Patient-Patient Centered Outcomes Research最新文献

Objective: Nearly 30% of kidneys from deceased donors are discarded annually in the USA. A recent study indicated that a significant number of patients would accept lower-quality kidneys to avoid long waits. We expand on previous work to assess how the distribution of patient preferences for lower-quality kidneys would change with patient time on the transplant list.

Methods: We conducted a discrete-choice experiment with US pre-transplant patients waitlisted for kidneys from deceased donors. Respondents were asked to evaluate tradeoffs between expected graft survival and waiting time. We used a logit-based regression with patient covariates to explain membership of three patient-preference phenotypes previously identified with these data. Specifically, we tested the degree to which phenotype membership changed with waiting time and how such changes were moderated by observable patient characteristics such as age, insulin use, recipient function, time on dialysis, and household income.

Results: Waiting time had a nonlinear effect on phenotype probabilities, with more patients expected to be willing to accept lower-quality kidneys as waiting time increases. Patients with longer insulin dependence, lower income, and limited function were more likely to accept lower-quality kidneys. Higher income was significantly associated with the probability of being willing to wait for better future kidneys. Dialysis time had no significant effect.

Conclusions: Our analysis provides insights into time-varying effects using cross-sectional data. Results suggest that patient preferences for organ acceptability vary with waiting time and are moderated by health status and socioeconomic factors. Longer waits and worse health statuses were generally associated with greater willingness to accept lower-quality kidneys.

Background: Patient and public involvement (PPI) is crucial for aligning research with public needs, reducing research waste, and enhancing the relevance and quality of evidence. Evaluating PPI is necessary to ensure its effectiveness. However, despite its recognised importance, researchers have reported a lack of robust tools for evaluating PPI systematically. To clarify which tools are used to evaluate PPI in health research, we conducted a scoping review.

Objective: We aimed to identify and map evaluation tools that have been used in empirical health research studies to assess PPI, and to describe reported outcomes related to PPI.

Methods: A scoping review was conducted in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews (PRISMA-ScR) guidelines. A comprehensive search was undertaken in MEDLINE, Embase, CINAHL and Scopus to identify studies published between 2021 and 2024 describing evaluation tools for PPI in health research contexts. Studies evaluating PPI were included, irrespectively of tool validation. Study selection and data charting were guided by principles from structured extraction frameworks and results were synthesised descriptively and narratively.

Results: Thirty studies were included. Positive personal outcomes for PPI partners were reported, including increased well-being and skill development. Despite the existence of robust validated evaluation tools, many were adapted or developed de novo. An 'us vs them' dynamic was noted, reflecting differing engagement levels between PPI partners and researchers during evaluations. The need for additional training for both PPI partners and researchers to enhance collaboration was a recurring theme.

Conclusions: Patient and public involvement evaluation tools are often developed or adapted to fit specific contexts, with multiple methods used for assessment. Challenges include low researcher response rates in evaluations and the need for better researcher preparedness for PPI.

One-to-one interviews and focus groups are used to generate qualitative data about patients' health outcomes and inform the development of patient-reported outcome measures (PROMs). In the development of PROMs for young people, visual elicitation tools can be used with one-to-one interviews and focus groups to enhance the data generation process and data quality. This article aims to (1) provide a detailed description of how visual elicitation tools can be applied in the development of youth-specific PROMs using the GENDER-Q Youth study as an example and (2) share the lessons learned from the GENDER-Q Youth study with PROM developers who are considering using visual elicitation tools in their own virtual qualitative studies. This article discusses processes that took place within the context of a mixed-methods, multi-step study to develop a PROM for youth receiving gender-affirming care called GENDER-Q Youth. Step one was an applied qualitative health research study using an interpretive description approach. Virtual concept elicitation interviews were conducted with transgender and gender diverse youth aged 12-18 years at recruitment using an optional timeline-based visual elicitation tool (i.e., before interviews, youth were invited to create a timeline about their gender-affirming care journeys). The research team navigated ethical and logistical challenges associated with using timeline activities during the data generation process. These challenges occurred during the pre-interview stage (e.g., mailing activity supplies) and during interviews (e.g., incorporating the timeline activity into the interview). Details about the approach used by the research team, challenges faced, and lessons learned are discussed. When conducting one-to-one virtual concept elicitation interviews, visual elicitation tools have the potential to enhance the quality of data generated about participants' outcomes and experiences of healthcare. Visual elicitation tools can also improve the interview experiences of both participants and researchers and are feasible to implement within the context of qualitative PROM development research with young people.

