Use Of Patient-reported Outcome Measures Research Articles

The use of patient-reported outcome measures to improve patient-related outcomes – a systematic review

BackgroundPatient-reported outcome measures (PROMs) provide invaluable information on patients’ health outcomes and can be used to improve patient-related outcomes at the individual, organizational and policy levels. This systematic review aimed to a) identify contemporary applications and synthesize all evidence on the use of PROMs in these contexts and b) to determine characteristics of interventions associated with increased effectiveness.MethodsFive databases were searched for studies providing quantitative evidence of the impact of PROM interventions. Any study design was permitted. An overall benefit (worsening) in outcome was defined as a statistically significant improvement (deterioration) in either a PROM, patient-reported experience measure or clinical outcome. Study quality was assessed using the Effective Public Healthcare Panacea Project’s Quality Assessment Tool for Quantitative Studies. A narrative synthesis was conducted.ResultsSeventy-six studies of the 11,121 articles identified met the inclusion criteria. At the individual level, 10 (43%) of 23 studies that fed back PROMs to the patient or healthcare provider showed an improvement in outcome. This percentage increased in studies which used PROMs to monitor disease symptoms and linked these to care-pathways: 17 (68%) of 25 studies using this mechanism showed an improvement. Ten (71%) of 14 studies using PROMs to screen for disease found a benefit. The monitoring and screening approach was most effective using PROMs covering cancer-related, depression and gastro-intestinal symptoms. Three studies found that the mere collection of PROMs resulted in improved outcomes. Another three studies used PROMs in decision aids and found improved decision quality.At the organizational/policy level, none of the 4 studies that used PROMs for benchmarking found a benefit. The three studies that used PROMs for in-depth performance analyses and 1 study in a plan-do-study-act (PDCA) cycle found an improvement in outcome.Studies employing disease-specific PROMs tended to observe improved outcomes more often. There are concerns regarding the validity of findings, as studies varied from weak to moderate quality.ConclusionsThe use of PROMs at the individual level has matured considerably. Monitoring/screening applications seem promising particularly for diseases for which treatment algorithms rely on the experienced symptom burden by patients. Organizational/policy-level application is in its infancy, and performance evaluation via in-depth analyses and PDCA-cycles may be useful. The findings of this review may aid stakeholders in the development and implementation of PROM-interventions which truly impact patient outcomes.

Patient-Reported Outcome Measures in Routine Hematology Cancer Care

Background Hematological cancers have devastating effects on patients’ physical, emotional, and psychosocial health. There is growing evidence to support the use of patient-reported outcome measures (PROMs) through validated tools. Although PROMs are widely adopted in oncology, uptake in hematology remains limited in routine clinical care. Objective This review seeks to explore the utility of PROMs in routine hematology clinical practice and to understand the extent and type of evidence in relation to benefits of PROMs to patients. Methods The review was conducted in accordance with the Joanna Briggs Institute methodology for scoping reviews and the Preferred Reporting Items for Systematic reviews and Meta-Analysis scoping review model for organizing information. Results Ten thousand and seventy-one articles were identified, 110 full texts were reviewed, and 14 articles met the final inclusion criteria. More than 20 individual outcome measures were identified that fell into the following categories: accessibility and usability, self-efficacy, shared decision-making, and implementation. Conclusions Studies focused primarily on the choice of PROM, acceptability and usability, motivation for use, patient and healthcare professionals’ experience of using PROMs, the stated value of PROMs, and implementation advice. However, there are limited published studies supporting how PROMs can be adopted into routine care for people with hematological cancer. Implications for Practice There are various validated PROMs but limited research on how to meaningfully implement them to improve clinical and patient outcomes in the routine care of hematology patients.

