Patient-Patient Centered Outcomes Research最新文献

Background: China faces the world's largest diabetes burden, with a 12.4% prevalence. A wide range of second-line therapies is available for patients with type 2 diabetes mellitus (T2DM), each with distinct characteristics. Although current guidelines advocate for a patient-centered approach in selecting second-line treatments, empirical evidence on Chinese patient's preferences for these therapies remains limited. We therefore elicited population‑level preferences using a Best-Worst Scaling profile‑case (BWS‑2) survey to inform decision making.

Method: A seven-attribute instrument was designed through literature review, expert input, and a BWS object-case study used for attribute refinement (reported elsewhere). The final study employed BWS‑2 to elicit preferences across benefit, risk, and cost attributes. An orthogonal main‑effects design generated the choice sets. The survey was conducted through in-person interviews with patients with T2DM at healthcare institutions in three Chinese regions (eastern, central, and western). Data were analyzed using counting approach, conditional, and mixed logit regression. Preference heterogeneity was explored with subgroup analyses and latent class analysis (LCA).

Results: Among 1517 respondents, counting approach and logit estimates were consistent; most attribute coefficients were statistically significant in logit analysis, with patients showing the highest preference for reduction in HbA1c, cardiovascular protection, and zero out-of-pocket costs compared with the reference levels. Weight change and mode of administration exerted modest influence. LCA identified six unobserved subgroups. Preferences heterogeneity were also explored by splitting the sample according to patient urban-rural residence and income characteristics.

Conclusions: Chinese patients with T2DM prioritized HbA1c reduction, cardiovascular benefits, and cost, while weight change was rated as the least important attribute. LCA further revealed preference heterogeneity, including limited concern for adverse events and neutral attitudes toward all attributes.

Introduction: Poly(ADP-ribose) polymerase inhibitors (PARPi) have survival benefits for patients with high-risk (High-risk disease is defined per the phase III OlympiA trial as follows: for triple-negative breast cancer, residual disease after neoadjuvant chemotherapy or node-positive or ≥ 2 cm tumors after adjuvant chemotherapy; for hormone receptor-positive disease, four or more positive nodes after adjuvant chemotherapy or a CPS + EG score ≥ 3 after incomplete response to neoadjuvant chemotherapy. The CPS + EG score accounts for clinical/pathologic stage, ER status, and grade (Giaquinto et al. in CA Cancer J Clin 72:524-541, 2022)), human epidermal growth factor receptor 2 (HER2)-negative early breast cancer (eBC) with germline BReast CAncer gene mutations (gBRCAm). However, many patients are unaware of their gBRCA status; this can impact eligibility for targeted treatment. We sought to evaluate patient preferences for BRCA testing and treatment decision-making as they relate to HER2-negative eBC.

Methods: We conducted an online survey, including a best-worst scaling exercise (BWS) and discrete-choice experiment (DCE), among patients with self-reported HER2-negative eBC residing in the USA who were either untested, unsure if they were tested, or tested positive for the gBRCAm. The BWS generated a rank ordering of 16 barriers and facilitators to BRCA testing. The DCE evaluated patient preferences for adjuvant therapies versus no treatment based on seven treatment attributes: invasive disease-free survival, targeted treatment, nausea risk, risk of serious side effects, regimen, treatment duration, and cost. BWS and DCE exercises were analyzed using hierarchical Bayesian models.

Results: Among the 359 women included in our sample, the top facilitators for BRCA testing were determining eligibility for targeted therapy that may prevent or delay metastasis, a physician's recommendation, and absence of out-of-pocket costs (OOPC). In contrast, the top barriers were an OOPC of $250, potential anxiety from test results, and the possibility of a 3- to 4-week delay in treatment. The DCE showed that most participants preferred adjuvant treatment (77.6%) over no treatment, and reducing treatment OOPC from $900 to $0, reducing the risk of serious side effects from 77 to 24%, and having a BRCA-targeted treatment influenced treatment choice most.

Conclusions: Individuals reported that a key benefit of BRCA testing was the insight it provided into their treatment options, allowing for more personalized care. However, OOPC was a barrier to testing. Their choice to receive adjuvant therapy was most influenced by OOPC, followed by the tolerability of the treatment and the ability to receive a targeted therapy.