Aim: Patient-perceived treatment tolerability can affect patient ability and willingness to remain on therapy. We sought to examine completion rates for a single item of overall side effect bother at baseline and at the first on-treatment assessment, the association between this item with other patient-reported outcomes (PROs) and the odds of early discontinuation due to clinician-assessed adverse events or reasons other than disease progression.

Methods: Data were from three commercial cancer trials in solid tumours, focusing on the safety population. The GP5 item from the Functional Assessment of Cancer Therapy (FACT) was used for side effect bother. Other PROs included items on specific symptoms, functional impacts and global health status, all drawn from validated measures. Descriptive statistics were used for completion rates, and correlation and logistic regression analyses were used to examine associations. GP5 was dichotomised as 0-1 ('low') versus 2-4 ('high').

Results: Completion rates were at or above 90% at baseline for all items. GP5 completion rates were 5% lower than completion rates for other items (89.8% versus 94.9%) at baseline, but this was not seen after baseline. Among patients with non-missing baseline GP5, 11.8-15.7% of cancer treatment-naïve patients reported high bother, compared with 23.9% of treatment-experienced patients. Patients with high bother at baseline had higher odds of early discontinuation compared with those with low bother, but this was not statistically significant after covariate adjustment.

Conclusions: Continued collection of the GP5 item and concomitant work aiming to understand reasons for missingness as well as interpretation is important for evaluating tolerability in cancer trials.

Purpose: The aim of this study was to evaluate the impact of an intervention-comprising a context-adapted decision aid (DA) and online shared decision-making (SDM) training for intensivists-on the quality of goals-of-care discussions (GCDs) and the level of involvement of elderly patients in those discussions within an intensive care unit (ICU).

Methods: This was a three-phase before-after mixed-methods implementation study conducted in an ICU in Lévis, Québec, Canada. We followed the StaRI and COREQ reporting guidelines. We recruited patients aged ≥ 65 years and their attending intensivists. We video-recorded GCDs in three phases: Phase I: GCDs without a DA; Phase II: GCDs with a DA about goals of care but no online training; and Phase III: GCDs with both a DA about goals of care and online training about SDM. All GCDs recordings were transcribed verbatim. We measured the level of patient engagement by intensivists in SDM about goals of care through the OPTION scale and evaluated GCDs quality using the Audit of Communication, Care Planning, and Documentation (ACCEPT) indicators. A qualitative thematic analysis of transcriptions of the encounters was also performed.

Results: Out of 359 eligible patients, the study included 21 patients (71% men; median age, 77 years; 57% without high school diploma) and five intensivists (80% men; median age, 35 years). Despite completing online training, the DA was never used in recorded encounters. We did not perform any tests of statistical significance to compare results in each study phase because of small sample sizes over each phase. OPTION and ACCEPT scores were low in each phase, but physicians did engage in GCDs. We found that 76% of documented goals of care for life-sustaining therapy were consistent with patient preferences expressed during recorded GCD, a determination made by two independent observers. Several patients expressed confusion about GCDs. Regarding the use of a DA to support SDM in GCDs, intensivists identified several barriers and facilitators. Barriers included physician attitudes, lack of training, and systemic pressures, while a patient-centered approach and positive patient attitudes were seen as key facilitators.

Conclusion: Despite implementing a context-adapted DA and online SDM training, this study found no significant improvement in the quality of GCDs or patient engagement, notably as the DA was not utilized. Communication barriers and patient confusion about goals of care highlight the persistent challenges in achieving true SDM in the ICU. Future strategies should focus on overcoming identified barriers for successful integration of such interventions.

Trial registration number: NCT04034979.

