Journal of Patient-Reported Outcomes最新文献

Background: PRO measures of symptoms in clinical trials have historically utilized visual and numerical scales but verbal descriptors may make it easier for patients to clearly differentiate between response options. This study assessed content validity and meaningful change in five verbal response scales (VRSs) used to assess chronic rhinosinusitis with nasal polyp (CRSwNP) symptom severity.

Methodology: This qualitative, semi-structured interview study recruited adults from the US, Germany, and China with confirmed moderate-to-severe CRSwNP. Interviews included a concept elicitation section, where participants were asked about their experience of living with CRSwNP including symptoms and health-related quality-of-life impacts, and a cognitive debriefing section, where participants were debriefed and participant understanding and real-life relevance of the CRSwNP symptom VRS content were assessed. Interview transcripts were qualitatively analyzed using thematic analysis methods.

Results: Among the 24 participants interviewed, the most frequently reported CRSwNP symptoms were nasal obstruction, runny nose, mucus in the throat, loss of smell and facial pain/pressure. Participants demonstrated good understanding of the CRSwNP symptom VRS instructions, items, recall period, and response options. The five CRSwNP symptom VRS items were relevant to the majority of participants' experience of CRSwNP. At the item level, a one-category within-person improvement was the level most frequently reported by participants to be a meaningful change.

Conclusion: The CRSwNP symptom VRSs assess relevant and bothersome symptoms experienced by patients with moderate-to-severe CRSwNP, supporting content validity of this measure. The findings of this study provided preliminary insights into meaningful change in the VRS. Further quantitative assessment of meaningful change is needed, and psychometric evaluation of the CRSwNP symptom VRSs will be required to evaluate their appropriateness for assessment of clinical trial endpoints in patients with CRSwNP.

Background: This psychometric analysis generated evidence to support the use of the Functional Assessment of Cancer Therapy item GP5 (GP5) as a measure of tolerability and confirms the appropriateness of categorizing "high side-effect burden" using a rating of 3 or 4 (score ranges 0-4) in patients with advanced/metastatic RET-mutant medullary thyroid cancer (MTC).

Methodology: Blinded, pooled interim data from the safety population (n=290) enrolled in the phase 3 LIBRETTO-531 trial (NCT04211337) were used. Intraclass correlation coefficients (ICC) were calculated for test-retest reliability using data from cycles 1-2 post-baseline. Construct validity was evaluated by examining the correlations of GP5 ratings with (a) symptomatic adverse events (AEs; measured by the PRO-CTCAE), and (b) functioning scores of EORTC QLQ-C30. The ability to detect change over time was examined by Cochrane-Mantel-Haenszel tests for GP5 ratings and PRO-CTCAE. The relationship of "high side-effect burden" categories with QLQ-C30 functioning scores was examined.

Results: ICCs for the GP5 ratings after cycle 1 ranged between 0.80 and 0.85, indicating good reliability. Correlations between GP5 and PRO-CTCAE items ranged from 0.18 to 0.62 and ranged from -0.37 to -0.50 for QLQ-C30 functioning scores, consistent with study assumptions. Post-baseline GP5 ratings showed significant associations with PRO-CTCAE scores (p<0.001). Participants with GP5 ratings of 3 or 4 had worse physical function than those with GP5 ratings of 0 to 2 (p<0.0001).

Conclusions: This analysis generated evidence supportive of the psychometric properties of the GP5 as a fit-for-purpose measure to assess treatment tolerability in patients with advanced/metastatic MTC. The definition of "high side-effect burden" was associated with the clinical feature of tolerability.

Background: Condition-specific health-related quality-of-life (HRQoL) instruments can be more responsive and sensitive to specific conditions and diseases than generic HRQoL instruments. This systematic review aims to identify the condition-specific preference-based instruments that have been used with young people and identify how preference values have been obtained for these instruments. This review will inform future researchers about the methods used to elicit utilities for condition-specific HRQoL instruments.

Methods: A comprehensive search strategy was used to identify condition-specific HRQoL instruments used in young people and the methods used to value these instruments. Published medical and health economic databases were searched from January 1990-March 2022. Articles were deemed eligible for inclusion if a condition-specific preference-based instrument was used in young people (age < 18). Screening, data extraction and quality assessment were conducted independently by at least two reviewers.

