Validated patient reported outcome measures in speech-language pathology: A scoping review of adult practice.

PURPOSE: Patient reported outcome measures (PROMs) are increasingly used to inform value-based healthcare. Within speech-language pathology (SLP), there is no synthesis of validated PROMs to guide professional practice. This scoping review systematically identifies and evaluates condition-specific PROMs across adult SLP practice. METHOD: A literature search was performed to identify studies published until 18th February 2022 from MEDLINE, Embase, CINAHL, PsycInfo, Scopus, Cochrane Collaboration, and Web of Science. Abstracts and full texts were screened in Covidence. Relevant studies that validated PROMs in English were extracted and assessed using the "Checklist to operationalise measurement characteristics of PROMs" by two independent reviewers. RESULT: Ninety-seven articles provided validation data for 71 PROMS across seven SLP practice areas. These included voice (n = 18), swallowing (n = 14), language (n = 11), fluency (n = 8), speech (n = 4), laryngectomy (n = 3), and cognitive-communication (n = 2). No PROMs were identified for augmentative and alternative communication (AAC) (n = 0). Quality ratings were variable on the Francis et al. checklist. CONCLUSION: A range of validated PROMs were identified to guide SLP practice in measuring patient perceptions across a range of practice areas in adults. Opportunities for further development in SLP practice areas with limited PROMs, such as speech, cognitive-communication, and AAC are also highlighted.

A Collection of Components to Design Clinical Dashboards Incorporating Patient-Reported Outcome Measures: Qualitative Study.

BACKGROUND: A clinical dashboard is a data-driven clinical decision support tool visualizing multiple key performance indicators in a single report while minimizing time and effort for data gathering. Studies have shown that including patient-reported outcome measures (PROMs) in clinical dashboards supports the clinician's understanding of how treatments impact patients' health status, helps identify changes in health-related quality of life at an early stage, and strengthens patient-physician communication. OBJECTIVE: This study aims to determine design components for clinical dashboards incorporating PROMs to inform software producers and users (ie, physicians). METHODS: We conducted interviews with software producers and users to test preselected design components. Furthermore, the interviews allowed us to derive additional components that are not outlined in existing literature. Finally, we used inductive and deductive coding to derive a guide on which design components need to be considered when building a clinical dashboard incorporating PROMs. RESULTS: A total of 25 design components were identified, of which 16 were already surfaced during the literature search. Furthermore, 9 additional components were derived inductively during our interviews. The design components are clustered in a generic dashboard, PROM-related, adjacent information, and requirements for adoption components. Both software producers and users agreed on the primary purpose of a clinical dashboard incorporating PROMs to enhance patient communication in outpatient settings. Dashboard benefits include enhanced data visualization and improved workflow efficiency, while interoperability and data collection were named as adoption challenges. Consistency in dashboard design components is preferred across different episodes of care, with adaptations only for disease-specific PROMs. CONCLUSIONS: Clinical dashboards have the potential to facilitate informed treatment decisions if certain design components are followed. This study establishes a comprehensive framework of design components to guide the development of effective clinical dashboards incorporating PROMs in health care practice.

Truth and dare: patients dare to tell the truth when using PROMs in clinical practice.

PURPOSE: As patient-reported outcome measures (PROMs) are increasingly used in clinical practice for screening, monitoring, and management, the potential for response bias has been raised (e.g., over-reporting problems for attention, under-reporting to avoid treatment changes/discontinuation). We investigated whether patients systematically bias their responses when they know clinicians will review their PROM results. METHODS: We conducted secondary analyses of three experimental studies evaluating PROMs in adult and pediatric care. Prior to PROM completion, intervention group patients were informed that the results would be shown to their clinicians ("feedback" arm), whereas control group patients were told that their clinicians would not see their responses ("no feedback" arm). Independent sample t-tests compared the "feedback" and "no feedback" arms' PROM scores at baseline. Effect sizes and 95% confidence intervals were estimated using Cohen's d statistics with Hedges' g correction, and effect sizes > 0.50 were considered clinically relevant. RESULTS: Across the 29 domains assessed in the three studies, no between-arm differences reached an effect size of ± 0.50. Only 3/29 effect sizes exceeded ± 0.30. The confidence intervals for 14 domains included ± 0.50, with 4 favoring the "no feedback" arm and 10 favoring the "feedback" arm. Two domains reached statistical significance, one favoring the "no feedback" arm and one favoring the "feedback" arm. CONCLUSION: This study does not support the hypothesis that patients systematically bias their PROM responses if they know that clinicians will see their results. These findings support using PROMs in clinical practice as a valid mechanism to promote patient-centered care.