Patient-reported outcome measures-use in diagnosing depression, anxiety, and stress

Patient-reported outcome measures (PROMs) is an umbrella term for various self-report instruments used to assess subjective health-related impressions and treatment success from the patient's perspective. In psychosomatic medicine, PROMs are often used to record subjective symptoms, psychosocial distress, and changes in health status, particularly in patients with comorbid (affective) disorders and frequent contact with physicians, but also in preventive health care and to monitor the effectiveness of treatment. In otolaryngology (ENT), self-report questionnaires (PROMs) are used, among other things, to assess the impact of hearing, speech, swallowing, and breathing disorders on patients' quality of life. Such scientifically validated tools make it possible to better understand the needs and priorities of patients and to integrate them into clinician-patient communication and treatment to promote individual, patient-centered care. Three dimensions for the assessment of subjective health-related experiences (depression, anxiety, stress) with corresponding instruments for the German-speaking area for use in clinical and research settings are presented in more detail, specifically for otolaryngology.

Factors that impact the implementation of patient reported outcomes in routine clinical care for peripheral artery disease from the patient perspective.

Patient reported outcome measures (PROMs) are well-suited for the longitudinal assessment of quality of life, including depression and physical limitations associated with peripheral artery disease (PAD) that are not routinely assessed in clinical care. This study was designed to gain the patient perspective to facilitate implementation of PROMs into clinical practice for PAD management. Twenty-three patients with PAD at a single vascular surgery clinic were enrolled for a qualitative interview, July-December 2022. Patients completed PROMIS Physical Function and Depression assessments before undergoing semi-structured interviews. Two researchers used an inductive thematic analysis to analyze emergent themes from transcribed interviews. The average age of participants was 69.5 ± 8.2years; 91% were Caucasian, and 39% were female. Qualitative interviews revealed three implementation-related themes: (1) patient preferences on the timing and type of PROMs collected, (2) PROMs applications in outpatient PAD care, including discussions with their physician, and (3) the clinical value of PROMs. Overall, patients with PAD prefer PROMs related to quality of life and physical function over other domains. Patients appreciate the convenience to complete PROMs before their appointment. Patients would like to verbally discuss meaningful score changes with their providers without the use of graphical aids. Most patients believe PROMs are valuable in their clinical care if their physician reviews the results and the PROM questions apply to their disease symptoms. Patient preferences inform future successful implementations and will improve patient completion rates for the collection and clinical use of PROMs in PAD clinical care.

Current use of patient-reported outcome measures by musculoskeletal physiotherapists in Portugal.

Musculoskeletal conditions are the leading cause of disability, requiring continuous outcome measurement, including the utilization of patient-reported outcome measures (PROMs). The aims of the study were a) to characterize the use PROMs by Portuguese physiotherapists in patients with musculoskeletal conditions; b) to analyze the associated factors of their use and; c) to identify the barriers and facilitators to their implementation. A cross-sectional online survey was carried out with musculoskeletal physiotherapists working in Portugal. Closed questions were used to describe the use of PROMs. The factors associated with the non-use of PROMs were analysed through logistic regression models (p= 0.05). Barriers and facilitators were assessed through open questions and analyzed using thematic analysis. One hundred and fifty-six physiotherapists answered the online survey. Most physiotherapists do not use outcome measures consistently and physical tests are often chosen (70.5%) over PROMs (17.3%). The work sector (p= 0.01; p= 0.015; p= 0.009). the workplace stimulus (p= 0.001) and the physiotherapists' knowledge about PROMs (p= 0.017) were identified as factors associated with the non-use of PROMs. Barriers and facilitators to the use of PROMs are related to physiotherapists' skills, the patients' characteristics, the workplace conditions, and the instruments' characteristics. This study showed the poor use of PROMs by musculoskeletal physiotherapists and identified the associated factors, barriers, and facilitators that should be considered to increase its future use by musculoskeletal physiotherapists in Portugal.

Value-based outcome evaluation methods used by occupational therapists in primary care: a scoping review.