Results: After deduplication, a total of 4273 articles were eligible for title and abstract screening. Of these, 98 articles were eligible for full-text screening. After full-text screening, 18 articles were included in the review. Valuation studies were the most prevalent study design in the review (44%), followed by mapping studies (38%) and then other designs (18%). Among the valuation studies, the choice of HRQoL instrument, preference elicitation method, anchoring method and perspective varied considerably.

Conclusion: To our knowledge, this review is the first to explore what condition-specific HRQoL instruments have been used in young people. Findings from this review could inform researchers in their choice of methods for measuring and valuing HRQoL. This review illustrates that to date there does not appear to be clear consensus of how to measure and value HRQoL in young people when using condition-specific instruments. The lack of consensus could be influenced by challenges identified in prior research such as limited guidance, ethical issues, and uncertain normative decisions regarding the choice of preference elicitation method. Ordinal methods such as discrete choice experiment and best-worst scaling appear to be preferable for use in this population.

Background: Eptinezumab's impact on self-reported work productivity in adults with migraine and 2‒4 prior preventive migraine treatment failures is not fully understood.

Methodology: Electronic diaries captured monthly migraine days (MMDs) reported by patients enrolled in the randomized, double-blind, placebo-controlled DELIVER trial (NCT04418765). The migraine-specific Work Productivity and Activity Impairment questionnaire, administered at baseline and each monthly visit, was a secondary outcome of DELIVER and used to model changes from baseline in self-reported monthly hours of absenteeism (decreased work attendance) and presenteeism (reduced work efficiency while at work with a migraine) in Canada, as the base case. Path analysis illustrated eptinezumab's impact on work productivity beyond MMDs.

Results: As MMDs increased, monthly hours of absenteeism increased linearly while those of presenteeism increased quadratically. Best-fit models were improved after including an eptinezumab treatment effect, showing benefit over placebo after controlling for MMD frequency. Compared to placebo, patients treated with eptinezumab (pooled) had a modeled reduction from baseline of 7.2 h/month (absenteeism) (95% CI: -9.9, -4.5; P < 0.001) and 21.4 h/month (presenteeism) (95% CI: -26.3, -16.5; P < 0.001) over weeks 1‒24. Beyond MMD reductions, improvements in patient-identified most bothersome symptom (PI-MBS) and reductions in percent of severe migraine attacks contributed to eptinezumab's effect.

Conclusions: Eptinezumab decreased absenteeism and presenteeism based on patient reports, with data highlighting the importance of considering the PI-MBS. The greater change from baseline than placebo in self-reported absenteeism and presenteeism was only partly explained by changes in MMDs, presenting a potential opportunity to decrease the cost of migraine in the workplace.

Trial registration: ClinicalTrials.gov (Identifier: NCT04418765); EudraCT (Identifier: 2019-004497-25).

Background: Orthopaedic surgeon engagement with patient-reported outcome measures (PROMs) data has not been comprehensively evaluated, despite increasing uptake of orthopaedic PROMs programmes globally. The aim of this review was to systematically identify, appraise and synthesise qualitative evidence on barriers and facilitators to orthopaedic surgeons' engagement with PROMs data and their use of these data to support clinical practice.

Methods: Six databases (MEDLINE, EMBASE, COCHRANE CENTRAL, PSYCINFO, CINAHL and EMCARE) were searched from January 2000-March 2024 to identify potentially eligible qualitative studies. Established systematic review methods were used for screening and data extraction, applying PRISMA guidelines. Quality assessment was undertaken using the Joanna Briggs Institute tool for qualitative research.

Results: Eight studies were eligible for inclusion; of these, five studies were qualitative and three studies were mixed-method designs incorporating a qualitative component. Three studies were specific to orthopaedic surgeons and the remaining five studies comprised of mixed samples of health professionals including orthopaedic surgeons. Only one study was classified as being of high methodological quality. Key barrier themes for orthopaedic surgeons were logistical issues, difficulty interpreting and understanding PROMs, and scepticism of the value of PROMs in clinical care. Key enabler themes included improvements to PROMs infrastructure, surgeon education around the potential value, uses and interpretation of PROMs data, aggregate reporting of PROMs data and early involvement of surgeons in the planning and development of PROM systems.

Conclusion: While these studies highlight some practical considerations and opportunities that can be addressed through clinician education, there is little high-quality evidence on factors that influence orthopaedic surgeon engagement with PROMs data. Robust qualitative research is needed to better inform tailored support and assist surgeons in integrating PROMs data within orthopaedic care.