Patient-Reported Outcome Measures for Pelvic Organ Prolapse: A Systematic Review Using the COnsensus-Based Standards for the Selection of Health Measurement Instruments (COSMIN) Checklist.

BACKGROUND: Patient-reported outcome measures (PROMs) are recommended to measure the impact of a health condition or intervention effectiveness as they aim to capture what is most meaningful to patients. Several PROMs are used to evaluate pelvic organ prolapse (POP)-related domains, yet the measurement properties of these instruments have not been fully explored with a rigorous analysis of the methodological quality and quality of evidence. OBJECTIVE: To conduct a systematic review reporting on the measurement properties of PROMs used for the assessment of POP-related domains in accordance with the COSMIN guidelines. SEARCH STRATEGY: Five databases were searched from inception to December 2023. SELECTION CRITERIA: Studies were eligible if they involved (1) at least one group of female adults diagnosed with or presenting with symptoms of POP; (2) a self-reported outcome measure (PROMs, questionnaires) to evaluate POP-related domains; and (3) at least one measurement property. DATA COLLECTION AND ANALYSIS: Methodological quality and measurement quality were assessed using the COSMIN risk of bias (ROB) checklist and the COSMIN criteria for good measurement properties. MAIN RESULTS: A total of 13 PROMs were included. The BIPOP had the lowest ROB for Content Validity. The POP-SS was the only PROM with sufficient evidence of adequate construct validity and responsiveness to be used in both surgical and conservative management settings. CONCLUSION: This original work identified a gap in evidence regarding the measurement qualities of identified PROMs used in the POP population.

Quality of life and psychological distress in end-stage renal disease patients undergoing hemodialysis and transplantation.

BACKGROUND: Among diverse profound impacts on patients' quality of life (QoL), end-stage renal disease (ESRD) frequently results in increased levels of depression, anxiety, and stress. Renal replacement therapies such as hemodialysis (HD) and transplantation (TX) are intended to enhance QoL, although their ability to alleviate psychological distress remains uncertain. This research posits the existence of a significant correlation between negative emotional states and QoL among ESRD patients, with varying effects observed in HD and TX patients. AIM: To examine the relationship between QoL and negative emotional states (depression, anxiety, and stress) and predicted QoL in various end-stage renal replacement therapy patients with ESRD. METHODS: This cross-sectional study included HD or TX patients in the Eastern Region of Saudi Arabia. The 36-item Short Form Survey and Depression Anxiety Stress Scale (DASS) was used for data collection, and correlation and regression analyses were performed. RESULTS: The HD and TX transplantation groups showed statistically significant inverse relationships between QoL and DASS scores. HD patients with high anxiety levels and less education scored low on the physical component summary (PCS). In addition, the results of the mental component summary (MCS) were associated with reduced depression. Compared with older transplant patients, TX patients' PCS scores were lower, and depression, stress, and negative working conditions were highly correlated with MCS scores. CONCLUSION: The findings of this study revealed notable connections between well-being and mental turmoil experienced by individuals undergoing HD and TX. The PCS of HD patients is affected by heightened levels of anxiety and lower educational attainment, while the MCS of transplant patients is influenced by advancing age and elevated stress levels. These insights will contribute to a more comprehensive understanding of patient support.

Advancing patient-centered cancer care: a systematic review of electronic patient-reported outcome measures.