This scoping review aimed to map how occupational therapists evaluate the outcomes of services they provide within primary care. This evidence was considered in relation to how identified outcome evaluation methods align to principles of value-based health care. Primary care services are experiencing unprecedented demands. Occupational therapy is an allied health profession that supports health and care provision in primary care, using a timely and proactive approach. There has been a notable increase in occupational therapy roles across primary care services in the past decade; however, the mechanisms for evaluating outcomes and the wider impact of these services remain under-researched. The aim of value-based health care, a global transformative approach, is to establish better health outcomes for individuals and communities through addressing value in system-wide care. However, it is not yet clear how evaluation methods used within occupational therapy align to the principles of a value-based agenda. Peer-reviewed journal articles and gray literature written in English were included to identify outcome evaluation methods used by occupational therapists to evaluate the effectiveness and impact of occupational therapy services provided in a primary care setting. Outcome evaluation methods used exclusively for the purpose of conducting research and not for capturing data within an occupational therapy primary care setting as part of routine clinical practice were excluded. This review followed JBI methodology for scoping reviews. The literature search was undertaken during June and July 2022. The following databases were searched from their earliest dates of availability: Cochrane Library, MEDLINE via Ovid, Embase via Ovid, CINAHL via EBSCOhost, Scopus, AMED, and Web of Science. Two reviewers extracted data, supported by use of an extraction form developed by the reviewers. Findings were mapped using a framework developed based on key principles of value-based health care. From 2394 articles, 16 eligible studies were included in the review. Of these, 9 were quantitative and 7 were of mixed methods design. Studies were from the UK, USA, Sweden, Spain, and Canada. The occupational therapy services represented were mainly heterogeneous. Four services were part of multidisciplinary programs of care and 12 services were specific to occupational therapy. Identified outcome evaluation methods broadly aligned to principles of value-based health care, with most alignment noted for measures demonstrating the aim of establishing better health. A wide range of evaluation methods were described to address both individual-level and service-level outcomes, with the use of patient-reported outcome measures identified in 13 studies. To capture patient experience, most studies reported a variety of methods. The aim of reducing the per capita cost of health care was least represented in the literature. This scoping review highlights a multifaceted but inconsistent approach to measuring the outcomes of occupational therapy provided in primary care. This has implications for establishing effectiveness and capturing data at scale to assist with wider planning of care and to enable the profession to demonstrate its contribution to value-based health care. Open Science Framework https://osf.io/hnaq4/.

The use of PROMs (patient-reported outcome measures) and PREMs (patient-reported experience measures) in Cardiology: current state and applications to improve patient care

The integration of patient-reported outcomes and experiences through PROMs and PREMs tools represents a significant evolution in the quality of care and clinical practice. This article reviews the state of the art and the application experiences of these tools, focusing on their impact on the management of cardiovascular diseases. The use of PROMs and PREMs allows for a more comprehensive assessment of patient well-being by integrating with traditional clinical indicators. An increasing number of studies suggests that the systematic collection of these data can help physicians in better identifying individual patient's needs, improving doctor-patient communication, and personalizing therapies. In aggregate form, these data can be used to improve the quality of services. This article explores the main international experiences, starting from the activities carried out in this field by the European Society of Cardiology, the challenges in implementing such tools, and the prospects for a more widespread adoption in daily clinical practice. It emphasizes the activities carried out in Italy by the Fondazione Toscana Gabriele Monasterio and the Management and Healthcare Laboratory of the Scuola Superiore Sant'Anna of Pisa, highlighting the importance of validated tools, appropriate technologies, and a cultural change to promote patient-centered care.

Identifying Clusters of Curatively-Treated Esophageal Cancer Patients Using Patient-Reported Outcome Measures at Three Months from Discharge: A Secondary Analysis from a Longitudinal Single Center Study.

