Patient-Patient Centered Outcomes Research最新文献

Background: Children with congenital colorectal conditions require care from multiple health professionals. However, evidence on the value of a multidisciplinary care model is lacking. This study aimed to elicit public preferences and willingness to pay for a multidisciplinary care model for children with congenital colorectal conditions.

Methods: We developed a discrete choice experiment (DCE) that was administered to 807 members of the Australian public online. A Bayesian D-efficient design consisting of 20 choice tasks was split into 2 blocks of 10 choice tasks per respondent. Five attributes elicited through mixed methods included make-up of the multidisciplinary team; responsibility for care coordination; duration of access; provision of educational information; and cost. Choice data were analysed with a panel error component mixed logit model. Willingness to pay for each DCE attribute and level was estimated using the unconditional population moments estimates.

Results: The Australian public demonstrated preference for a multidisciplinary care model. They showed preference for long-term access, having a care coordinator and provision of additional information. The public was willing to pay Australian dollars (AU) $64,275 for a multidisciplinary care model comprising an essential multidisciplinary team (including a surgeon, clinical nurse consultants, a psychologist, a social worker, stomal therapists, a child life therapist and a dietitian) with care coordination and information booklets and ongoing care until the child reached adulthood. We observed preference heterogeneity associated with gender, parenthood status and household income.

Conclusions: The Australian public valued the multidisciplinary care model for children with complex colorectal conditions. Our findings can be used to inform the design of a multidisciplinary care model and to inform cost-benefit analyses as part of broader healthcare system implementation.

Patient engagement is expected to improve the safety and efficacy of anticoagulant medication. By identifying the factors and patient characteristics influencing patients' decisions regarding anticoagulant medication, we aim to support healthcare professionals regarding patient engagement. This review of discrete choice experiments (DCEs) explores the considerations and preferences of patients with various underlying conditions when choosing their anticoagulant medication. Two international databases were searched in March 2024: PubMed and Web of Science Core Collection. The search was updated in July 2025. Eligible studies included original DCE studies that explored the considerations and preferences of patients and covered long-term anticoagulant use beyond the hospital setting (direct oral anticoagulants, vitamin K antagonists, antiplatelet agents, and low molecular weight heparins). The initial search identified a total of 174 records, after which two authors independently assessed the articles for both the title and abstract and the full-text inclusion rounds. After the update, this resulted in the inclusion of 13 articles. The PRISMA 2020 statement was followed and the quality of the included studies was assessed. The results show that overall patients prioritize safety and effectiveness of anticoagulants over convenience factors. If only convenience factors were taken into account, frequency of administration emerges as the most important. Subgroup analysis showed that patient preferences vary based on geographic, demographic, and socioeconomic factors, health status, as well as previous experiences with anticoagulation medication or related health issues. By acknowledging the impact of these diverse factors on patient preferences, healthcare professionals can better support safe and effective anticoagulant care tailored to the needs of individual patients.

Background: Early pregnancy loss (EPL) is associated with significant emotional burden. While multiple management options exist, psychological aspects are often underrepresented in decision-making support. The objective of this study is to identify criteria that are important to women when choosing between expectant, medical, and surgical management of EPL, with particular attention to psychological aspects.

Methods: A mixed-methods study using the Analytic Hierarchy Process (AHP) was conducted. Criteria were identified via literature review and a focus group. Thirty-seven women with EPL history completed structured telephone interviews with pairwise comparisons. Quantitative data were analyzed to calculate criterion weights and assess consistency. Logistic regression explored factors associated with inconsistent responses.

Results: Among 37 participants, 'Minimizing psychological distress' emerged as the most important criterion (local weight = 0.381), with sub-criteria such as 'Experiencing miscarriage as a natural process' (0.384; 0.252) and 'Avoiding hospital treatment' (0.355; 0.425) ranked most important for women preferring expectant and medical management, respectively. Women preferring surgical management prioritized 'Short time to miscarriage completion' (0.389). Sixty-five percent of participants demonstrated acceptable preference consistency (consistency ratio ≤ 0.2). Lower education showed the greatest and statistically significant negative influence on the consistency of the preferences in the regression models.

Conclusion: Preferences varied by treatment choice, indicating that psychological distress is defined differently across women. The AHP method enabled nuanced insights into individual decision-making. Emotional factors may influence preference patterns and should be considered in clinical counselling. These findings support the need for individualized counselling and shared decision-making. The results may inform the development of clinical tools and guidelines.

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: 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.

In phase I clinical trials, the recommended phase II dose (RP2D) is usually set at or near the maximum tolerated dose (MTD), which is determined based on the observation of dose-limiting toxicities (DLTs). Clinicians typically evaluate toxicities using the National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE), with grade three or higher toxicities classified as DLTs. However, it has been repeatedly demonstrated that physicians tend to underestimate patient's symptoms. Therefore, patient-reported outcomes (PROs), especially the NCI PRO-CTCAE questionnaire, can complement clinician assessments by providing direct patient input on adverse events. This integration could lead to a more accurate definition of DLT and better informed RP2D decisions. Moreover, PROs could optimize sample size strategies in later-stage trials and enable comparison of health-related quality of life (HRQoL) data with synthetic control arms to confirm the benefit of a drug, especially in rare oncogene-driven subsets. Whilst stakeholders and regulatory authorities acknowledge the value of integrating PROs early in drug development, they emphasize the lack of methodological guidelines to support broader adoption. The integration of PROs represents an opportunity to improve the patient-centeredness of phase I trials, ultimately strengthening the drug development process.

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.

Objective: This study presents findings from a pilot study that aimed to examine the feasibility of routine measurement of quality of life in residential aged care, including the examination of barriers to and facilitators of collecting and using that data to improve quality of care.

Methods: This study was conducted at two not-for-profit residential aged care facilities in Melbourne, VIC, Australia. All residents were eligible to participate if consent was provided. Self-reported quality-of-life data were collected from residents, alongside proxy-reported data from aged care staff and relatives, primarily using the EQ-5D-5L in addition to a randomly assigned second measure (i.e. The Adult Social Care Outcomes Toolkit [ASCOT], Quality of Life-Aged Care Consumers [QOL-ACC], EQ Health and Wellbeing Instrument [EQ-HWB]). Feasibility was assessed in terms of missing data, residents' level of engagement and understanding, and difficulty experienced by staff and relatives in providing proxy reports. Perceived facilitators and barriers were identified via qualitative interviewers with staff who collected the data.

Results: From 103 consenting participants, we gathered quality-of-life data through self-report (n = 90), staff proxy-report (n = 101) and family proxy-report (n = 49). Most residents (94%) were able to respond to the EQ-5D-5L questions and residents' level of engagement was rated by staff as good. Only a few missing values (0-10%) were recorded for the EQ-5D-5L. Qualitative findings indicate that while quality-of-life data collection has benefits, barriers include time pressures, residents being too unwell to self-report, staff uncertainty about responding on their behalf and issues with the measure itself.

Conclusions: While it is feasible to routinely collect quality-of-life data in residential aged care, addressing the barriers identified will optimise the efficiency of the process and maximise the use of data to guide quality improvement strategies.

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.