Selection Of Patient-reported Outcome Measures Research Articles

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.

Heterogeneity of patient-reported outcome measures in clinical research

BackgroundThe use of patient-reported outcome measures (PROMs) in clinical research increases and use of heterogeneous instruments reflects how well diverse traits are captured by a medical specialty. In order to reflect the heterogeneity of current PROM use in ophthalmology, we reviewed the available literature.MethodsThe medical literature database Web of Science was searched for the most cited articles in clinical ophthalmology. Titles, abstracts and full text articles were reviewed for the use of PROMs and a list of the 100 most cited articles using PROMs was obtained and stratified by year of publication.ResultsA total of 1,996 articles were screened. Seventy-seven out of the 100 articles identified included one PROM, and the average number of instruments was 1.5 ± 1.1. The most widely used PROMs were the National Eye Institute Visual Function Questionnaire (33%), the Ocular Surface Disease Index (14%) and the Medical Outcomes Study Short Form (13%). A simulation analysis suggested that the distribution of PROM use in ophthalmology study did not significantly differ from a power law distribution. Twenty-two percent and fifteen percent of articles did not reference and did not specify the PROM used, respectively. This rate decreased in the more recently published articles (p = 0.041).ConclusionsOur data suggest that the heterogeneity of PROMs applied in ophthalmology studies is low. The selection of PROMs for clinical studies should be done carefully, depending on the research goal.

PCR20 Assessing Activity Limitations Caused by Rheumatoid Arthritis to Inform Appropriate Selection of Patient-Reported Outcomes Measures

PCR20 Assessing Activity Limitations Caused by Rheumatoid Arthritis to Inform Appropriate Selection of Patient-Reported Outcomes Measures

Selecting patient-reported outcome measures for a patient-facing technology.

This article provides insight into our process and considerations for selecting patient-reported outcome measures (PROMs) designed for self-reporting symptoms and quality-of-life among breast cancer (BCA) patients undergoing oral anticancer agent treatment via a patient-facing technology (PFT) platform. Following established guidelines, we conducted a thorough assessment of a specific set of PROMs, comparing their content to identify the most suitable options for studying BCA patients. We recommend utilizing the combination of EORTC QLQ-C30 + EORTC QLQ-BR45 as the preferred instrument, especially when developing a dedicated "breast cancer-only" application. When developing and maintaining a dashboard for a PFT platform that includes multiple cancer types, it is important to consider the feasibility of interface design and workload. To achieve this, we recommend using PRO-CTCAE+PROMIS 10 GH for the PFT. Moreover, it is important to consider adding ad hoc items to complement the chosen PROM(s). This article describes our efforts to identify PROMs for self-reported data while considering patient and developer burdens, providing guidance to PFT developers facing similar challenges in PROM selection.

Patient Reported Outcome Measures in the Foot and Ankle Literature: A Systematic Review

Category: Other Introduction/Purpose: Optimising clinical practice requires emphasis on patient-orientated care and therefore patient reported outcome measures (PROMs) play a key role in shaping evidence-based clinical guidelines. As such, it is crucial that we use appropriate, valid and reliable PROMs to quantify outcomes in orthopaedic research. A previous systematic review evaluating evidence published between 2002-2011 identified 139 different PROMs used in the foot and ankle literature and found the most common PROM was The American Orthopaedic Foot and Ankle Society (AOFAS) score. However, in 2011, AOFAS issued a position statement outlining the lack of reliability and validity of the AOFAS score, describing it as a ‘less than ideal’ measure. This systematic review aims to provide an updated analysis of the PROMS used in the foot and ankle literature. Methods: This systematic review was performed according to PRIMSA guidelines. An electronic title/abstract screen of every article published between January 2012- December 2022 in Foot and ankle international, Clinical Orthopaedics and Related Research, Knee Surgery Sports Traumatology and Arthroscopy, The Bone and Joint Journal and The American Journal of Sports Medicine was performed by two reviewers. Articles with a foot and ankle focus were selected for full text screening and papers reporting at least one PROM were included in the final review. The following data was extracted from each article: journal of publication, month/year of publication, pathology/intervention described, PROMS reported and use of the minimally clinically important difference (MCID) to evaluate post intervention changes in PROMs. The number of studies using each individual PROM was calculated and a subgroup analysis was performed to investigate variation in PROM selection according to year of publication, journal of publication and the condition being investigated. Results: A total of 3,174 foot and ankle articles were identified, of which 1,168 (36.8%) articles reporting at least one PROM were included. The most common PROM was the AOFAS score, reported in 50.3% of studies, followed by visual analogue scale (VAS) pain score (44.4%) and 36-item short form survey (SF-36) (15.3%). Use of the AOFAS score declined across the review period. Between 2012-2016 AOFAS was the most common PROM, whilst VAS pain was most common in four of the last six years. Although some variation in PROMs according to clinical pathology exists, AOFAS score is featured in the top 3 most common PROMs for every analysed condition. Only 8/1,168 (0.7%) articles reported the number of patients achieving a minimally clinically important difference (MCID) post-intervention. Conclusion: Whilst use of the AOFAS score declined over the review period, it remains the most common PROM overall across all included journals and many conditions. There exists a lack of consensus regarding the most suitable alternative PROMs, with multiple validated instruments including FAOS, FAAM and FFI used inconsistently with relatively low frequency. There is a heavy reliance on statistical significance to evaluate and compare post-intervention PROM changes. However, it is important to quantify PROMs according to the patient perspective. Reporting the proportion of patients achieving MCID will allow clinicians to interpret PROMs and develop management guidelines in a patient-focused manner.