Background: Electronic Patient-Reported Outcome Measures (ePROMs) have emerged as valuable tools in cancer care, facilitating the comprehensive assessment of patients' physical, psychological, and social well-being. This study synthesizes literature on the utilization of ePROMs in oncology, highlighting the diverse array of measurement instruments and questionnaires employed in cancer patient assessments. By comprehensively analyzing existing research, this study provides insights into the landscape of ePROMs, informs future research directions, and aims to optimize patient-centred oncology care through the strategic integration of ePROMs into clinical practice. Methods: A systematic review was conducted by searching peer-reviewed articles published in academic journals without time limitations up to 2024. The search was performed across multiple electronic databases, including PubMed, Scopus, and Web of Science, using predefined search terms related to cancer, measurement instruments, and patient assessment. The selected articles underwent a rigorous quality assessment using the Mixed Methods Appraisal Tool (MMAT). Results: The review of 85 studies revealed a diverse range of measurement instruments and questionnaires utilized in cancer patient assessments. Prominent instruments such as the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) and the Patient Reported Outcome-Common Terminology Criteria for Adverse Events (PRO-CTCAE) were frequently referenced across multiple studies. Additionally, other instruments identified included generic health-related quality of life measures and disease-specific assessments tailored to particular cancer types. The findings indicated the importance of utilizing a variety of measurement tools to comprehensively assess the multifaceted needs and experiences of cancer patients. Conclusion: Our systematic review provides a comprehensive examination of the varied tools and ePROMs employed in cancer care, accentuating the perpetual requirement for development and validation. Prominent instruments like the EORTC QLQ-C30 and PRO-CTCAE are underscored, emphasizing the necessity for a thorough assessment to meet the multifaceted needs of patients. Looking ahead, scholarly endeavours should prioritize the enhancement of existing tools and the creation of novel measures to adeptly address the evolving demands of cancer patients across heterogeneous settings and populations.

The evaluation of daily activity questionnaire for stroke survivors: cross-cultural adaptation, content validity and digitisation.

OBJECTIVE: The study's objective was to culturally adapt and digitise the Evaluation of Daily Activity Questionnaire (EDAQ), originally designed for rheumatoid arthritis and various other musculoskeletal conditions, creating a version for stroke survivors (EDAQ-SS). This adaptation also aimed to develop a comprehensive electronic Patient Reported Outcome Measure (ePROM) intended to refine stroke survivors' self-assessment of their daily activity limitations. MATERIALS AND METHODS: Cross-cultural adaptation of the EDAQ was completed by a review of expert panel, which included healthcare professionals to increase the clarity and relevancy of the items, followed by cognitive debriefing interviews with British stroke survivors to rate their understanding of the questionnaire items. After developing the paper version of the questionnaire, this was digitised (eEDAQ-SS) and disseminated online via the Stroke Survivors Hub (SSHUB). Content validity of the EDAQ-SS was evaluated using the International Classification of Functioning (ICF) Core Set for Stroke. RESULTS: The expert panel meeting (n:11) and cognitive debriefing interviews with stroke survivors (n:10) resulted in an EDAQ-SS with 160 items across 15 domains, which was understandable and relevant to stroke survivors. The SSHUB was deemed to be a user-friendly platform, providing easy access to eEDAQ-SS and aid self-assessment of daily activities of stroke survivors. Mapping the EDAQ-SS items to the ICF Core Set for Stroke demonstrated good content validity with 44/55 matching categories. CONCLUSION: The EDAQ-SS offers a comprehensive measure for self-assessment, which may serve to guide stroke survivors' self-management by overcoming limitations of existing PROMs. Further psychometric testing of the EDAQ-SS and wider testing of the digital version is recommended. CONTRIBUTION OF THE PAPER.

A retrospective study on patient satisfaction and Oral Health-Related Quality of Life with fixed 4- or 6-implant supported prostheses over 3-7 years.

OBJECTIVES: To compare patients' satisfaction and oral health-related quality of life (OHRQoL) in edentulous patients treated by All-on-4 and All-on-6 implant-supported fixed prostheses rehabilitation over 3-7 years. METHODS: All patients (n = 125) were divided into short-term (3-5 years, n = 71) and long-term (5-7 years, n = 54) follow-up groups. Patients' satisfaction was examined in a cross-sectional survey. OHRQoL and psychological impact were evaluated using the Oral Health Impact Profile (OHIP). Comparison of All-on-4 and All-on-6, including OHIP values, satisfaction scores, treatment duration and complications, were analyzed using the Chi-square test and the Mann-Whitney U test. Potential influence of patient-related factors on patient-reported outcome measures (PROMs) were analyzed via the Mann-Whitney U test, the Kruskal-Wallis test and multiple regression analyses. Power calculation was conducted in R Studio. RESULTS: Both All-on-4 and All-on-6 follow-up groups showed high scores in total satisfaction and PROMs, including chewing comfort, aesthetics, cleanability, and stability, with no significant difference (p > 0.05). Meanwhile, no significant differences were observed in the OHIP scores between two groups in both follow-up durations (p > 0.05). However, in patients with 3-5 years follow-up, All-on-4 group had significantly lower phonetic satisfaction scores than All-on-6 group (p < 0.05). Additionally, during both follow-up periods, All-on-4 patients exhibited notably superior psychological effect in contrast to All-on-6 patients (p < 0.05). CONCLUSIONS: Edentulous patients treated by four or six implant-supported fixed prostheses showed same high satisfaction in oral health-related quality of life in both 3-5 years and 5-7 years follow-ups.

