbal or written presentation. Almost all participants considered it important to discuss their fracture risk and the consequences of fractures with their healthcare professionals in addition to receiving information in a printed format or access to an online website showing their fracture risk. CONCLUSIONS: There is a significant communication gap between healthcare professionals and patients when discussing osteoporosis fracture risk. The RICO study provides insight into preferred approaches to rectify this communication gap.

Patients' values and preferences for health states in allergic rhinitis-An artificial intelligence supported systematic review.

BACKGROUND: Allergic rhinitis (AR) impacts patients' physical and emotional well-being. Assessing patients' values and preferences (V&P) related to AR is an essential part of patient-centered care and of the guideline development process. We aimed to systematically summarize the information about patients' V&P on AR and its symptoms and impact on daily life. METHODS: We conducted systematic review in a MEDLINE, Embase, PsychInfo, and CINAHL databases. We included studies which quantitatively assessed patients' V&P for specific outcomes in AR by assessing utilities, applying discrete choice approaches, or rating and ranking outcomes. We grouped outcomes as AR symptoms, functional status, and care-related patient experience. Study selection and data extraction were supported by the Laser AI tool. We rated the certainty of evidence (CoE) using the GRADE approach. RESULTS: Thirty-six studies (41 records) were included: nine utility studies, seven direct-choice studies and 21 studies of rating or ranking outcomes. Utilities were lower with increased AR severity and with the concomitant presence of asthma, but not with whether AR was seasonal or perennial (CoE = low-high). Patients rated AR symptom-related outcomes as more important than those related to care-related patient experience and functional status (CoE = very low-moderate). Nasal symptoms (mainly nasal congestion) followed by breathing disorders, general and ocular symptoms were rated as the symptoms with the highest impact. CONCLUSIONS: This systematic review provides a comprehensive overview of V&P of patients with AR. Patients generally considered nasal symptoms as the most important. Future studies with standardized methods are needed to provide more information on V&P in AR.

Factors Informing High-Risk Primary Care Patient Choice around Telehealth Use: a Framework.

BACKGROUND: While telehealth's presence in post-pandemic primary care appears assured, its exact role remains unknown. Value-based care's expansion has heightened interest in telehealth's potential to improve uptake of preventive and chronic disease care, especially among high-risk primary care populations. Despite this, the pandemic underscored patients' diverse preferences around using telehealth. Understanding the factors underlying this population's preferences can inform future telehealth strategies. OBJECTIVE: To describe the factors informing high-risk primary care patient choice of whether to pursue primary care via telehealth, in-office or to defer care altogether. DESIGN: Qualitative, cross-sectional study utilizing semi-structured telephone interviews of a convenience sample of 29 primary care patients between July 13 and September 30, 2020. PARTICIPANTS: Primary care patients at high risk of poor health outcomes and/or acute care utilization who were offered a follow-up primary care visit via audiovisual, audio-only or in-office modalities. APPROACH: Responses were analyzed via grounded theory, using a constant comparison method to refine emerging categories, distinguish codes, and synthesize evolving themes. KEY RESULTS: Of the 29 participants, 16 (55.2%) were female and 19 (65.5%) were Black; the mean age (SD) was 64.6 (11.1). Participants identified four themes influencing their choice of visit type: perceived utility (encapsulating clinical and non-clinical utility), underlying costs (in terms of time, money, effort, and safety), modifiers (e.g., participants' clinical situation, choice availability, decision phenotype), and drivers (inclusive of their background experiences and digital environment). The relationship of these themes is depicted in a novel framework of patient choice around telehealth use. CONCLUSIONS: While visit utility and cost considerations are foundational to participants' decisions around whether to pursue care via telehealth, underappreciated modifiers and drivers often magnify or mitigate these considerations. Policymakers, payers, and health systems can leverage these factors to anticipate and enhance equitable high-value telehealth use in primary care settings among high-risk individuals.

Examining Patient Preferences for Express, Telemedicine, and Standard Visits in a Sexual Health Clinic in New York City.

ABSTRACT: This study, completed at an sexually transmitted infection (STI) clinic in 2019 to 2020, evaluated patient preferences for telemedicine, express, and standard visits. Active PrEP users preferred telemedicine and express visits, patients with prior STIs preferred express visits, and cisgender women preferred standard visits. Configuring STI clinic visit types requires shared decision making and individualization.

Understanding the experience, treatment preferences and goals of people living with chronic lymphocytic leukemia (CLL) in Australia.