Selection of patient-reported outcomes measures for implementation in the Metabolic and Bariatric Surgery Accreditation Quality Improvement Program.

The Metabolic and Bariatric Surgery Accreditation Quality Improvement Program (MBSAQIP) assesses safety after metabolic and bariatric surgery and the impact on weight and obesity-related diseases. However, changes in quality of life are likely what matters most to patients, and these are not currently assessed. The best way to measure health-related quality of life (HRQoL) is to use validated patient-reported outcomes measures (PROMs), which capture patients' perspectives of their quality of life both before and after surgery. Identify the outcomes most important to bariatric surgery patients and identify the most appropriate validated PROMs to implement in a national program for the MBSAQIP. Five hospitals from a single healthcare system in New England. A series of 18 focus groups and/or interviews conducted with patients, patients' family members, and bariatric health providers determined the outcomes most important to bariatric patients and which validated PROMs would accurately measure those outcomes. Immersion crystallization was used to analyze focus group data and identify appropriate PROMs. Focus group participants ranked health as the most important outcome for metabolic and bariatric surgery. Self-confidence, mobility, and everyday activities were the next highest ranked HRQoL domains. The Patient-Reported Outcomes Measurement Information System 10-Item Global Health Survey was selected as the general health measure. The Obesity-Related Problems scale and the Obesity and Weight-Loss Quality of Life Instrument were the disease-specific measures selected for inclusion in the MBSAQIP PROMs program. The addition of PROMs to the MBSAQIP provides a unique opportunity to monitor HRQoL at the national level, which can foster improved shared decision-making before surgery.

Approach to patient reported outcome measures selection and implementation in a chronic norovirus clinical efficacy trial for patients after solid organ transplant.

Norovirus is the second most common cause of diarrhea among solid organ transplant recipients. There are currently no approved therapies for Norovirus, which can substantially impact quality of life, particularly in an immunocompromised patient population. In order to establish clinical efficacy and support any medication claims related to a patient's symptoms or function, the Food and Drug Administration requires that a trial's primary endpoints be derived from patient reported outcome measures- an outcome that comes directly from the patient, without interpretation of the patient's response by a clinician or anyone else. In this paper, we describe our study team's approach to the definition, selection, measurement and evaluation of patient reported outcome measures as part of establishing clinical efficacy of Nitazoxanide for acute and chronic Norovirus in solid organ transplant recipients. We specifically describe our approach to measuring the primary efficacy endpoint- days to cessation of vomiting and diarrhea after randomization through 160 days, measured through daily symptom diaries as well as the impact of treatment on exploratory efficacy endpoints, particularly change in the impact of norovirus on psychological function and quality of life.

Routine patient assessment and the use of patient-reported outcomes in specialized palliative care in Japan