The impact of NHS outsourcing of elective care to the independent sector on outcomes for patients, healthcare professionals and the United Kingdom health care system: A rapid narrative review of literature.

The NHS is increasingly turning to the independent sector, primarily to alleviate elective care backlogs. However, implications for the healthcare system, patients and staff are not well understood. This paper provides a rapid narrative review of research evidence on NHS-funded elective care in the independent sector (IS) and the impact on patients, professionals, and the health care system. The aim was to identify the volume and evaluate the quality of the literature whilst providing a narrative synthesis. Studies were identified through Medline, CINAHL, Econlit, PubMed, Web of Science and Scopus. The quality of the included studies was assessed in relation to study design, sample size, relevance, methodology and methodological strength, outcomes and outcome reporting, and risk of bias. Our review included 40 studies of mixed quality. Many studies used quantitative data to analyse outcome trends across and between sectors. Independent sector providers (ISPs) can provide high-volume and low-complexity elective care of equivalent quality to the NHS, whilst reducing waiting times in certain contexts. However it is clear that the provision of NHS-funded elective care in the IS has a range of implications for public provision. These surround access and outcome inequalities, financial sustainability and NHS workforce impacts. It will subsequently be important for future empirical work to incorporate these caveats, providing a more nuanced interpretation of quantitative improvements.

Translation and Validation of Dizziness Handicap Inventory in Hindi Language.

The dizziness handicap inventory (DHI) is a widely used questionnaire for assessing the impact of dizziness on daily life. There is a need for validated translations of the DHI to accommodate diverse linguistic populations, enabling clinical and research comparisons. This study aimed to translate and validate the DHI into Hindi language. The DHI was translated into Hindi following established guidelines. The translated version underwent validation among Hindi-speaking patients presenting with dizziness at an outpatient ENT clinic in this cross-sectional study. Reliability and validity analyses were conducted, including Cronbach's alpha, split-half reliability, and factor analysis. A total of 153 participants completed the DHI-Hindi, with a mean score of 37.28 (SD = 15.63). Ceiling and floor effects were absent. The Cronbach's alpha coefficient for the overall scale was 0.785, indicating good reliability. Factor analysis revealed a three-factor solution, consistent with previous studies, indicating good construct validity.The translation and validation process yielded a reliable and valid version of the DHI in Hindi language, suitable for assessing the impact of dizziness in Hindi-speaking population.

Health-related quality of life among women and men living with migraine: a Canada-wide cross-sectional study.

BACKGROUND: Migraine is a prevalent neurologic disorder that affects women more than men. Examining health-related quality of life (HRQoL) by gender can aid decision makers in prioritizing future treatment and prevention programs. We aimed to quantify HRQoL by different levels of migraine disability and by gender. METHODS: As part of a Canada-wide cross-sectional study, we administered an online survey to employed adults who self-reported a diagnosis of migraine. Migraine disability level was assessed using the Migraine Disability Assessment questionnaire (MIDAS). MIDAS scores were used to categorize respondents as having little to no, mild, moderate, or severe level of migraine-related disability. Physical and mental component summary scores (PCS and MCS) and health utilities were derived from responses to the Veterans Rand 12 Item Health Survey. PCS, MCS, and health utilities were summarized by migraine-related disability levels and gender. Covariate-adjusted linear regressions were used to examine the association between migraine disability level and health utility by gender. RESULTS: A total of 441 participants completed the survey. The sample was predominantly women (60.1%), White race (75.5%), and had a mean age of 37 years. Mean health utility, PCS, and MCS scores were 0.61 (0.22), 45.0 (7.7), and 43.4 (11.0), respectively. All three scores decreased with increased migraine disability level. Gender differences on HRQoL within each migraine disability level were not statistically significant, except in the little to no disability level where women had lower mean MCS scores and health utility relative to men [mean (SD) MCS: women 44.0 (11.3); men 55.1 (8.1), p < 0.001; health utility: women 0.66 (0.18); men 0.81 (0.18), p < 0.001]. Linear regressions showed women with severe migraine-related disability had reduced health utility compared to women with little to no disability [adjusted difference: -0.16 (95%CI -0.24,-0.09)]. Associations among men increased in magnitude with migraine disability level [adjusted differences: mild - 0.16 (95%CI -0.24,-0.09); moderate - 0.18 (95%CI -0.26,-0.10); severe - 0.28 (95%CI -0.37,-0.20)]. CONCLUSIONS: Findings contribute to the literature on the association between migraine disability level and HRQoL by examining trends by gender. Model results emphasize the importance of future treatments reducing severe disability due to migraine among both women and men.