BACKGROUND: Listening to patient voices is critical, in terms of how people experience their condition as well as their treatment preferences. This research explored the patient journey, therapy attributes and goals among treatment experienced adults with chronic lymphocytic leukemia (CLL). We sought to understand patient experiences, needs and expectations to identify areas for improvement of treatment and care delivery. METHODS: Two online surveys were developed for completion by CLL patients. In Stage 1, participants completed a best-worst scaling (BWS) task to evaluate eleven previously validated healthcare journey moments that matter (MTM). Responses were used to generate the patient experience index (PEI) score. In Stage 2, participants completed a survey that included both a discrete choice experiment (DCE) to assess drivers of treatment preferences by evaluating the relative attribute importance (RAI) of seven features and a BWS exercise which explored long-term treatment goals. RESULTS: Twenty-five patients completed Stage 1 and thirty patients Stage 2. Treatment experience was balanced between oral and intravenous medication. The most important/least satisfied MTM were treatment effectiveness, access to support and other treatments as well as monitoring progress. The median PEI score was 66.2 (out of 100). DCE results demonstrated that patients most value treatments for CLL that are associated with prolonged progression free survival (PFS; RAI: 24.6%), followed by treatments that have a lower risk of severe side effects and lower out-of-pocket costs (RAI: 19.5%, 17.4%, respectively). The remainder of the weight in decision making (38.5%) was split between the remaining attributes, namely 'mild to moderate side effects' (13.4%), 'long-term risks' (12.2%), type of treatment (i.e., oral, IV or a combination of oral and IV; 8.7%) and treatment duration (i.e., ongoing versus fixed; 4.2%). Patients preferred oral to intravenous therapy. The most valued long-term treatment goal was to be physically healthy, followed by living a long life, spending time with family/friends, and avoiding hospitalization. CONCLUSION: Treatment experienced patients with CLL are focused on receiving effective, safe therapies and value long PFS. Consideration and discussion of other attributes, such as once daily dosing, oral only medication, out-of-pocket costs and access to support services may affect patient treatment choices and ultimately enhance their healthcare experience and outcomes.

Patients' preferences for chronic lymphocytic leukemia treatment: The CHOICE study.

Chronic lymphocytic leukemia (CLL) therapies differ in efficacy, side effects, route, frequency, and duration of administration. We assessed patient preferences for treatment attributes and evaluated associations with disease stage, treatment line, and socio-demographic characteristics in a cross sectional, observational study conducted at 16 Italian hematology centers. Study visits occurred between February and July 2020; 401 adult patients with CLL (201 Watch and Wait (W&W), 200 treated) participated in a discrete choice experiment (DCE), composed of 8 choices between pairs of treatment profiles with different levels of 5 attributes of currently available CLL treatments (length of response, route and duration of administration, risk of side effects including diarrhea, infections, or organ damage). Health-related quality of life was assessed with the EQ-5D-5L, EORTC QLQ-C30 and QLQ CLL-16. Previously treated patients had longer disease duration (7 vs. 5 years), higher prevalence of serious comorbidities (45.5% vs. 36.2%) and high-risk molecular markers (unmutated IGHV 55.6% vs. 17.1%; TP53 mutation 15.2% vs. 4.0%). Health-related quality of life scores were similar between groups. In the DCE, W&W patients rated "possible occurrence of infections" highest (relative importance [RI] = 36.2%), followed by "treatment and relevant duration" (RI = 28.0%) and "progression-free survival (PFS)" (RI = 16.9%). Previously treated patients rated "treatment and relevant duration" highest (RI = 33.3%), followed by "possible occurrence of infections" (RI = 28.8%), "possible occurrence of organ damage" (RI = 19.4%), and "PFS" (RI = 9.8%). Concern over infection was rated highest overall; unexpectedly PFS was not among the most important criteria in either group, suggesting that the first COVID-19 pandemic wave may have influenced patient preferences and concerns about CLL therapy options.

Patient and caregiver preferences for haemophilia treatments: A discrete-choice experiment.

