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INTRODUCTION/BACKGROUND: This study aimed to describe patient and caregiver preferences for treatments of relapsed or refractory multiple myeloma (MM). MATERIALS AND METHODS: A survey including discrete-choice experiment (DCE) and best-worst scaling (BWS) exercises was conducted among US patients with relapsed or refractory MM and their caregivers. The DCE included six attributes with varying levels including progression-free survival (PFS), toxicity, and mode and frequency of administration. In addition, the impact of treatment cost was assessed using a fixed-choice question. The BWS exercise included 18 items (modes and frequency of administration, additional treatment convenience, and toxicity items). The survey was administered online to patients recruited from the Multiple Myeloma Research Foundation CoMMpass study (NCT01454297). RESULTS: The final samples consisted of 94 patients and 32 caregivers. Avoiding severe nerve damage was most important to patients, followed by longer PFS. Caregivers considered PFS to be the most important attribute. We estimate that a third or more of patients were cost-sensitive, meaning their treatment preference was altered based on cost implications. Caregivers were not cost-sensitive. The three most bothersome treatment features in the BWS exercise were risk of kidney failure, lowering white blood cell counts, and weakening the immune system. CONCLUSION: Patients with relapsed or refractory MM and their caregivers consider many factors including efficacy, toxicity, mode/frequency of administration, and cost in their decisions regarding treatment options. The study provides a basis for future Research on patient and caregiver treatment preferences, which could be incorporated into shared decision-making with physicians.

Background: Genetic testing can offer early diagnosis and subsequent treatment of rare neuromuscular diseases. Options for these tests could be improved by understanding the preferences of patients for the features of different genetic tests, especially features that increase information available to patients. Methods: We developed an online discrete-choice experiment using key attributes of currently available tests for Pompe disease with six test attributes: number of rare muscle diseases tested for with corresponding probability of diagnosis, treatment availability, time from testing to results, inclusion of secondary findings, necessity of a muscle biopsy, and average time until final diagnosis if the first test is negative. Respondents were presented a choice between two tests with different costs, with respondents randomly assigned to one of two costs. Data were analyzed using random-parameters logit. Results: A total of 600 online respondents, aged 18 to 50 years, were recruited from the U.S. general population and included in the final analysis. Tests that targeted more diseases, required less time from testing to results, included information about unrelated health risks, and were linked to shorter time to the final diagnosis were preferred and associated with diseases with available treatment. Men placed relatively more importance than women on tests for diseases with available treatments. Most of the respondents would be more willing to get a genetic test that might return unrelated health information, with women exhibiting a statistically significant preference. While respondents were sensitive to cost, 30% of the sample assigned to the highest cost was willing to pay $500 for a test that could offer a diagnosis almost 2 years earlier. Conclusion: The results highlight the value people place on the information genetic tests can provide about their health, including faster diagnosis of rare, unexplained muscle weakness, but also the value of tests for multiple diseases, diseases without treatments, and incidental findings. An earlier time to diagnosis can provide faster access to treatment and an end to the diagnostic journey, which patients highly prefer. [ABSTRACT FROM AUTHOR]

Atopic dermatitis (AD) is a chronic, inflammatory disease characterized by sensitive and dry skin, eczematous lesions and intense pruritus. Patients with moderate-to-severe AD often require systemic treatments if their symptoms are insufficiently controlled with topical treatments or phototherapy. While numerous systemic therapies are currently available for moderate-to-severe AD, more data evaluating patient satisfaction with various characteristics of these treatments are needed to help guide physicians and their patients with joint treatment decision-making. This study aims to investigate patient satisfaction with AD treatments according to the degree and speed of itch improvement and the degree of skin clearance. A cross-sectional, web-based survey was used to evaluate patient preferences for characteristics associated with systemic AD therapies. Respondents were required to have a physician-confirmed diagnosis of AD (currently moderate or severe), to be aged ≥18 years, to be a US resident, and to read and understand English. Assessments included worst itch experienced in the past 24 h (using a scale from 0 to 10, with 0 defined as no itch and 10 defined as the worst imaginable itch), time to noticeable itch reduction following initiation of their current treatment (1–6 days, 7–13 days or ≥14 days) and the amount of body surface area still affected by AD (≤2%, 3–10% or >10%; patient-assessed). Patient satisfaction with itch improvement, speed of itch improvement and skin clearance with their current treatment was also assessed and classified into three categories: satisfied, dissatisfied or neither. P values were determined by Chi-square tests (or Fisher's exact test where appropriate). Of the 213 individuals recruited by physicians to participate in the survey, 200 respondents fulfilled the eligibility criteria, consented to participate and completed surveys. A total of 186 respondents indicated that they were currently receiving treatment for their AD; treatments included over-the-counter creams, ointments or medicines (26.9%, n = 50), prescription creams (72.0%, n = 134), oral corticosteroids (16.7%, n = 31) or dupilumab (11.3%, n = 21). Treatment satisfaction differed based on the patient's itch level within the past 24 h (P < 0.001): 85.2% (n = 23) of the 27 patients reporting the lowest levels of itch (0–1) indicated they were satisfied with their current treatment, whereas only 7.1% (n = 1) of the 14 patients reporting the worst levels of itch (8–10) were satisfied with their current treatment. Among the 73 respondents who experienced the fastest itch reduction (within 1–6 days), 65.8% (n = 48) were satisfied with their treatment. For the 36 respondents experiencing itch reduction ≥14 days post treatment, 13.9% (n = 5) were satisfied with their treatment (P < 0.001). Of the 75 respondents with the highest degree of skin clearance (≤2% of body surface area still affected), 65.3% (n = 49) were satisfied with their treatment. Among the 21 respondents with the lowest degree of skin clearance (>10% of body surface area still affected), 14.3% (n = 3) were satisfied with their treatment (P < 0.001). Safety was not evaluated, which may limit the findings in this analysis. In patients with moderate-to-severe AD, the majority of respondents experiencing the lowest levels of itch, the fastest onset of itch improvement and the highest degree of skin clearance also reported satisfaction with their current treatment. These findings underscore the stringent thresholds for both the degree and speed of symptom improvement required to achieve patient satisfaction. This highlights patients' desire for treatments offering both rapid and extensive itch reduction and skin clearance and may help broaden physicians' understanding of patient preferences and inform treatment decisions. [ABSTRACT FROM AUTHOR]