This study aims to identify meaningful clusters based on Patient-Reported Outcome Measures (PROMs) in curatively-treated esophageal cancer patients at three months post-discharge. This secondary analysis of a longitudinal single-center study included 46 esophageal cancer patients who underwent curative surgery. Patients were selected based on their completion of PROMs surveys at three months post-discharge, were aged 18 years or older, and had undergone surgical resection (esophagectomy) with or without neoadjuvant chemotherapy and/or radiotherapy. The analysis utilized t-distributed Stochastic Neighbor Embedding (t-SNE) for dimensionality reduction and hierarchical clustering to analyze PROMs data collected three months post-discharge. Clustering was performed on physical, emotional, cognitive, and social functioning variables, symptom burden, and health literacy. Three distinct clusters were identified: Cluster 1 (n = 24) with higher functioning and moderate symptoms, Cluster 2 (n = 14) with moderate functioning, higher symptoms, and lower health literacy, and Cluster 3 (n = 8) with the highest functioning, lowest symptoms, and highest health literacy. Significant differences between squamous cell carcinoma and adenocarcinoma subtypes were observed across several PROMs domains, including critical health literacy, general health status/quality of life, nausea and vomiting, and insomnia. These clusters provide an exploratory framework for tailoring post-operative interventions to enhance patient recovery, which necessitates further confirmatory investigations, including outcomes such as complications and mortality, in the analysis. This study fills a research gap by demonstrating the utility of PROMs in identifying distinct recovery patterns in esophageal cancer patients post-surgery. The findings support the use of PROMs to guide personalized post-operative care, potentially improving patient outcomes and quality of life. Further research is needed to validate these findings in larger, diverse populations.

Enhancing Clinicians' Use of Electronic Patient-Reported Outcome Measures in Outpatient Care: Mixed Methods Study.

Despite the increasing use of patient-reported outcome measures (PROMs) for collecting self-reported data among hospital outpatients, clinicians' use of these data remains suboptimal. Insight into this issue and strategies to enhance the use of PROMs are critical but limited. This study aimed to examine clinicians' use of PROM data for value-based outpatient consultations and identify efforts to enhance their use of PROMs in a Dutch university hospital. First, we aimed to investigate clinicians' use of outpatients' PROM data in 2023, focusing on adoption, implementation, and maintenance. Second, we aimed to develop insights into the organizational-level strategies implemented to enhance clinicians' use of PROM data from 2020 to 2023. This included understanding the underlying rationales for these strategies and identifying strategies that appeared to be missing to address barriers or leverage facilitators. Third, we aimed to explore the key factors driving and constraining clinicians' use of PROMs in 2023. We integrated data from 4 sources: 1-year performance data on clinicians' use of PROMs (n=70 subdepartments), internal hospital documents from a central support team (n=56), a survey among clinicians (n=47), and interviews with individuals contributing to the organizational-level implementation of PROMs (n=20). The Reach, Effectiveness, Adoption, Implementation, and Maintenance framework was used to analyze clinicians' adoption, implementation, and maintenance of PROMs. Strategies were analyzed using the Expert Recommendations for Implementing Change taxonomy, and results were structured around the constructs of capability, opportunity, and motivation. On average, around 2023, clinicians accessed PROM data for approximately 3 of 20 (14%) patients during their outpatient consultation, despite numerous strategies to improve this practice. We identified issues in adoption, implementation, and maintenance. The hospital's strategies, shaped organically and pragmatically, were related to 27 (37%) out of 73 Expert Recommendations for Implementing Change strategies. These strategies focused on enhancing clinicians' capability, opportunity, and motivation. We found shortcomings in the quality of execution and completeness of strategies in relation to addressing all barriers and leveraging facilitators. We identified variations in the factors influencing the use of PROMs among frequent PROM users, occasional users, and nonusers. Challenges to effective facilitation were apparent, with certain desired strategies being unfeasible or impeded. Enhancing clinicians' use of PROMs has remained challenging despite various strategies aimed at improving their capability, opportunity, and motivation. The use of PROMs may require more substantial changes than initially expected, necessitating a shift in clinicians' professional attitudes and practices. Hospitals can facilitate rather than manage clinicians' genuine use of PROMs. They must prioritize efforts to engage clinicians with PROMs for value-based outpatient care. Specific attention to their professionalization may be warranted. Tailored strategies, designed to address within-group differences in clinicians' needs and motivation, hold promise for future efforts.

