Patient-Patient Centered Outcomes Research最新文献

Background: Cystic fibrosis (CF) is a progressive and multi-organ genetic condition most renowned for reduced lung function. The recent introduction of elexacaftor/tezacaftor/ivacaftor (ETI) has transformed physical health and life expectancy for many people with CF.

Objectives: This mixed methods study aimed to compare levels of physical health and self-concept before and after ETI commencement and explore lived experiences of ETI impact on self-concept: an individual's overarching sense of identity.

Methods: This study applied a mixed methods approach, using qualitative data to enrich and augment the insights gained by the quantitative data. Longitudinal quantitative data were collected from 20 adults to compare mean lung function, frequency of pulmonary exacerbations, body mass index (BMI), and self-concept scores before and after ETI commencement. Qualitative data were collected from 13 of these adults through in-depth, unstructured interviews, which were thematically analysed to identify key insights.

Results: Descriptive and comparison statistical analysis demonstrated a significant increase in mean lung function and a significant decrease in mean frequency of pulmonary exacerbations following ETI commencement. No significant difference in BMI was identified. While mean self-concept scores before and after ETI commencement were not significantly different, thematic analysis of interview data highlighted that many participants had experienced self-concept recalibration across physical, social, and psychological domains of life related to ETI. The overarching theme of Self-concept recalibration and six subsequent themes emerged: Physical self: A 'new' CF and Weight gain - friend or foe; Social self: The gift of time and Goals and possibilities; and Psychological self: from Surviving to thriving and Into the unknown.

Conclusions: Findings highlight significant physical health improvement for many people with CF, and the potential for ETI to positively impact self-concept. However, challenges and nuanced self-concept experiences were also identified.

Practice implications: Integration of a self-concept approach to CF care is recommended.

Background and objective: Advances in prostate cancer (PC) therapy have improved survival and expanded systemic treatment options. To support treatment decision-making, it is important to understand tradeoffs patients are willing to make between survival and other factors such as side effects. This targeted literature review evaluated the results of discrete-choice experiment (DCE) studies assessing patients' and healthcare professionals' (HCPs') preferences for systemic therapies for PC.

Methods: PubMed was searched for PC DCE studies evaluating patient or HCP preferences for systemic treatment published through April 2025. Attribute importance rankings were summarized descriptively and stratified by respondent type and by PC stage corresponding to the treatments assessed. Studies primarily assessing preferences for androgen deprivation therapy, which is used in patients with biochemically recurrent non-metastatic castration-sensitive PC, were categorized as "earlier stage"; those assessing systemic combination therapies for metastatic or castration-resistant PC were categorized as "late stage."

Results: Seventeen eligible studies were included (late-stage PC treatments, n = 10 patient studies and n = 3 HCP studies; earlier-stage PC treatments, n = 4 patient studies, with one reporting an HCP subgroup). Overall, patients and HCPs showed the strongest preferences for attributes related to efficacy outcomes (e.g., extending overall survival, clinical progression). Preferences for avoidance of long-term safety attributes, such cardiovascular side effects, were generally ranked higher in late-stage studies than in earlier-stage studies. Meanwhile, avoidance of short-term tolerability attributes, such as injection-site reaction and sexual function, were prioritized and measured only in earlier-stage studies. Preferences differed between respondent types for late-stage treatments, with HCPs placing greater importance on tolerability attributes (fatigue, central nervous system side effects) and patients prioritizing safety (avoiding falls, cardiovascular side effects).

Conclusions: Despite differences in safety and tolerability attribute rankings among respondents across earlier-stage and late-stage PC treatments, efficacy outcomes were most important to patients and HCPs.

Background: Bacillus Calmette-Guerin (BCG) following transurethral resection of bladder tumor (TURBT) is the current standard of care (SOC) for high-risk non-muscle invasive bladder cancer (HR-NMIBC). Several emerging therapies, such as immune checkpoint inhibitor (ICI) therapies in combination with BCG will provide new treatment options for patients. Patient preference studies can provide quantitative evidence of the trade-offs patients are willing to make among attributes of current and emerging treatments. Qualitative research is critical to developing preference elicitation instruments that capture decision-relevant treatment attributes. This study aimed to elicit information about HR-NMIBC patients' treatment priorities and evaluate a preliminary list of attributes and levels to be used in a patient preference survey.