Aim: Patient preferences for the features of targeted chronic lymphocytic leukemia (CLL) therapies may differ. Materials & methods: A discrete-choice experiment (DCE) survey was administered to 229 respondents recruited through the CLL Society. Results: Respondents placed most importance on increasing the chance of progression-free survival (PFS) at 2 years from 70 to 90% and confirming results with measurable residual disease (MRD) testing instead of routine testing. Respondents also preferred daily oral administration over intravenous infusion every 4 weeks, fixed-duration treatments over treat-to-progression treatments and treatments with lower side effect risks. Reducing risk of tumor lysis syndrome was least important relative to changes in other attributes. Conclusion: The combination of improving PFS combined with confirming results using MRD testing was more important than changes in all other study attributes included in the DCE. Results from this study can help inform shared decision-making when selecting therapies for CLL.

Background: Patients with moderate-to-severe atopic dermatitis (AD) experience skin lesions and intense itch that substantially affect quality of life. Patients have choices among systemic AD treatments that offer varied benefit-risk profiles., Objective: Measure patients' willingness to trade off the risks and benefits of systemic treatments among individuals with a physician-confirmed diagnosis of moderate-to-severe AD., Methods: Patients participated in a discrete choice experiment online survey with a series of choices between hypothetical AD treatments defined by six attributes reflecting benefits and risks of treatments (itch reduction, time until noticeable itch reduction, chance of clear or almost clear skin, risk of serious infection, risk of developing acne, and need for prescription topical steroids). Data were analyzed with a random parameters logit model to quantify preferences and the relative importance of attributes for treatment alternatives., Results: Respondents ( n  = 200) placed the highest relative importance on itch reduction, speed of itch reduction, and skin clearance, and were generally willing to accept clinically relevant levels of risk of serious infection and acne in exchange for treatment benefits., Conclusions: Patients with moderate-to-severe AD were willing to trade clinically relevant treatment risks for greater or more rapid itch reduction and skin clearance offered by systemic therapies.

Objectives: Low optimism and low numeracy are associated with difficulty or lack of participation in making treatment-related health care decisions. We investigated whether low optimism and low self-reported numeracy scores could help uncover evidence of decisional conflict in a discrete-choice experiment (DCE)., Methods: Preferences for a treatment to delay type 1 diabetes were elicited using a DCE among 1501 parents in the United States. Respondents chose between two hypothetical treatments or they could choose no treatment (opt out) in a series of choice questions. The survey included a measure of optimism and a measure of subjective numeracy. We used latent class analyses where membership probability was predicted by optimism and numeracy scores., Results: Respondents with lower optimism scores had a higher probability of membership in a class with disordered preferences ( P value for optimism coefficient = 0.032). Those with lower self-reported numeracy scores were more likely to be in a class with a strong preference for opting out and disordered preferences ( P = 0.000) or a class with a preference for opting out and avoiding serious treatment-related risks ( P = 0.015)., Conclusions: If respondents with lower optimism and numeracy scores are more likely to choose to opt out or have disordered preferences in a DCE, it may indicate that they have difficulty completing choice tasks., Competing Interests: The author(s) declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article: JS and BH were salaried employees of RTI Health Solutions when the study was conducted. CL and CM are salaried employees of RTI Health Solutions. RLD is a salaried employee of Jansen Research & Development, LLC., (© The Author(s) 2021.)