The patient-reported outcome measure for older people living with frailty receiving acute care (PROM-OPAC): field-testing and validation

BackgroundCurrent acute healthcare service metrics are not meaningful for older people living with frailty. Healthcare knowledge, situational security, and physical and psychosocial function are important outcomes typically not collected. The use of patient-reported outcome measures (PROMs) could support these assessments. Existing instruments are not comprehensive as they typically consider function, while older people with frailty also value enablement (self-determination and security in health and healthcare). This study field-tested and validated a PROM for older people with frailty receiving acute care (PROM-OPAC) to measure enablement.MethodsPeople aged 65+ with Clinical Frailty Scale 5–8 were recruited within seventy-two hours of an emergency attendance. Iterations of the novel instrument were administered over three stages: (1) preliminary field-testing for reliability (response distribution and internal consistency) and structure (exploratory factor analysis, EFA); (2) intermediate field-testing of an improved instrument for reliability and structure; (3) final draft validation assessing reliability, structure (confirmatory factor analysis, CFA), and construct validity based on a priori hypotheses. Feasibility was appraised throughout using data completeness and response rates and times.Results241 people participated. Three items of a preliminary seven-item measure had poor response distribution or loading and were accordingly improved. The intermediate instrument had interpretability issues and three items required further improvement. The final eight-item draft had acceptable reliability (Cronbach’s alpha: 0.71), structure (two factors for self-determination and security; RMSEA: 0.065; TLI: 0.917; CFI: 0.944), and construct validity (lower scores from respondents waiting longer and requiring admission). Feasibility was promising (response rate 39%; 98% responses complete; median completion time 11 (IQR: 12) minutes).ConclusionsAdministration of the PROM-OPAC appeared feasible and the instrument had acceptable psychometric properties. Further evaluation is required to assess generalisability.

The use of respiratory patient reported outcome measures (PROMs) in the management of COPD: Perceptions of Australian osteopaths

BackgroundPatient reported outcome measures (PROMs) are widely used to capture patients' perspectives of their health status and treatment outcomes and to enable practitioners to evaluate the effectiveness of interventions. The aim of this study was to explore Australian osteopaths’ perceptions of the use of respiratory PROMS in the management of chronic lung conditions. MethodsThis study drew on the social ecological model (SEM), which highlights the interdependence of contextual structures and processes. Osteopaths who were initially part of a larger mixed-methods study were invited to participate in semi-structured interviews. Transcripts were thematically analysed using the SEM. ResultsSeven of the original study osteopaths were interviewed. When presented with a patient who had a co-morbid respiratory condition, they focused on the musculoskeletal aspects of that condition (intrapersonal factors). Participants described sporadic use of musculoskeletal and quality of life PROMs in practice, and scarce use of respiratory PROMs (intrapersonal and organizational factors). Participants’ perceptions of their scope of practice strongly influenced their use of respiratory PROMs (environmental factors). ConclusionIntrapersonal and environmental factors were dominant drivers for participants’ scant use of respiratory PROMs. Further education on the scope and contribution of osteopaths to the management of chronic lung conditions and the use of respiratory PROMS is called for to promote multidisciplinary patient care.

Patient-reported outcome measures in orthopaedics.

The Patient Activity Treatment Outcome Scale (PATOS) is a novel patient-reported outcome measure (PROM). This study explored patients' and orthopaedic surgeons' experiences with PATOS as part of a PROM battery. It aimed to investigate its influence on patient involvement and healthcare decisions about knee or hip osteoarthritis patients scheduled for knee/hip arthroplasty. A qualitative research design was adopted using semi-structured interviews with 19 patients and nine orthopaedic surgeons at a Danish orthopaedic surgery department. Thematic analysis was used to develop key themes from interview transcripts. This study adds nuanced insights related to the use of PROMs in routine orthopaedic clinical practice. Home-based completion of PROMs prompted patients to reflect on their situations, priorities and goals regarding potential hip or knee arthroplasty, leading to better-prepared discussions with surgeons. The integration of PATOS with other PROMs made it challenging to discuss PATOS exclusively. Therefore, the results relate to the full PROM battery. The experiences of patients and surgeons were organised into four themes: 1) Patient involvement is key, 2) Questionnaire load, 3) Meaningful home-based completion and 4) PROMs were not used in the decision-making process. The questionnaire load and limited integration into healthcare decision processes raised concerns about the application of the PROM battery. The results emphasise the importance of refining the application of PROMs in orthopaedic practice from the perspectives of patients and surgeons alike. This work was supported by Innovation Fund Denmark [0172-01258B] TRIAL REGISTRATION. Not relevant.