Forefoot inflammation in recent-onset ACPA-positive and ACPA-negative RA: clinically similar, but different in underlying inflamed tissues.

OBJECTIVES: Although joint swelling is traditionally interpreted as synovitis, recent imaging studies showed that there is also inflammation of tenosynovium and intermetatarsal bursae in the forefoot. We aimed to increase our understanding of differences and similarities regarding forefoot involvement between ACPA-positive and ACPA-negative rheumatoid arthritis (RA) at diagnosis. Therefore, we (1) compared metatarsophalangeal (MTP) joint counts, walking disabilities and inflamed tissues between ACPA groups and (2) studied associations of joint swelling/tenderness and walking disabilities with underlying inflamed tissues within ACPA groups. METHODS: 171 ACPA-positive and 203 ACPA-negative consecutively diagnosed patients with RA had a physical joint examination (swollen joint count-66/tender joint count-68), filled a Health Assessment Questionnaire including the domain walking and underwent MRI of the MTP joints at diagnosis. Synovitis, tenosynovitis, osteitis and intermetatarsal bursitis (IMB) were assessed. Findings in age-matched healthy controls were applied to define abnormalities on MRI. RESULTS: While ACPA-negative RA patients had more swollen joints (mean SJC 8 vs 6 in ACPA-positives, p=0.003), the number of swollen MTP joints was similar (mean 1 in both groups); walking disabilities were also equally common (49% vs 53%). In contrast, inflamed tissues were all more prevalent in ACPA-positive compared with ACPA-negative RA. Within ACPA-positive RA, IMB was associated independently with MTP-joint swelling (OR 2.6, 95% CI 1.4 to 5.0) and tenderness (OR 3.0, 95% CI 1.8 to 5.0). While in ACPA-negatives, synovitis was associated independently with MTP-joint swelling (OR 2.8, 95% CI 1.4 to 5.8) and tenderness (OR 2.5, 95% CI 1.3 to 4.8). Tenosynovitis contributed most to walking disabilities. CONCLUSIONS: Although the forefoot of ACPA-positives and ACPA-negatives share clinical similarities at diagnosis, there are differences in underlying inflamed tissues. This reinforces that ACPA-positive and ACPA-negative RA are different entities.

Development of a web-based ecological momentary assessment tool to measure day-to-day variability of the symptoms in patients with Sjögren's disease.

OBJECTIVES: To develop and validate a web-based ecological momentary assessment (EMA) tool to enhance symptoms monitoring among patients with Sjögren's disease (SjD). METHODS: Consecutive adults with SjD were enrolled in this pilot observational study. Participants used the WebApp over a 3-month period, for the daily collection of individual EULAR Sjögren's Syndrome Patient Reported Index (ESSPRI) scales and separate assessment of eyes and mouth dryness, using 0-10 numerical scales. Primary outcome was the measure of the interdaily variability of symptoms. Data collected through the WebApp were compared with those obtained with paper-based questionnaires administered during a final visit, using distinct approaches (predicted error, maximum negative error and maximum positive error). User experience was assessed using the System Usability Scale (SUS) score. RESULTS: Among the 45 participants, 41 (91.1%) were women. Median age was 57 years (IQR: 49-66). Daily variability of symptoms ranged between 0.5 and 0.8 points across the scales. Over the 3-month period, the predicted error ranged between -1.2 and -0.3 points of the numerical scales. The greatest differences were found for fatigue (-1.2 points (IQR: -2.3 to -0.2)) and ESSPRI score (-1.2 points (IQR: -1.7 to -0.3)). Over the last 2 weeks, the predicted error ranged between - 1.2 and 0.0 points. Maximum negative error ranged between -2.0 and -1.0 points, and maximum positive error between -0.3 and 0.0 points. Median SUS score was 90 (IQR: 85-95). CONCLUSION: Our results demonstrate the usability and the relevance of our web-based EMA tool for capturing data that closely reflects daily experiences of patients with SjD.