INTRODUCTION: An evolving haemophilia treatment landscape provides new possibilities for previously unattainable lifestyles. AIM: We sought to understand how people with haemophilia (PwH) and their caregivers value the potential benefits of novel prophylactic treatments. We conducted a discrete-choice experiment (DCE) to quantify preferences for features of haemophilia treatments among adults and caregivers of children with haemophilia. A best-worst scaling (BWS) exercise measured the perceived burden of treatment administration features. METHODS: A cross-sectional, web-based survey was administered to male adults (≥18 years) and caregivers of male children (≤17 years) living with haemophilia in the United States. Respondents evaluated eight pairs of hypothetical haemophilia treatment profiles defined by six attributes in the DCE and 15 features in the BWS. RESULTS: In the DCE, both adults with haemophilia (n = 151) and caregivers (n = 151) prioritised avoiding the risk of developing inhibitor/ anti-drug antibodies and treatments that allowed for a more active life. They placed a lower priority on reducing the number of spontaneous bleeding episodes, route and frequency of administration, and avoiding the risk of hospitalisation due to adverse events. The BWS documented the burdensomeness of IV infusions and medications that require mixing and refrigeration. CONCLUSION: PwH and caregivers prefer treatments that enable a more active lifestyle with a lower risk of inhibitor development. Both groups valued the ability to lead an active life over reducing spontaneous bleeding, with caregivers placing the most weight on this attribute. As new treatments expand possibilities, healthcare professionals and PwH should continue to share decision-making, incorporating clinical judgment and individual preferences.

Preference of treatment characteristics among people with haemophilia or their caregivers, and physicians in the Japanese healthcare environment.

INTRODUCTION: Studies of treatment preferences in haemophilia have been conducted in many countries. This study is the first to examine treatment characteristic preferences among people with haemophilia (PWH) and their caregivers, and physicians in Japan. AIM: To examine current treatment preferences of PWH and their caregivers, plus those of physicians at haemophilia treatment centres (HTCs) and non-HTCs for different treatment characteristics in Japan. METHODS: Physicians listed on a survey panel were invited to participate in the survey and to refer PWH and caregivers to participate in the survey. Web-based surveys were conducted to examine physician and PWH/caregiver background, prophylaxis background, prophylaxis goals, understanding of haemophilia treatment products, important information sources, preferences while choosing prophylaxis products, understanding of the patient's condition, and potential product switching. A discrete choice experiment exercise was included in the survey. RESULTS: A total of 107 physicians and 44 PWH/caregivers participated in the study. Key treatment goals of physicians included optimisation of haemophilia management. PWH/caregivers were focused on quality of life and reduced treatment burden. Consistent differences in haemophilia treatment strategies at HTCs and non-HTCs were observed for prescribed treatments, preferences in choosing prophylaxis products, understanding of patients' condition, and reasons for potential product switch. CONCLUSION: Our study utilises real-world survey data and presents preferences for haemophilia treatment characteristics among physicians, PWH and their caregivers in Japan, which could encourage improvements in individualised treatment and disease management. Alignment between treatment approaches at HTCs and non-HTCs could facilitate improvements in the quality of care for PWH across Japan.

The EASEMENT study: A multicentre, observational, cross-sectional study to evaluate patient preferences, treatment satisfaction, quality of life, and healthcare resource use in patients with multiple myeloma receiving injectable-containing or fully oral therapies.

OBJECTIVE: As multiple myeloma (MM) therapies advance, understanding patients', caregivers', and physicians' perspectives on, and satisfaction with, available treatment options and their impact on quality of life (QoL), is important. METHODS: EASEMENT is a real-world, observational, cross-sectional study conducted in 19 sites within the UK, Canada, and Italy using retrospective chart reviews and surveys. Enrolled patients had clinical history available since diagnosis and had received ≥1 cycle of their current line of therapy. Primary objectives were to describe patient/caregiver QoL (EQ-5D-5L questionnaire), patient preference for oral/injectable therapies (single discrete-choice question), and patient satisfaction (TSQM-9 questionnaire). RESULTS: Between October 2018 and March 2020, 399 patients were enrolled (n = 192 newly diagnosed multiple myeloma [NDMM], n = 206 relapsed/refractory multiple myeloma [RRMM], n = 1 missing). Among NDMM and RRMM patients, 78%/22% and 42%/58% were receiving injectables/orals, respectively. Both NDMM and RRMM patients significantly preferred orals versus injectables (p < .0001). No significant differences were reported in treatment satisfaction or QoL, but treatment convenience favoured orals over injectables with near significance (p = .053). CONCLUSION: MM patients perceived greater convenience and preference for orals versus injectables. Oral treatments are useful for patients who cannot or prefer not to travel to clinics, or cannot perform self-injection within the community.