MyMS: An Interface for Patient-Reported Outcomes for Finnish Individuals With Multiple Sclerosis.

Patient-generated data are a cornerstone of individualized multiple sclerosis (MS) treatment. MyMS, an interface for patient-reported outcomes (PROs) was developed by the Finnish MS Register to enable systematic collection of PROs. MyMS collects data on demographics, lifestyle factors, disease-related factors, and validated questionnaires, including the Quality of Life Questionnaire (15D), the Multiple Sclerosis Impact Scale (MSIS-29), and the Fatigue Severity Scale (FSS). At the end of 2020, the patient-reported Expanded Disability Status Scale (PREDSS), the EuroQOL-5 Dimension (EQ-5D), the Fatigue Scale for Motor and Cognitive Functions (FSMC), and the Multiple Sclerosis Neuropsychological Questionnaire (MSNQ) were added. As of January 1, 2023, 1201 individuals with MS (79% female) have added data to MyMS. Of the validated PRO measures (PROMs), the 15D, MSIS-29, and FSS are the most used. The mean PREDSS score is 3.0 and median disease duration is 6.4 years. According to the existing PROMs, patients report mildly compromised quality of life and problems with fatigue and cognition. The patient interface of the Finnish MS Register consists of data from 17 of 21 counties with well-being services. The interface is used by 10% of Finnish individuals with MS. The addition of the PREDSS, EQ-5D, FSMC, and MSNQ to the interface has increased health care professional and patient interest in the use of PROMs. We suggest that PROs should be integrated into electronic health records to improve shared decision-making and diminish documentation burden.

Using Implementation Science to Evaluate the Implementation of Patient-Reported Outcome Measures (PROMs) in a Clinical Heart Failure Care Setting

BackgroundPatients with heart failure (HF) can experience a poor quality-of-life (QOL), recurring hospitalizations, and progressive disease symptoms. Patient-reported outcome measures (PROMs) include integration of patients’ voices into clinical care, by assessing patient symptoms, function, and QOL. In 2022, PROMs were incorporated into the electronic health record system (Epic) at a large academic hospital in Toronto, Ontario, Canada. The purpose of this study was to use implementation-science frameworks to systematically evaluate the uptake and integration of PROMs into clinical HF care. MethodsThe Reach, Effectiveness, Adoption, Implementation, and Maintenance (RE-AIM) framework guided this mixed-methods, 1-year, quality-improvement project. Data sources included the following: clinician use of PROMs; patient-level data on completed PROMs; and semistructured interviews with clinicians. The PROM was the Kansas City Cardiomyopathy Questionnaire-12, which captures 4 domains related to HF—symptom frequency, physical limitations, social limitations, and QOL. Quantitative data were analyzed using descriptive statistics; qualitative data were analyzed using behaviour-change frameworks and latent content analysis. ResultsOver the course of 1 year, more patients were assigned to PROMs, a higher proportion of patients completed PROMs, and approximately 80% of patients had high scores on the questionnaire. Clinicians experience barriers—related to attention and decision processes, the environmental context, and their professional role—to integrating PROMs into practice. Suggested changes to improve PROM uptake include adding language licenses for PROM translations, reducing cognitive load for clinicians who are assigning and interpreting PROMs in the Epic system, and championing modelling of use of PROMs in practice. ConclusionsThis study demonstrates the benefit of using implementation science frameworks, to evaluate the implementation of PROMs in practice and provide actionable recommendations to health systems.

A 15-Year Bibliometric Analysis of Sports Medicine Studies in The Journal of Bone and Joint Surgery: A Systematic Review.

