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Background Hypertrophic cardiomyopathy (HCM) is a primary myocardial disorder defined by left ventricular hypertrophy that cannot be explained by another cardiac or systemic disease. There is a general lack of knowledge about patients’ perspectives on the symptoms and day-to-day limitations they experience as a result of HCM. We therefore sought an in-depth understanding of patients’ experiences of obstructive (oHCM) and nonobstructive (nHCM) forms of the disease, including symptoms and their quality of life impacts, and to develop a conceptual model to capture them. Methods Development of the HCM conceptual model involved a web-based survey to capture patients’ insights, a targeted literature review (which included relevant guidelines and patient advocacy websites), one-to-one interviews with clinical experts, and one-to-one qualitative concept elicitation interviews with patients. Key symptoms and their impacts most important to patients’ experiences were identified and used to develop a conceptual model of the patient experience with HCM. Results The HCM symptoms reported by patient interviewees (n = 27) were largely consistent with findings from the patient web survey (n = 444), literature review, and interviews with three expert clinicians. The symptoms most commonly reported in patient interviews included tiredness (89%), shortness of breath (89%), shortness of breath with physical activity (89%), and dizziness/light-headedness (89%). Other symptoms commonly reported included chest pain (angina) (70%), chest pain (angina) with physical exertion (70%), and palpitations (fluttering or rapid heartbeat) (81%). The most commonly reported impacts of HCM symptoms on patients’ lives included limitations to physical activities (78%), emotional impacts, including feeling anxious or depressed (78%), and impacts on work (63%). Symptoms and impacts were similar for both oHCM and nHCM. Conclusions A conceptual model was developed, which identifies the core symptoms that patients with oHCM and nHCM reported as most frequent and most important: shortness of breath, palpitations, fatigue/tiredness, dizziness/light-headedness, and chest pain, as well as the impacts those symptoms have on patients’ lives. This HCM conceptual model reflecting patients’ experiences and perspectives was used in the development of a patient-reported outcomes instrument for use in clinical trials and it may also help inform the clinical management of HCM.

Objectives: To provide information on systemic lupus erythematosus (SLE) patients' experiences, satisfaction, and expectations with treatments and examine the association between treatment satisfaction and patient-reported outcomes (PRO). Methods: A cross-sectional, non-interventional, online survey of US adult patients with SLE was conducted in 2019. The survey consisted of 104 questions about SLE and the following PRO instruments:

Background As the number and type of cancer treatments available rises and patients live with the consequences of their disease and treatments for longer, understanding preferences for cancer care can help inform decisions about optimal treatment development, access, and care provision. Discrete choice experiments (DCEs) are commonly used as a tool to elicit stakeholder preferences; however, their implementation in oncology may be challenging if burdensome trade-offs (e.g. length of life versus quality of life) are involved and/or target populations are small. Objectives The aim of this review was to characterise DCEs relating to cancer treatments that were conducted between 1990 and March 2020. Data Sources EMBASE, MEDLINE, and the Cochrane Database of Systematic Reviews were searched for relevant studies. Study Eligibility Criteria Studies were included if they implemented a DCE and reported outcomes of interest (i.e. quantitative outputs on participants' preferences for cancer treatments), but were excluded if they were not focused on pharmacological, radiological or surgical treatments (e.g. cancer screening or counselling services), were non-English, or were a secondary analysis of an included study. Analysis Methods Analysis followed a narrative synthesis, and quantitative data were summarised using descriptive statistics, including rankings of attribute importance. Result Seventy-nine studies were included in the review. The number of published DCEs relating to oncology grew over the review period. Studies were conducted in a range of indications (n = 19), most commonly breast (n =10, 13%) and prostate (n = 9, 11%) cancer, and most studies elicited preferences of patients (n = 59, 75%). Across reviewed studies, survival attributes were commonly ranked as most important, with overall survival (OS) and progression-free survival (PFS) ranked most important in 58% and 28% of models, respectively. Preferences varied between stakeholder groups, with patients and clinicians placing greater importance on survival outcomes, and general population samples valuing health-related quality of life (HRQoL). Despite the emphasis of guidelines on the importance of using qualitative research to inform attribute selection and DCE designs, reporting on instrument development was mixed. Limitations No formal assessment of bias was conducted, with the scope of the paper instead providing a descriptive characterisation. The review only included DCEs relating to cancer treatments, and no insight is provided into other health technologies such as cancer screening. Only DCEs were included. Conclusions and Implications Although there was variation in attribute importance between responder types, survival attributes were consistently ranked as important by both patients and clinicians. Observed challenges included the risk of attribute dominance for survival outcomes, limited sample sizes in some indications, and a lack of reporting about instrument development processes. Protocol Registration PROSPERO 2020 CRD42020184232.

Background: Aortic stenosis (AS) treatments include surgical aortic valve replacement (SAVR) and transcatheter aortic valve replacement (TAVR). Choosing between SAVR and TAVR requires patients to trade-off  benefits and risks. The objective of this research was to determine which  TAVR and SAVR outcomes patients consider important, collect quantitative data about how patients weigh benefits and risks, and evaluate patients’ preferences for SAVR or TAVR. Methods: Patients  were recruited from advocacy organization databases. Patients self-reported as being diagnosed with AS, and as either having received AS treatment or as experiencing AS-related physical activity limitations. An online adapted swing weighting (ASW) method – a pairwise comparison of attributes – was used to elicit attribute trade-offs from 219 patients. Survey data were used to estimate patients’ weights for AS treatment attributes, which were incorporated into a quantitative benefit-risk analysis (BRA) to evaluate patients’ preferences for TAVR and SAVR. Results: On average, patients put greater value on attributes that favored TAVR than SAVR. Patients’ valuation of the lower mortality rate, reduced procedural invasiveness, and quicker time to return to normal quality of life associated with TAVR, offset their valuation of the time over which SAVR has been proven to work. There was substantial heterogeneity in patients’ preferences. This was partly explained by age, with differences in preference observed between patients <60 years to those ≥60 years. A Monte Carlo Simulation found that 79.5% of patients prefer TAVR. Conclusions: Most AS patients are willing to tolerate sizable increases in clinical risk in exchange for the benefits of TAVR, resulting in a large proportion of patients preferring TAVR to SAVR. Further work should be undertaken to characterize the heterogeneity in preferences for AS treatment attributes. Shared decision-making tools based on attributes important to patients can support patients’ selection of the procedure that best meets their needs.