Implementing a Streamlined Hypoglossal Nerve Stimulator Pathway: Cost, Time to Surgery, and Outcomes.

Objective: To evaluate the costs, time to surgery, and clinical outcomes associated with implementing a streamlined hypoglossal nerve stimulator (HGNS) implantation pathway. Study Design: Retrospective cohort study. Setting: Single tertiary care center in the United States from 2016 to 2023. Methods: Patients with a lack of complete concentric collapse of the velum during volitional snore on in-office laryngoscopy qualified for the streamlined HGNS pathway. This pathway consisted of confirmatory drug-induced sleep endoscopy (DISE) followed immediately by HGNS implantation during the same surgical encounter. Outcomes were compared to patients in the traditional pathway (standalone DISE followed by HGNS implantation on a later date). Results: A total of 68 patients (13 streamlined, 55 traditional) with obstructive sleep apnea who underwent HGNS implantation were included. Patients were predominately male (70.6%) and White (95.6%) and had a mean (SD) age of 63.5 (10.0) years. The streamlined pathway was associated with a significant reduction in both hospital costs (mean difference $9258, 95% confidence interval [CI]: 3690-14,825; P = .002) and time to surgery (mean decrease of 3.82 months, 95% CI: 0.83-6.80 months; P = .013) compared to the traditional pathway. Patients in both groups had reduction in apnea-hypopnea index and Epworth Sleepiness Scale score, with no significant differences in comparisons between groups. Conclusion: In select patients, the streamlined HGNS pathway may expedite time to surgery and reduce hospital costs with comparable clinical outcomes to a traditional 2-stage pathway. Further research is warranted to validate patient selection and better understand longitudinal outcomes.

Positive experiences with the use of an eHealth smartphone application during the treatment of an abdominal aortic aneurysm.

OBJECTIVE: To evaluate the patient perceived satisfaction and feasibility of a personalized eHealth application (app) for abdominal aortic aneurysm (AAA) patients undergoing surgery. METHODS: Patients were offered to download the app prior to undergoing AAA surgery, in a prospective single centre cohort study, using a mixed methods sequential explanatory design. It offers information via the timely delivery of push notifications with text, images, and videos. The information includes chapters regarding the AAA, surgical techniques (endovascular aneurysm repair and open surgical repair), and perioperative lifestyle advice such as; physical exercise programmes, healthy and protein rich diet, geriatric care, and to stop smoking or drinking alcohol. RESULTS: The app was installed by 59/65 patients (91%). After installation, six patients deactivated the app (10%). The mean age was 74 years (SD = 7) and 85% of patients were male. The app was opened a median of 67 times (interquartile range [IQR] 33-127) and with a median time interval of 50 hours (IQR 28-74). Overall, 90% (53/59) completed a satisfaction questionnaire. On a numeric rating scale from 0 to 10, the median scored satisfaction for guidance was 8 (IQR 6-8), provided information was 8 (IQR 6-8), usefulness was 7 (IQR 6-8.5), and for recommending it to others 8 (IQR 6-9). Using purposeful sampling, seven patients underwent a semi-structured interview on user-experience of the app. They described experiencing positive changes to lifestyle habits, and appreciating the ability to share it with loved ones or informal caregivers. Several areas of improvement were reported. CONCLUSION: The personalized eHealth app is feasible in older AAA patients and valued as a useful supplement to the standard of care. We argue that the app aids in managing a prehabilitation programme, aids in the digital transformation of healthcare, and thereby decreases the workload of hospital staff.

Health-related Quality of Life Assessment in Renal Cell Cancer: A Scoping Review.