Orthopaedic sports medicine is among the most popular subspecialties. Understanding the trends in sports medicine research over time can offer insight into progress and innovation within the field. The purpose of this study was to assess both the quality of the current literature and trends in gender diversity and inclusion by evaluating publishing characteristics of sports medicine studies in The Journal of Bone and Joint Surgery-American Volume (JBJS-A) from 2007 to 2021. Sports medicine studies in JBJS-A from 2007 to 2021 were identified using JBJS subspeciality tags for "sports medicine" articles and organized by study type, number of authors, sex of the authors, academic degree(s) of the first and last authors, level of evidence, country of publication, citations, and use of patient-reported outcomes (PROM). A total of 784 studies were reviewed, and 513 met inclusion criteria. Clinical therapeutic studies were the most common publication (48%). There was an increase in the publication of clinical prognostic studies (17%-25%, p = 0.037) and a significant increase in the use of PROM measures over time (13%-47%, p < 0.001). The total number ofauthors increased over the study period (4.8-6.3), but there was no significant increase in female authorship. Only 15% of the 784 studies included a female author, with an average of 0.8 female authors per article (range 0-8) compared with 4.6 males (range 1-14). The significant increase in the use of PROMs in sports medicine studies indicates that the quality of research has improved over the 15-year period. The gender disparity in authorship has remained stagnant. Only 11% of all first authors and 9% of senior authors were female. The number of included international studies improved over time; however, the United States remains the most prolific publisher. Despite these areas of growth, this study suggests that there is room for improvement of authorship gender diversity in orthopaedic sports medicine research. Level III. See Instructions for Authors for a complete description of levels of evidence.

Patient-reported assessments of chronic cough in clinical trials: accessory or primary endpoints?

Chronic cough is a complex disorder that affects up to 5-10% of the general population. It can be challenging to manage as there are few effective treatments, although several novel antitussives are in clinical development. The endpoints used to assess their efficacy in clinical trials should be optimal; most large clinical trials currently use objective measures as the primary outcome, especially cough frequency. There are strengths in this approach, although taking the view that other measures of chronic cough are less important, including patient-rated cough severity, psychosocial impact and other associated symptoms. Patient-reported outcome measures (PROMs) explore patients' personal experiences of health and disease, and the effects of particular conditions on their lives. Numerous validated PROMs exist for chronic cough, from simple visual analogue scales, to those that focus on cough hypersensitivity and cough-specific quality of life. Medicine regulators in the European Union (EU) and United States of America (USA) encourage the use of PROMs in clinical trials but have voiced concerns over their content validity, clinically meaningful thresholds for change, and discordance with objective measures. There are recent and ongoing studies to address these limitations. This review discusses currently available PROMs used to assess chronic cough and discusses their potential role as primary outcome measures in clinical trials.

Breast-cancer specific comprehensive archive of Patient-Reported Outcome Measures (PROMs) for clinical research and clinical practice in oncology: Results from the PRO4All project

BackgroundInclusion of patient-reported outcomes (PROs) in oncology clinical trials is strongly recommended. However, selecting the most appropriate patient-reported outcome measures (PROMs) is not easy. This study aimed to develop a breast cancer (BC) specific comprehensive archive of PROMs. MethodsAs part of the PRO4All project, we identified available PROMs in oncology by searching facit.org, eortc.org, eprovide.mapi-trust.org, PubMed, ema.europa.eu (European Public Assessment Reports) and published reviews. For this analysis, only BC tools were extracted. We described information about PROM name, type of questionnaire, questionnaire variant(s), recall period, number of items, and presence of minimum clinically important difference (MCID) reference in literature. Then, we assigned each item to a specific domain according to a predefined taxonomy of 38 items for outcome classification. ResultsWe identified and analyzed 383 PROMs. Of these, 29 were BC specific, but 2 were excluded because the questionnaires description was not available. 6 (22.2 %) were variants of another questionnaire. All questionnaires were self-reported. In 6 cases (22.2 %) the recall period to consider was the “last week”. The mean number of items per questionnaire was 25.81 (range 6–71). 602 items were assigned to an outcome domain: emotional functioning/wellbeing in 26.6 % of cases, physical functioning in 14.1 %, delivery of care in 10.8 %, and general outcomes in 10.5 %. MCID reference was found only in 4 (14.8 %) cases. ConclusionsThe newly developed archive represents a useful tool to optimize the use of PROMs in the evaluation of treatments in BC patients, promoting a patient-centered approach both in clinical research and practice.