Objectives: The EuroQol Valuation Technology (EQ-VT) uses composite time trade-off (cTTO) with a 10-year lead-time to value health states worse-than-dead (WTD) ranging between - 1 and 0 (dead). While WTD responses are common in EQ-5D-5L studies, their drivers remain understudied in Asia. This mixed-methods study explored socio-demographic factors and reasoning behind WTD preferences in Singapore.

Methods: We recruited 500 adult Singaporeans using quota sampling. Each participant completed 20 cTTO tasks through computer-assisted interviews, followed by open- and closed-ended questions. Two-part regression models assessed both the likelihood and the extent of WTD ratings, and qualitative content analysis evaluated participants' reasoning for their health state valuations.

Results: Of the 500 participants (mean age 48.1 years; 52.6% had tertiary education), 76.8% identified as Chinese. Overall, 33.0% assigned WTD values, and 16.1% assigned '- 1' values. Age, education, marital status, interviewer and caregiving experience were associated with WTD ratings, though none remained significant in the multivariable analysis. Similar factors were linked to '- 1' values, with middle age being the only factor that remained significant in the multivariable analysis. Qualitative data showed that middle-aged participants often cited worries about imposing physical, mental, or financial burdens on their families.

Conclusions: Preferences for immediate death over living in poor health are common in Singapore, particularly among middle-aged respondents. Concerns about burdening family members appear to drive these preferences, reflecting broader cultural values. These insights may clarify the high frequency of '-1' values in EQ-5D valuation studies across Asia.

Patient-reported outcome measures (PROMs) are widely implemented in Wales to achieve Value-Based Healthcare (VBHC). All patient-facing information in Wales must be available bilingually; therefore, Welsh-language PROM translation is necessary. Conceptually equivalent and patient-friendly translations of PROMs are essential to ensure that the data they collect are equivalent to that collected via their corresponding original language versions. As a result, the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) have developed best practice principles for PROM translations. This article reflects on a body of work undertaken over 8 years between 2016 and 2024 to translate a series of PROMs from English to Welsh by attempting to follow these principles. While effective in most areas, they do not provide guidance on reaching an appropriate register in languages with more than one register, dealing with multiple dialects, or how to recruit enough participants for cognitive debriefing when there are limited numbers of native speakers. Lessons have been learned while trying to manage these challenges in Wales, and recommendations for future practice are provided.

Background: Bypassing primary healthcare (PHC) in favor of higher-level providers is a persistent issue in many low- and middle-income settings, particularly in rural China, where primary eye care (PEC) remains underutilized. Understanding patient preferences and determinants of care-seeking behavior is crucial for promoting patient-centered outcomes and strengthening PHC utilization.

Objectives: This study aimed to identify the key service attributes and patient characteristics influencing rural residents' preferences for PEC in Xinjiang, China, and to assess the role of health literacy in mitigating PHC bypass behavior.

Methods: A discrete choice experiment (DCE) was conducted among 465 rural adults (aged ≥ 18 years) in northern Xinjiang, who were selected using a multistage stratified sampling approach. Six PEC service attributes-cost, convenience, organizational form, provider type, service frequency, and feedback mechanism-were tested. The cost and convenience attributes each had four levels, while the other four attributes had three levels. Respondents completed a structured questionnaire assessing knowledge, attitudes, and practices related to eye health. Data were analyzed using mixed logit and latent class models to estimate preference heterogeneity and willingness to pay (WTP) for different attributes.

Results: Rural residents demonstrated strong preferences for services provided by county hospital ophthalmologists, lower costs, shorter travel times, and comprehensive feedback, including medical advice and verbal explanations. PHC-based PEC delivered by township or village doctors was significantly less preferred, requiring monetary compensation to offset perceived utility loss. Enhanced feedback mechanisms and higher health literacy were associated with greater acceptance of PHC-based services. Latent class analysis revealed significant heterogeneity, identifying subgroups that were sensitive to cost and convenience, as well as a large subgroup with low engagement and a tendency to rely on higher-tier care. This subgroup exhibited the lowest preference for PEC services at the PHC level, highlighting a potential barrier to the utilization of local services.

