Patient-Patient Centered Outcomes Research最新文献

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.

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.