Patient-reported outcome measures for acute rhinosinusitis in adults and children: a systematic review of the quality of existing instruments

BackgroundAcute rhinosinusitis (ARS) is a self-limiting inflammation of the nose and sinuses caused by viral or bacterial infections that requires primarily symptomatic treatment. Patient-reported outcome measures (PROMs) are suitable tools for the assessment of the effectiveness of remedies for ARS from the patient’s perspective in clinical trials and real-world studies. Data regarding the quality of existing PROMs for ARS are limited.PurposeTo conduct a systematic review of the quality of existing disease-specific PROMs for use in adults and children with ARS according to the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) methodology, and to derive recommendations for use of the identified instruments in future clinical studies.MethodsWe systematically searched PubMed, Web of Science and Embase for studies reporting on the development and/or validation of PROMs for ARS. We assessed the methodological quality of each included study, evaluated the quality of measurement properties per PROM and study, and graded the evidence. Based on the overall evidence, we derived recommendations for use of the instruments.ResultsWe identified four studies on three PROMs measuring symptoms of ARS and quality of life in adults (Sinonasal Outcome Test-16, SNOT-16; Measurement of Acute Rhinosinusitis, MARS; Rhinosinusitis Quality-of-Life Assessment, RhinoQoL). For ARS in children, we identified two studies on two PROMs measuring symptoms of ARS (Pediatric Rhinosinusitis Symptom Scale, PRSS; Sinus Symptom Questionnaire, S5). Our assessment of measurement properties indicates that all instruments require further validation before they can be unrestrictedly recommended for use in future research (COSMIN category B). In particular, there were no content validity studies for any of the identified instruments, but also data on other important measurement properties, e.g., structural validity, are lacking.ConclusionCurrently, no PROM for ARS in adults and children can be unrestrictedly recommended based on the evaluation of their quality. Further validation is required for all identified PROMs. Content validation involving patients and experts should be given priority.Systematic review registrationOSF (https://doi.org/10.17605/OSF.IO/VAP8U).

1852P The use of patient-reported outcome measures to assess toxicities and quality of life associated with immune checkpoint inhibitor therapy: A systematic review

1852P The use of patient-reported outcome measures to assess toxicities and quality of life associated with immune checkpoint inhibitor therapy: A systematic review

COSMIN guideline for systematic reviews of patient-reported outcome measures version 2.0

PurposeSystematic reviews of patient-reported outcome measures (PROMs) are important tools to select the most suitable PROM for a study or clinical application. Conducting these reviews is challenging, and the quality of these reviews needs to be improved. We updated the COSMIN guideline for systematic reviews of PROMs, including the COSMIN Risk of Bias checklist, and the COSMIN criteria for good measurement properties.MethodsAdaptations to the methodology were based on our experience with applying the COSMIN guideline, through discussions among the authors, and results from two related Delphi studies.ResultsThe updated guideline places more emphasis on key aspects that are often missing or sub optimally conducted in published systematic reviews of PROMs, such as formulating a well-defined research question and developing a comprehensive search strategy, assessing risk of bias, applying criteria for good measurement properties, summarizing results, and grading the quality of the evidence. We also stress the importance of evaluating the measurement properties of each subscale of a PROM separately and evaluating content validity of all included PROMs.ConclusionThe quality of systematic reviews of PROMs can be improved by using this updated version of the COSMIN guideline for systematic reviews of PROMs. Improved quality will lead to better PROM selection and increased standardization of PROM use.