Methods: A targeted literature review produced a preliminary list of 11 attributes. Qualitative, semi-structured, multi-method telephone interviews with 12 patients with HR-NMIBC in the USA were used to explore treatment decision drivers and assess comprehension and relevance of the attributes and levels. Patients with HR-NMIBC were recruited via patient advocacy groups. A combination of concept elicitation, cognitive debriefing, and rating and ranking exercises were used to assess patients' priorities and perspectives regarding decision-relevant treatment attributes.

Results: Efficacy (event-free survival) was the most important attribute to all patients. The risk of serious/life-threatening events was also very important to patients. The risk of experiencing bladder problems and the choice between different administration procedures (route, frequency) were also important in the context of patients' treatment choices. The preliminary list of attributes and levels was subsequently refined to reflect patient priorities and incorporate feedback from expert advisors.

Conclusions: This is the first study, to our knowledge, that provides qualitative evidence regarding patients' preference for potential future treatment options for HR-NMIBC. Findings will inform the final selection and framing of attributes and levels to be included in an upcoming benefit-risk preference study. Future research is warranted to quantify any trade-offs that patients with HR-NMIBC are willing to make regarding administration, benefit, and risk attributes.

Background: Qualitative interviews can supplement clinical trial results by providing in-depth insights into participant experiences. In the recent SURMOUNT-OSA (NCT05412004) phase III trials, tirzepatide demonstrated favorable efficacy and safety in patients with moderate-to-severe obstructive sleep apnea and obesity. To explore participants' obstructive sleep apnea and treatment-related experiences, qualitative exit interviews were held with a subset of trial participants.

Methods: One-on-one qualitative telephone interviews were conducted as participants exited the trial. Participants described their obstructive sleep apnea symptoms, impacts, and sleep quality before and after participation in the trial. They further reported on the meaningfulness of treatment-related changes, experiences, and satisfaction. Interview transcripts were analyzed using ATLAS.ti.

Results: Seventy US-based and 12 Germany-based participants (tirzepatide n = 49, placebo n = 33) had a mean age of 52.7 years. Most were male (61.0%), non-Hispanic (87.8%), and White (91.5%). The "worst" obstructive sleep apnea symptoms experienced overall included snoring and temporary breath cessation, and the "worst" impacts were sleep-related impairment and sleep disturbance. Greater proportions of tirzepatide-treated than placebo-treated patients reported improvements in key symptoms and impacts of obstructive sleep apnea and considered them to be meaningful. Tirzepatide-treated participants also reported improved sleep quality and daytime functioning. Treatment satisfaction was higher among tirzepatide-treated participants than placebo-treated participants. The most common treatment-related likes were ease of treatment administration, weight reduction, and obstructive sleep apnea symptom improvement.

Conclusions: These findings highlight the range of symptoms and impacts experienced by individuals with obstructive sleep apnea and obesity, and support a positive effect of treatment with tirzepatide.

Background and objective: Patient preference studies have been used to identify aspects of medical products that matter to patients. This paper sought to summarise patient preferences for treatment processes (mode and frequency).

Methods: Search strategies based on existing validated search filters were applied to databases including MEDLINE, Embase, Scopus, EconLit and Health Preference Research. Additional references were obtained by searching the reference lists of systematic reviews. English-language peer-reviewed studies involving patients that quantitatively elicited preferences and compared different modes or frequencies were eligible. Data extraction was performed by two reviewers using Covidence. As a heuristic, we considered if process attributes were ranked in the top two attributes. The Purpose, Respondents, Explanation, Findings, Significance (PREFS) checklist (0-5, higher = better) was used to evaluate study quality.

Results: Data were extracted from 164 articles of an identified 16,357 (12,872 screened by title/abstract and 795 full text screened). Mean study quality was 3.48 on PREFS (standard deviation = 0.70), but patient engagement was rarely reported in studies (n = 20, 12.2%). Most studies included both mode and frequency attributes, either presented as a combined attribute (n = 65, 39.6%) or as individual separate attributes (n = 43, 26.2%). Regardless of how attributes were presented, mode/frequency attributes were infrequently in the top two attributes. In terms of level preference, when an oral mode was included as a level, the oral level was the most preferred level in nearly all studies. Patients with less severe disease or who were less experienced with treatment tended to prefer oral modes or have preferences against injection.