Background: The Netherlands reformed its long-term care policy to encourage older adults to age in place with the support of informal caregivers. It remains unclear whether the available care and support options align with the needs and preferences of older adults and caregivers. Discrete choice experiments (DCE) are increasingly used to identify individual preferences. This study describes the development of attributes (e.g., emotional support) and attribute levels (e.g., psychologist and case manager) for a DCE on aging-in-place preferences among older adults and informal caregivers in The Netherlands.

Methods: Semi-structured interviews were conducted with older adults and informal caregivers to identify key components for successful aging in place. Interviews were transcribed, and reflexive thematic analysis identified patterns that led to a list of attributes. Visuals of these attributes were created and presented to a new sample of informal caregivers and older adults in focus groups to rank attributes and define attribute levels.

Results: Attributes identified through the interviews (N = 28) included housing, personal care, household tasks, transportation, social activities, digital skills, and help navigating the healthcare system. Focus groups (N = 35) found that older adults prioritized housing, while informal caregivers prioritized navigating the healthcare system. Transportation and digital skills were ranked as the least important and were excluded from the final list of attributes.

Conclusions: Our findings provide a detailed understanding of aging-in-place preferences of older adults and informal caregivers. These insights will inform a DCE to quantify preferences and provide evidence for policymakers. This study increases transparency about the process of attribute development and level selection, contributing to the quality of the final DCE study.

Background: Primary immune thrombocytopenia (ITP) adversely impacts a patient's health-related quality of life (HRQoL).

Objective: This study explored patients' experiences with signs, symptoms, and impacts of ITP and updated the conceptual disease model for HRQoL in patients with ITP.

Methods: Adult patients with ITP were included in the study. Patients with any comorbidity where fatigue was a key symptom (e.g., anemia) were excluded. A concept elicitation interview assessed signs, symptoms, and impacts from the patients' perspective, and cognitive debriefing assessed the validity of the selected patient-reported outcome instruments (ITP-Patient Assessment Questionnaire, Patient Global Impression of Severity of Fatigue, Patient Global Impression on Severity, and Patient Global Impression on Change scales). Symptoms/impacts mentioned by ≥ 50% of patients and a "bothersomeness" rating of ≥ 5 were considered salient. The preliminary conceptual model was updated based on the concepts reported by more than one patient.

Results: A total of 18 patients (mean age 42.7 ± standard deviation 14.7 years; female 78%) were interviewed. Six salient symptoms included fatigue (94%; n = 17), bruising (83%; n = 15), petechiae (72%; n = 13), difficulty staying awake during the day, difficulty falling asleep, and difficulty staying asleep (all symptoms related to sleep: 50%, n = 9). Six key impacts that emerged included those on work (72%; n = 13), worry and fear (67%; n = 12), limited physical or sporting activities, the perceived need to be cautious to avoid getting hurt, and family, friends, and social life (all 56%; n = 10).

Conclusion: This study highlighted six key symptoms of ITP and its impacts on patients with ITP. The concept elicitation findings were used to update the conceptual model, and-during cognitive debriefing-patients found the patient-reported outcome instruments easy to understand and relevant to their experience.

Background: Controlling the syphilis epidemic in Australia is a public health priority. Regular intramuscular (IM) injections of benzathine penicillin G (BPG) are the current standard of care for late latent syphilis in Australia; however, repeated IM BPG injections are painful, and treatment completion rates are low. Early-phase clinical trials have demonstrated the tolerability and safety of high-dose subcutaneous infusions of BPG (SCIP), where the total treatment dose can be delivered at a single visit. Here we describe the experiences and preferences of attendees of Western Australian sexual health clinics in the Perth metropolitan region who have syphilis and were treated with SCIP.

Methods: This was a mixed methods sub-study of a phase IIa clinical trial (ACTRN12622000349741). Eligible participants attended South Terrace Sexual Health Clinic in Fremantle, Western Australia, after referral from physicians to the South Metropolitan Health Service. Nine participants were followed up with for 24 weeks after receiving SCIP. Semi-structured interviews were conducted at three time points. All interviews were audio recorded, transcribed verbatim and underwent content and thematic analyses.

