Patient-Patient Centered Outcomes Research最新文献

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.

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.

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: People receiving treatment for advanced cancer invest substantial portions of their survival time receiving healthcare, labelled the 'time toxicity' of treatment. Although qualitative research has examined the impact of time burden on patients and their caregivers, its influence on treatment decision-making is unclear.

Objective: Our objective was to explore treatment decision-making with patients with advanced gastrointestinal cancer, their caregivers, and oncologists, and unmask the role of time burden in those decisions. The objective was to inform the design of a subsequent discrete-choice experiment (DCE) investigating the importance of time burden in treatment decision-making.

Methods: A two-step process was used. Factors relevant to treatment decision-making were discussed as part of semi-structured interviews. Responses were analysed using thematic analysis with a focus on measurable themes relevant to the development of candidate attributes for a DCE. Second, we reviewed stated-preferences studies in the field of treatment decision-making in cancer and compared the results with the candidate attributes identified from interviews.

Results: Interviews with 45 participants (20 patients, 10 caregivers,15 gastrointestinal oncologists; 53% metropolitan) revealed 4 themes and 6 candidate attributes: expected survival benefit of treatment, impact of physical side effects, effect on day-to-day functioning, route of administration, healthcare contact days, and planned length of the treatment course. Review of 45 published studies yielded no additional attributes.

Conclusions: This study identified six candidate attributes for a forthcoming DCE on time burden in advanced cancer care. These findings support growing efforts to quantify and address time toxicity in cancer treatment decision-making.

Purpose: The benefits and barriers of using patient-reported outcome measures (PROMs) in routine clinical practice have been examined extensively, but there has been limited in-depth exploration of the experience of PROMs by stakeholders and consumers. This study sought to understand the experiences of children with life-altering skin conditions, their caregivers and treating health professionals regarding the routine use of electronic PROMs.

Methods: Using interpretive description, data were gathered through qualitative interviews and researcher observations in two outpatient clinics within a major metropolitan paediatric hospital. In total, 48 interviews were conducted with children (aged 11-16 years) attending burn scar or vascular anomalies clinics, their caregivers (children of all ages) and treating health professionals.

Results: The therapeutic relationship was a central organising concept and crucial for providing person-centred care. Within the therapeutic relationship, ePROMs were used to prioritise where to start alongside a high-quality conversation. Study themes identified were shaping (or not shaping) care, taking a considered approach to the use of ePROMs, and aligning values and priorities with the everyday reality. ePROMs did not shape care or capture the priorities of all children and caregivers. A spectrum of sentiment from negative to positive was expressed by families regarding the routine use of ePROMs, differing to the predominantly positive sentiment by health professionals.

Conclusions: ePROMs should be used alongside high-quality conversations to assist in eliciting, understanding and evaluating what matters to children and caregivers but must be used within a therapeutic relationship.

Background and objective: Telephone cancer information and support services (CISS) deliver essential evidence-based resources for people living with cancer. This research aimed to describe how attributes and levels were developed for a future discrete choice experiment to elicit preferences for operational characteristics of a CISS, focusing on Cancer Council Victoria's service.

Methods: Using a mixed-methods approach guided by the ISPOR checklist for conjoint analysis in healthcare, initial attributes were developed using an artificial intelligence framework to analyse CISS calls (January 2018-December 2021), focus groups with people with cancer and carers using the CISS (July-August 2022), and a systematic literature review of qualitative studies. A four-stage descriptive process guided attribute and level development. An expert panel of researchers (n = 10), a CISS staff member, a person with lived experience of cancer and a consumer-only panel (n = 7) met monthly to prioritise, refine and finalise attributes by consensus.

Results: Call data analysis (people with cancer n = 7701; carers n = 5500), six focus groups (people with cancer n = 10; carers n = 11) and a systematic literature review of qualitative studies generated 14 candidate attributes. The expert panels selected seven final attributes, each with three levels: follow-up call, operating hours, additional technology, operator type, operator consistency, call length and service fee.

Conclusions: Transparent reporting of the discrete choice experiment design process is essential for credible interpretation. The four-stage approach enhanced the comprehensibility of the experiment, as multi-modal data ensured the selected attributes and levels accurately reflect CISS caller priorities, which may be applicable to other choice-based studies.