Conclusions: Mode/frequency attributes are not necessarily the most important attributes, but patients, particularly those with less treatment experience or more mild disease, may have strong preferences. Although PREFS scores indicated high study quality, future studies would benefit from deeper patient engagement. In line with ISPOR's definition, patient engagement should extend beyond participation to meaningful involvement across the research lifecycle, including opportunities for co-authorship and partnership in study design, conduct and dissemination.

Background: Early detection of cancer reduces mortality and morbidity, but conventional screening methods often face challenges such as invasiveness, limited accessibility and high resource demands. Emerging cancer screening technologies could overcome these barriers, yet their adoption depends heavily on public acceptance. This systematic review synthesises evidence from discrete choice experiment (DCE) studies examining population preferences for emerging cancer screening technologies.

Methods: A systematic review was conducting following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. PubMed, Embase, Scopus, Web of Science and EconLit were systematically searched for studies published up to August 2025. Eligible studies employed DCEs to assess preferences for modalities including liquid biopsy, multi-cancer early detection tests, artificial intelligence reading systems, genetic and genomic testing, mobile Health applications, nanopill-based screening and breath analysis. Studies were screened independently by two reviewers and assessed for reporting quality using the DIRECT checklist. Data were summarised using descriptive statistics and narrative synthesis.

Results: In total, 23 studies published between 2014 and 2025 were included, most focusing on genomic testing (n = 8) or liquid biopsy (n = 5). Across studies, 32 attributes were identified and categorised under clinical risk, modality characteristics, screening process, outcomes, or ethical considerations. Respondents generally favoured screening programmes that were clinically effective, accessible and minimally disruptive. Sensitivity was most frequently reported as the most influential attribute. Modality specific attributes influencing public preferences were identified, including data privacy and insurability for genetic and genomic testing, and the role of test providers for mHealth technologies and AI screening tools. Heterogeneity in preferences, attribute importance and predicted uptake were observed across many studies, highlighting differential attitudes towards cancer screening and modality specific attributes within the population.

Conclusions: This systematic review provides the first synthesis of DCE evidence across a wide range of emerging cancer screening technologies. Findings reveal that public preferences were shaped not only by clinical performance, but also by convenience, cost and the perceived credibility of new modalities. Considerable heterogeneity in preferences signals the need for tailored strategies to engage different population subgroups and avoid widening disparities in uptake. As these technologies move towards clinical adoption, robust governance and regulatory safeguards will be essential to ensure their translation into equitable and effective cancer screening programmes.

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.

Background: Patient and public involvement (PPI) in health research is gaining global momentum through initiatives such as INVOLVE (UK), the Patient-Centred Outcomes Research Institute (USA), and the Strategy for Patient-Oriented Research (Canada). However, its implementation in Africa remains limited and lacks context-specific guidance. To address this gap, the Sickle Africa Data Coordinating Center supported the development of the Patients as Partners in Sickle Cell Disease Research (SCD-PAPIR) Framework to guide meaningful patient engagement and involvement in SCD research in Africa.

Methods: An iterative, participatory approach was adopted. The process involved the creation of a SCD PAPIR working group comprising SCD patient advocates in 14 African countries; 18 virtual working group meetings, one public webinar, and three in-person workshops. The framework was co-created through facilitated discussions reflecting on prior engagement in SCD research as a patient partner, and best practices for effective patient-researcher collaborations. Patient-only sessions and leadership roles were integrated to ensure safe spaces and to minimise power imbalances.

Results: The SCD-PAPIR Framework positions SCD patients and their caregivers as citizen researchers. Key to the framework is the principle of subsidiarity, which affirms the agency and experiential knowledge of patients while calling for epistemic humility from researchers. Its core pillars include valuing patient expertise, transparent communication, resource sharing, patient empowerment, collective learning, amplification of patient voice, continuous feedback, and shared benefits. Best practices emphasise two-way communication, addressing power asymmetries, co-learning and patient empowerment, co-ownership of outputs, and formalised PAPIR structures.

Conclusion: The SCD-PAPIR Framework provides a contextually grounded model for patient-engaged research in Africa and contributes to efforts to decolonise health research by positioning patients as co-creators of knowledge, and not merely a data source. The effective implementation of the framework will require investment in institutionalising PAPIR in SCD research. Future work should focus on designing implementation toolkits, developing PPI training modules for researchers and patient advocates, and adapting the framework to other health conditions.

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.