Patient-Patient Centered Outcomes Research最新文献

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.

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.