Background: Time to deterioration (TTD) endpoints are often utilized in the analysis of patient-reported outcome (PRO) data in oncology clinical trials but different endpoint definitions and analysis frameworks exist that can impact result interpretation. This review examined the analysis, reporting and heterogeneity of TTD endpoints in the literature, the impact of analysis methods on results, and provides recommendations for future trials.

Methods: A targeted literature review of articles published between 2017 and 2022 was performed to collate TTD endpoints reported in oncology randomized controlled trials (RCTs). Details of endpoints and results were extracted including; deterioration definition, PRO assessment schedule, methods for handling intercurrent events, statistical analysis methods, main trial results (overall survival and/or progression-free survival) and TTD endpoint results.

Results: Seventy RCTs were included covering 849 individual TTD endpoints. There were 17 primary cancer types, with lung (26%), breast (11%), and prostate (7%) cancers the most common. Most trials (71%) were for people with advanced cancer. Full definitions of TTD endpoints were often missing. There were no clear trends for a specific TTD definition within cancer types or stages. However, statistical analysis methods were consistent among trials.

Conclusion: The TTD definition can vary and is ultimately driven by the research question. Points to consider for successfully implementing PRO TTD endpoints in oncology include consideration of the trial setting (e.g., early vs. advanced cancer), expected treatment effect (e.g., improvement vs. worsening), likely adverse event profile (including early vs. delayed) and PRO data collection frequency in order to improve utility of these endpoints.

Background: To facilitate the maintenance or resumption of participation in work for patients with cardiovascular disease (CVD), there is a need for high-quality work-focused healthcare. According to the concept of value-based healthcare, quality of care can be enhanced by understanding the outcomes that matter most to patients. However, a major challenge in assessing quality of work-focused healthcare in practice is the lack of consensus on which work-related outcomes should be measured.

Objective: The objective of this study was to identify a standard set of key work-related outcomes for patients with CVD to be used in practice of work-focused healthcare in the Netherlands, including standardised outcome measures and associated case mix factors. This standard set is intended to assist occupational and other health professionals in delivering work-focused healthcare that meets a patient's individual needs regarding work participation, and to enhance patients' engagement in their own work-focused care process.

Methods: A 2-round RAND-modified Delphi process was conducted. The process included literature searches, consecutive research team meetings, and several meetings and rounds of voting by a working group. The working group consisted of patients with CVD (n = 6) and health professionals representing different stakeholders (n = 11) involved in work-focused healthcare for this patient population in the Netherlands. Consensus was reached over four phases: (1) establishing the scope of the standard set and defining the population, (2) prioritising and defining the outcome domains, (3) selecting the outcome measures for the most important domains, including clinical data and patient-reported data, and (4) selecting and defining case mix factors.

Results: A 23-item patient-reported questionnaire was developed, called the Value@WORK-Q23, including questions on nine work-related outcome domains considered most important for patients with CVD: (1) work participation, (2) physical work ability, (3) mental work ability, (4) suitable work, (5) support from the work environment, (6) flexibility of the work environment, (7) communication with the patient, (8) person-centredness, and (9) interdisciplinary communication. In addition, nine case mix variables was selected, comprising demographic-, disease-, and work factors.

Conclusions: The Value@WORK-Q23 provides guidance on measuring the most important work-related outcomes for patients with CVD. Using this work-related set in practice, in addition to existing disease-specific standard sets for CVD may facilitate the provision of high-value work-focused healthcare for this patient population.

Background: Robust and well-defined data collection is important when using electronic patient-reported outcome measures (ePROMs) in clinical studies. Questions have been raised as to whether older age may be a barrier to data collection due to patients' unfamiliarity with electronic devices. Older adults may also have underlying health conditions that affect their ability to fill out patient-reported outcome measures (PROMs) on electronic devices. The aim of this observational, qualitative research study was to evaluate the usability of electronic PROMs (ePROMs) on a tablet and smartphone in older participants with and without bullous pemphigoid (BP).

Methods: Older people with and without BP were recruited in the US and France. They participated in 60-min in-person interviews, with moderators observing their completion of various tasks, including ePROMs, using a tablet and smartphone. Participants were scored on ease of task completion using a scale from 1 to 5.