Conclusions: Efforts to strengthen PHC-based PEC in rural China must address both structural and perceptual barriers, prioritizing quality improvement, provider-patient communication, and health literacy interventions. Patient-centered service design, informed by DCE-derived preferences, may reduce bypassing and promote more equitable access to eye care.

Background: Acne-induced hyperpigmentation (AIH) has a considerable impact on patients' quality of life (QoL). In the phase 4 LEAP trial, patients treated with the topical retinoid trifarotene showed faster improvement in AIH compared to patients treated with its vehicle cream (VC) at week (W) 12.

Objective and methods: This was a cross-sectional, blinded, qualitative interview study embedded in the phase 4 LEAP trial (NCT05089708), which assessed the safety and efficacy of trifarotene (50 μg/g) versus its VC in the treatment of acne. The study was conducted with a subsample of participants between June and November 2022 at a subset of nine clinical sites in the United States (US). Participants were adults (18-34 years) who were randomized to receive trifarotene (50 μg/g) or VC, along with a daily skincare regimen (moisturizer, cleanser, photoprotection). After confirming participants' end-of-treatment visit during W24, telephone interviews were conducted using a semi-structured interview guide. Participants' perspectives regarding baseline AIH symptoms, changes in AIH from baseline to W24, and treatment satisfaction were assessed and analyzed using thematic analysis in ATLAS.ti to better understand trends across trial arms.

Results: Thirty participants (n = 12 in the trifarotene group and n = 18 in the VC group) (mean [standard deviation {SD}] age, 24.8 [4.7] years; 80.0% male) were interviewed. At W24, more participants in the trifarotene group (100%) than the VC group (83.3%) reported an improvement in AIH and that the change was meaningful (91.7 and 83.3%, respectively). AIH severity change (mean [SD]) from baseline to W24 was higher in the trifarotene group (-5.5 [2.5]) than the VC group (-3.5 [2.1]). More participants in the trifarotene group (≥ 90%) than in the VC group (≥ 73%) reported a meaningful improvement in their emotional functioning, personal care/hygiene, and social life/relationships. Treatment satisfaction was higher in the trifarotene group (mean [SD] = 8.6 [2.1]) than in the VC group (mean [SD] = 7.6 [3.1]).

Conclusion: All trifarotene-treated patients reported improvements in AIH and that their treatment expectations were met. Findings support that trifarotene plus a skincare regimen provides greater benefit in the improvement of AIH than its VC. However, VC plus a skincare regimen also appeared to improve patients' perceptions of their AIH.

Patient and caregiver involvement in developing clinical guidelines is widely recommended. However, among current atopic eczema guidelines, only a minority incorporates the patient perspective. Much of the general guidance on patient involvement can be applied to atopic eczema. However, there are specific factors to consider in order to embed sustainable patient involvement in the atopic eczema guideline development process. These include the selection of patients and caregivers to represent the heterogeneity of the disease and of the patient experience. The psychosocial impact of the disease suggests support requirements that enable patients to be equal partners with other stakeholders. The priorities and feedback of patients  with atopic eczema can differ from those of healthcare professionals and are central to shaping the research and implementation that form part of a guideline cycle. Barriers to patient involvement in terms of resources or healthcare policy need to be addressed so that patients and caregivers can be active participants in developing guidelines that are culturally, racially, socially and geographically appropriate. We suggest that steps can be taken to facilitate patient involvement in patients and caregivers living with atopic eczema and offer a framework to support patient involvement as standard practice in guideline development.

Background: Hospital overcrowding in Singapore has reached a critical level, primarily owing to an ageing population and an increased demand for medical services. Alternative care models such as hospital-at-home (H@H) programmes have been introduced to address this issue, utilising telehealth and home-based care. We conducted a choice experiment to determine the most preferred characteristics of a hospital-at-home programme in Singapore.

Methods: A desktop review, interviews with clinicians and an expert panel helped to decide on the attributes and levels of the choice sets. The attributes were the type of care team, communication method, care providers, out-of-pocket costs, additional services, and place of return to the hospital if needed. We obtained information from adult Singaporeans (age > 21 years) admitted to a hospital within the past 2 years using an online panel. The design was a fractional factorial study. A pilot study and a redesign helped to refine the choice experiment. The survey used two unlabelled alternatives.