Results: Two themes were identified: (1) tolerance of SCIP and (2) SCIP is preferred over IM BPG. SCIP was well-tolerated by participants, who praised the ease and convenience of the procedure. Participants described instances of mild to moderate pain during and after SCIP, substantiated by self-reported pain scores and adapted Skindex-16 scores. All participants stated that they would prefer SCIP over IM BPG as syphilis treatment, and that they would be willing to receive SCIP again should re-infection occur.

Conclusion: All participants described tolerable infusion experiences with a clear preference for SCIP over IM BPG to treat syphilis. SCIP has potential to replace IM BPG in clinical practice. Further research is needed to determine the acceptability and efficacy of SCIP in diverse cohorts.

Background: There is increasing interest in using patient-reported outcome measures (PROMs) to assess quality of life (QoL) following hematopoietic cell transplant (HCT). However, there is limited consensus on how such data should be collected within HCT services. This survey study investigated health professionals (HCPs) views towards QoL data collection and factors affecting the use of PROMs within HCT centres in the UK.

Method: Individual survey items were based upon the Consolidated Framework for Implementation Research (CFIR). The CFIR consists of five domains which are known to affect intervention (in this instance PROM) adoption and implementation. National coverage was achieved with survey responses received from all UK adult allograft HCT centres.

Results: Findings indicated PROMs use in UK HCT services is ad hoc with PROMs mostly used as screening or diagnostic tools for emotional health or for service improvement activities including audits. HCPs reported that patient-reported outcome (PRO) data collection is limited by a lack of resource, infrastructure and referral pathways if the PROM were to flag a patient issue. A large proportion of HCPs (> 70%) noted that PRO data within their centre would be best enabled through dedicated research staff and data management infrastructure to support data collection and storage. Despite growing evidence of the utility of electronic data capture, most HCPs (> 50%) believed electronic PROMs (ePROMs) may be difficult to implement due to data protection issues and limited access to electronic devices.

Conclusions: These findings highlight the opportunities and challenges to PRO data collection in UK HCT services and demonstrate the need for practical solutions. The development of a standardised approach to PROM use among HCT recipients and investment in workforce and data management infrastructure is needed to support an integrated approach to PRO data collection, storage and use.

Background: Atypical haemolytic uremic syndrome (aHUS) is a rare life-threatening disease. Lifelong treatment with intravenous eculizumab every 2/3 weeks was recommended, but evidence is emerging that many patients can stop complement inhibition and restart should they relapse. However, little is known about the opinions and needs of patients with aHUS on withdrawal.

Objective: We aimed to understand the factors that affect decisions to withdraw from treatment.

Methods: This was a qualitative study embedded in an eculizumab withdrawal trial. Data were collected through in-depth telephone interviews from January 2019 to June 2022. We conducted a thematic analysis using a constant comparative method. Interviewees included eight adults and the parents of 12 children with aHUS approached to participate in the trial. Follow-up interviews were conducted between January 2021 and June 2024 with those who withdrew from eculizumab.

Results: The onset of aHUS had been traumatic for most. Regarding eculizumab, withdrawal group participants talked of the disruptive treatment regimen and side effects, the time off work/school, and impacts on taking holidays. Decisions to withdraw from eculizumab were driven by the wish to lead more normal lives and concerns about long-term treatment. Drivers for declining withdrawal focused on relapse and its perceived impact. After 2 years, the withdrawal group had regained a semblance of normality, though fears about relapse remained, and they were aware of the need for long-term follow-up. Participants had a greater sense of control over the necessary steps should they/their child relapse.

Conclusion: An understanding of patient/parent experiences should guide discussions about eculizumab withdrawal. Although the availability of an alternative complement inhibitor reduces the frequency of infusions, most of the factors influencing withdrawal decisions remain unchanged. Support to alleviate fears in the early stages of withdrawal would be beneficial. Evidence from the main trial on successful withdrawal, and recovery time where eculizumab was reinstated, may provide reassurance to those who are uncertain about withdrawal.