BACKGROUND AND OBJECTIVE: In oncology, patient-reported outcome measures (PROMs) capturing health-related quality of life (HRQOL) play an increasing role in clinical trials, drug approval, and policy making. This scoping review aimed to identify and elaborate on HRQOL-focussed PROMs used in renal cell cancer (RCC) clinical trials. METHODS: MEDLINE, Web of Science, PsychINFO, Academic Search Elite, CINAHL, Embase, and the Cochrane Library were searched systematically for original peer-reviewed articles on clinical trials including RCC patients and using PROMs, published between 1950 and 2023. Prespecified trial characteristics and information on the PROMs used were extracted. Frequencies and proportions of categorical data, and ranges and medians of continuous variables were calculated. KEY FINDINGS AND LIMITATIONS: Of the 48 unique studies included, the majority followed a randomised controlled design (34, 71%) and evaluated systemic treatments (38, 79%). The trials used 27 different PROMs (max = 6, median = 2), of which only 4 (15%) were developed specifically for kidney cancer patients. Of the trials, 46% did not use any RCC-specific PROM. European Quality of Life-5 Dimensions (EQ-5D), European Organisation for Research and Treatment of Cancer Quality of Life Core Questionnaire (EORTC QLQ-C30), Functional Assessment of Cancer Therapy Kidney Symptom Index (FKSI) -15/19-item version, FKSI-Disease Related Symptoms, and Functional Assessment of Cancer Therapy-General (FACT-G) were the most frequently used questionnaires, with pain, ability to work, fatigue, worry, and sleep quality being the most commonly assessed issues. CONCLUSIONS AND CLINICAL IMPLICATIONS: A variety of PROMs are used in RCC patients, hindering interpretability across trials. The PROMs used differ in terms of both the domains assessed and how the issues are translated into questionnaire items. Though RCC-specific PROMs exist, these have flaws in terms of relevance to patients. To answer predefined relevant HRQOL research questions, revised RCC-specific PROMs and standardisation of their integration into clinical trials are warranted. PATIENT SUMMARY: Researchers are more and more interested in the health-related quality of life of kidney cancer patients and use questionnaires to measure it. This review shows that there are many different health-related quality of life questionnaires that are used in different combinations in clinical trials for kidney cancer patients. This makes it very difficult to compare these study results and draw reliable conclusions for the actual clinical treatment. It was even found that some of the questionnaires used do not capture things that patients actually consider important (eg, emotional issues such as dealing with thoughts about cancer and depression). Therefore, more work needs to be done to develop questionnaires that ask what is really important to kidney cancer patients' health-related quality of life. If these questionnaires are used in a consistent way in clinical trials, the results can be better compared. This will help treat kidney cancer patients in the best possible way.

Longitudinal assessment of health-related quality of life in Japanese patients with advanced urothelial carcinoma receiving immune check point inhibitors.

Real-world data on health-related quality of life (HRQoL) in advanced urothelial carcinoma (aUC) receiving immune checkpoint inhibitors (ICIs) are limited. This study included 42 patients with aUC who received second-line or later pembrolizumab (n = 19), maintenance avelumab followed by first-line chemotherapy (n = 13), or adjuvant nivolumab after radical surgery (n = 10). Time-course changes in the domains and scales related to HRQoL were evaluated using the EORTC QLQ-C30, FACT-G, and SF-8 questionnaires during ICI therapy. Anchor-based approaches for minimally important differences were determined as 'improved', 'stable', and 'deteriorated'. We found significant improvements after the start of pembrolizumab treatment on many scales. Almost none of the scales changed significantly in the avelumab and nivolumab groups. Approximately 80% of the pembrolizumab group had deteriorated social/family well-being in FACT-G. Approximately 60% of the patients in the avelumab group had deteriorated general health and vitality in SF-8. In the nivolumab group, none of the scales deteriorated in > 50% of the patients. Deterioration of physical function in the SF-8 was associated with occurrence of treatment-related adverse events ≥ grade 2 during ICI therapy (P = 0.013). Our findings demonstrated that majority of patients with aUC who received ICI therapy had a stable HRQoL, which was consistent with evidence from clinical trials.

Reporting of patient-reported outcomes amongst randomized clinical trials in plastic surgery: a systematic review using CONSORT-PRO.

BACKGROUND: Patient-reported outcomes (PROs) are key to investigating patient perspectives in randomized controlled trials (RCTs). Standardization of PRO reporting is critical for trial generalizability and the application of findings to clinical practice. This systematic review aimed to evaluate the reporting quality of RCTs published in the top plastic surgery journals according to the consolidated standards of reporting trials (CONSORT)-PRO extension. METHODS: We completed a comprehensive search of MEDLINE, Embase, and Cochrane Central Register of Controlled Trials. All RCTs with a validated PRO endpoint published in the top 10 plastic surgery journals (based on the 2021 Web of Science Impact Factor) from 2014 to 2023 were included. Two reviewers independently extracted data and scored the included studies using the CONSORT-PRO checklist. Univariate regression was applied to assess factors associated with reporting adherence. Studies were assessed for their risk of bias using the Cochrane Risk of Bias 2.0 tool. RESULTS: A total of 88 RCTs were included. PROs were the primary endpoint in 50 (57%) and the secondary endpoint of 38 (43%) studies. Mean overall reporting adherence was poor (39% (±12) and 36% (±13) in studies with PRO as primary and secondary endpoints, respectively). The presence of industry support was significantly associated with greater adherence. CONCLUSIONS: There is low adherence to the CONSORT-PRO extension among plastic surgery RCTs published in the top 10 plastic surgery journals. We encourage journals and authors to endorse and apply the CONSORT-PRO extension. This may optimize the dissemination of clinical findings from RCTs and assist patient-centered care.