Results: The total study sample consisted of 602 respondents. The respondents did not have a particular preference for the type of care team, communication method or additional services provider. However, there was a strong preference for having a doctor as part of the care team and being readmitted to a specific ward rather than the emergency department. The analysis of relative importance revealed that out-of-pocket costs (71%) and the composition of care providers (12%) were the most significant attributes in decision-making. We found an anticipated adoption rate of H@H was over 83% compared with inpatient care.

Conclusions: Clinically eligible patients in Singapore are highly likely to accept the H@H care model. An approved private provider could provide the service without reducing patient satisfaction and trust.

Background: Advances in genomic technologies have driven a substantial shift in cancer care, including early screening and targeted interventions for high-risk individuals who have not received a cancer diagnosis. Understanding patients' experience of care and their associated outcomes is essential to effectively delivering precision medicine. These outcomes are usually evaluated through patient-reported measures (PRMs), rather than administrative data.

Objective: We conducted a systematic review of literature to identify, describe and qualitatively summarise the PRMs employed when individuals underwent genetic testing for heritable cancers. Risk of bias was not assessed as the emphasis was on description.

Methods: Search terms included cascade screening OR carrier screening OR genetic testing OR genetic counselling AND cancer AND patient-reported outcomes AND NOT Review (publication type). Reviews, meta-analyses and protocols were excluded.

Results: A total of 474 articles were identified using PubMed in May 2025, with studies only included where the outcome measure was reported by the patient, who was in receipt of a germline genetic test for heritable cancer. Following full-text review, 39 studies involving 16,523 participants were included for data extraction, with grouping undertaken by one reviewer and checked by another. We identified 83 measures, with 30 of them being genetics-specific PRMs. These PRMs focussed on knowledge of genetics, perceptions, concerns, interest and acceptability. The majority of the included studies were conducted in the USA (61.5%) and included patients with breast cancer (n_breast = 8; n_{breast&ovarian} = 15) and those of Caucasian/European ancestry (70%) with at least a college education and full-time employment (77.7%). Notably, only 16 studies included only participants diagnosed with cancer, and of the remaining, 7 included participants with a strong family history.

Discussion: Given differences in legislative frameworks regarding the use of genetic information across countries, most included studies were conducted in countries where discrimination based on genetic features is illegal. It was also evident that studies on cancers other than breast are warranted and could inform standardised collection of key outcomes across the cancer spectrum. Likewise, there was an overrepresentation of studies including white, well-educated and employed participants. This review has identified, described and summarised what types of measures have been used when patients have undergone genetic testing for cancer and highlighted the urgent need for development of additional PRMs in this area, particularly in cancers other than breast.

Funding: This work was supported by funding from the Royal Hobart Hospital Research Foundation.

Background: Understanding the treatment experiences and satisfaction of people with HIV using two-drug regimens, including dolutegravir/lamivudine (DTG/3TC), is crucial to assess ongoing needs and meet Ending the HIV Epidemic goals.

Methods: PAIRED is a mixed-methods study comprising a quantitative cross-sectional survey (using validated instruments to assess treatment satisfaction, adherence, and health-related quality of life [QOL]) and qualitative interviews. Adults in the USA who switched to DTG/3TC while virologically suppressed and had been using DTG/3TC for ≥ 3 months were eligible. All participants were surveyed, and a sub-set participated in in-depth interviews. Thematic analysis was performed to discern overarching interview themes.

Results: Overall, 474 participants completed the survey (assigned female sex at birth, 31%; non-white, 48%; aged ≥ 50 years, 50%); 20 of them participated in qualitative interviews. Treatment satisfaction, adherence, and QOL scores were high. Six themes were extracted from interviews: (1) fewer drugs are important in HIV treatment because of associated reductions in long-term drug exposure and perceived risk of toxicity; (2) high DTG/3TC satisfaction is largely due to achieved expectations regarding efficacy and safety; (3) DTG/3TC simplicity and convenience enable more freedom and autonomy; (4) treatment advancements led to HIV no longer being a "death sentence"; (5) managing HIV is no longer the main concern relative to other health conditions; and (6) some HIV unmet needs remain.

Conclusions: A large representative population with HIV had primarily positive experiences and few needs after switching to DTG/3TC. These data complement clinical trial data and support DTG/3TC effectiveness and tolerability in real-world settings.