Results: A total of 12 participants were recruited (≥65 years old; six each with and without BP [all participants without BP were ≥75 years old]). Most participants (83%) could easily and confidently perform most assigned tasks on both the tablet and smartphone. Although select tasks required assistance, all participants were eventually able to complete all tasks. Overall, ePROM usability did not correlate with age, sex, country, or disease state. Feedback on the general usability of both electronic devices was largely positive, and most participants (n = 11; 92%) were willing to use them. Participants were generally pleased with the training modules offered on both devices, describing the training as sufficient, straightforward, and helpful. In total, 25 usability issues were identified, which fell into three categories: incomplete instructions, unclear language, and insufficient technical/visual design. Participants provided feedback on how to enhance device usability.

Conclusions: The results suggest that older people can confidently use a tablet or smartphone for ePROM completion, particularly with appropriate training. ePROMs should be designed with the needs of the target patient population in mind. These results can be leveraged to improve clinical data recording, optimize device usability, and enhance the user experience for older people and those with functional or physical limitations.

Background: Alemtuzumab is a humanized monoclonal antibody approved for the treatment of relapsing-remitting multiple sclerosis (RRMS). Its efficacy and safety have been widely demonstrated in clinical trials, but experience from real-world cohorts is also needed to support its clinical use. Quality of life (QoL) outcomes are an important complement to the clinical benefits of treatment, offering a patient-centered perspective on how the drug contributes to general well-being. In this line we aimed to evaluate the QoL of patients treated with alemtuzumab in clinical practice.

Methods: This prospective 3-year multicenter study was carried out in adult patients diagnosed with RRMS who had started alemtuzumab according to clinical practice within 8 weeks before inclusion. The primary endpoint was the change in QoL over three years of treatment with alemtuzumab using the 29-item Multiple Sclerosis Impact Scale (MSIS-29). Secondary endpoints included changes from baseline in the 21-item Modified Fatigue Impact Scale (MFIS-21), Beck Depression Inventory (BDI-II), Symbol Digit Modalities Test (SDMT, oral version) and Work Productivity. Disability worsening was also assessed based on the Expanded Disability Status Scale (EDSS), along with the annualized relapse rate (ARR) and radiological activity.

Results: A cohort of 165 patients was analyzed (mean age 38.6 years, mean disease duration 8.5 years, mean EDSS score 3.3). MSIS-29 physical domain scores decreased significantly from baseline by a mean of 7.2 ± 1.8 points at year 1, 6.4 ± 2.2 at year 2 and 5.6 ± 2.3 at year 3 (p < 0.05 in all cases). Similarly, MSIS-29 psychological domain scores decreased significantly by a mean of 7.9 ± 2.4 points at year 1, 12.8 ± 2.9 at year 2 and 13.2 ± 3.0 at year 3 (p < 0.05 in all cases). Significant reductions from baseline were also evidenced in MFIS-21 and BDI-II scores, while SDMT scores remained unchanged. During the 3 years on alemtuzumab, the ARR was 0.15, representing an 83% reduction from the 2 years before initiation. At 3 years, 81.5% of patients were free from radiological activity and 87% were free from disability worsening.

Conclusions: These results indicate early and substantial improvements in patients' perception of their QoL and functioning with alemtuzumab that were sustained over three years.

Objective: Translating the newly developed Empowerment Audiology Questionnaire, EmpAQ-15 to Swedish, and performing content validation on the Swedish version.

Design: Best-practice principles using forward and back translations which were revised by a committee prior to field testing. Field testing was conducted by cognitive interviews with hearing-aid users talking through and rating the items in the translated questionnaire. Content validation was assessed by examining equivalence, accessibility, acceptability, comprehensiveness, and relevance of interview data. Questionnaire introduction and scoring instructions were evaluated by Swedish audiologists.

Study sample: Ten adult native speaking Swedish hearing aid users, recruited with purposive sampling. Maximum variation based on age, gender, hearing aid usage, and degree of hearing loss. Seven Swedish audiologists assessing instructions for result calculations.

Results: The conceptual equivalence between the Swedish translation and the English original questionnaire was judged to be high overall. The instructions and majority of items were experienced as accessible, acceptable, comprehensive, and relevant. The audiologists showed that they could follow scoring instructions and reason about the results.

Conclusions: This content validity study was the first step towards a Swedish version of a self- report measure of Empowerment for people with hearing loss.