Agreement between patient-reported and clinician-rated speech and swallowing outcomes - Understanding the trend in post-operative oral cavity cancer patients.

AIM: To find the agreement between clinician-rated and patient-reported speech and swallowing outcomes in post-operative oral cavity cancer patients. METHODS: In this prospective observational study, a total of 53 post-operative oral cavity cancer patients were recruited. The Speech Handicap Index - Kannada (SHI-K) and the Dysphagia Handicap Index - Kannada (DHI-K) were used as the patient-reported outcome measures (PROMs), and the Mann Assessment of Swallowing Ability-Cancer (MASA-C) and Ali Yavar Jung National Institute of Speech & Hearing Disabilities (DIVYANGJAN) AYJNISHD(D)'s speech intelligibility rating scale were used as the clinician-rated scales to evaluate speech and swallowing status. RESULTS: Intraclass correlation coefficient (ICC) was poor, with a value of 0.480 between clinician-rated speech AYJNISHD(D)'s scale and patient-reported SHI-K scale. ICC was poor, with a value of 0.471 between clinician-rated swallowing MASA-C and patient-reported swallowing DHI-K. CONCLUSION: In our study, there was no agreement between patient-reported and clinician-rated speech and swallowing outcomes in post-operative oral cavity cancer patients. Incorporating PROMs into routine clinical practice is advisable, and clinicians need to balance PROMs with clinical and instrumental speech and swallowing assessments to ensure comprehensive care.

The Role of Biomarkers in Predicting Outcomes of Anterior Cruciate Ligament Reconstruction: A Systematic Review.

Background: Anterior cruciate ligament (ACL) injury is frequently associated with injuries to other parts of the knee, including the menisci and articular cartilage. After ACL injury and reconstruction, there may be progressive chondral degradation. Biomarkers in blood, urine, and synovial fluid can be measured after ACL injury and reconstruction and have been proposed as a means of measuring the associated cellular changes occurring in the knee. Purpose: To systematically review the literature regarding biomarkers in urine, serum, or synovial fluid that have been associated with an outcome measure after ACL reconstruction. Study Design: Systematic review; Level of evidence, 3. Methods: This review was performed according to the PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines. The MEDLINE, Embase, CINAHL, and Web of Science databases were searched to identify studies published before September 2023 that reported on patients undergoing ACL reconstruction where a biomarker was measured and related to an outcome variable. Of 9360 results, 16 studies comprising 492 patients were included. Findings were reported as descriptive summaries synthesizing the available literature. Results: A total of 45 unique biomarkers or biomarker ratios were investigated (12 serum, 3 urine, and 38 synovial fluid; 8 biomarkers were measured from >1 source). Nineteen different outcome measures were identified, including the International Knee Documentation Committee Subjective Knee Form, Knee injury and Osteoarthritis Outcome Score, numeric pain scores, radiological outcomes (magnetic resonance imaging and radiography), rates of arthrofibrosis and cyclops lesions, and gait biomechanics. Across the included studies, 17 biomarkers were found to have a statistically significant association (P < .05) with an outcome variable. Serum interleukin 6 (s-IL-6), serum and synovial fluid matrix metalloproteinase-3 (s-MMP-3 and sf-MMP-3), urinary and synovial fluid C-terminal telopeptide of type 2 collagen (u-CTX-II and sf-CTX-II), and serum collagen type 2 cleavage product (s-C2C) showed promise in predicting outcomes after ACL reconstruction, specifically regarding patient-reported outcome measures (s-IL-6 and u-CTX-II), gait biomechanical parameters (s-IL-6, sf-MMP-3, s-MMP-3, and s-C2C), pain (s-IL-6 and u-CTX-II), and radiological osteoarthritis (ratio of u-CTX-II to serum procollagen 2 C-propeptide). Conclusion: The results highlight several biomarkers that have been associated with clinically important postoperative outcome measures and may warrant further research to understand if they can provide meaningful information in the clinical environment.