BackgroundDiscrepancies in symptom assessment between providers and patients are reported in cancer care, and the use of patient-reported outcome measures (PROMs) has been recommended for patients receiving palliative care. However, the status of the routine use of PROMs in palliative care in Japan is presently unclear. Therefore, this study aimed to clarify this complex question. To this end, we administered a questionnaire survey either online or via telephone interviews (questionnaire: sent to 427 designated cancer hospitals, 423 palliative care units [PCUs], and 197 home hospices; interviews: conducted at 13 designated cancer hospitals, nine PCUs, and two home hospices).ResultsQuestionnaires were returned from 458 institutions (44% response rate). We found that 35 palliative care teams (PCTs, 15%), 66 outpatient palliative care services (29%), 24 PCUs (11%) and one (5%) home hospice routinely used PROMs. The most frequently implemented instrument was the Comprehensive Care Needs Survey questionnaire. Moreover, 99 institutions (92%) that routinely used PROMs responded these instruments as useful in relieving patients’ symptoms; and moreover, the response rate in regard to usefulness in symptom management was higher than that of institutions that did not routinely use PROMs (p = 0.002); > 50% of the institutions that routinely used PROMs stated that use of these instruments was influenced by disease progression and patients’ cognitive function. Moreover, 24 institutions agreed to be interviewed, and interviews demonstrated the benefits of and the barriers to the implementation of PROMs. Effective methods used in the implementation of PROMs were introduced as efforts to reduce the burden placed on patients and to promote healthcare providers’ education in the use of PROMs.ConclusionsThis survey quantified the status of the routine use of PROMs within specialized palliative care in Japan, revealed barriers to wider PROM use, and identified needed innovations. Only 108 institutions (24%) routinely used PROMs within specialized palliative care. Based on the results of the study, it is necessary to carefully consider the usefulness of PROs in clinical palliative care, perform careful selection of PROMs according to the patient's condition, and evaluate how specifically to introduce and operate PROMs.

Embedding patient-reported outcomes at the heart of artificial intelligence health-care technologies.

Integration of patient-reported outcome measures (PROMs) in artificial intelligence (AI) studies is a critical part of the humanisation of AI for health. It allows AI technologies to incorporate patients' own views of their symptoms and predict outcomes, reflecting a more holistic picture of health and wellbeing and ultimately helping patients and clinicians to make the best health-care decisions together. By positioning patient-reported outcomes (PROs) as a model input or output we propose a framework to embed PROMs within the function and evaluation of AI health care. However, the integration of PROs in AI systems presents several challenges. These challenges include (1) fragmentation of PRO data collection; (2) validation of AI systems trained and validated against clinician performance, rather than outcome data; (3) scarcity of large-scale PRO datasets; (4) inadequate selection of PROMs for the target population and inadequate infrastructure for collecting PROs; and (5) clinicians might not recognise the value of PROs and therefore not prioritise their adoption; and (6) studies involving PRO or AI frequently present suboptimal design. Notwithstanding these challenges, we propose considerations for the inclusion of PROs in AI health-care technologies to avoid promoting survival at the expense of wellbeing.

Modified e-Delphi Process for the Selection of Patient-Reported Outcome Measures for Children and Families With Type 1 Diabetes Using Continuous Glucose Monitors: Delphi Study.

Type 1 diabetes (T1D) management is complex and associated with significant psychosocial burden. Continuous glucose monitors (CGM) can improve disease management and outcomes and introduce new or exacerbate existing psychosocial concerns. Patient-reported outcome measures (PROMs) can be used to capture this information, but there is no consensus on which PROMs should be used in pediatric CGM research. Here we describe the process to (1) identify PROMs that could be used to assess the impact of CGMs on pediatric patients with T1D, (2) implement a modified electronic Delphi (e-Delphi) methodology to arrive at an expert consensus on which PROMs are most suitable for clinical and research applications, and (3) establish a periodicity table for the administration of PROMs over time in a real-world evidence study. To identify appropriate PROMs for pediatric patients and families with T1D and CGMs, we conducted an asynchronous, e-Delphi process with a multidisciplinary group of experts from around the country. We identified candidate instruments through a literature review. The 3-round e-Delphi process was conducted via a study website, email, and web-based forms. Participants provided opinions on the usefulness of instruments, age validation, feasibility, time, and frequency of administration. In total, 16 experts participated in the e-Delphi process; 4 of whom consistently participated in all 3 rounds. We identified 62 candidate instruments, which were narrowed down to 12 final PROMs across 5 domains: diabetes distress and burden (n=4), autonomy (n=2), quality of life (n=1), psychosocial (n=3), and technology acceptance (n=2). A quarterly administration schedule was developed to reduce burden on participants. PROMs can provide critical insights into the psychosocial well-being of patients. The specific measures identified in the paper are particularly well suited for pediatric patients with T1D using CGMs. Clinical implementation could help health care providers, patients, and families to engage in more comprehensive disease management.

Exploring Patient's Preference of Patient-Reported Outcome Measures in Laryngeal Movement Disorders.

Despite many available patient-reported outcome measures (PROMs) for laryngeal movement disorders, there is a lack of patient input regarding which PROM most accurately and conveniently captures aspects related to their vocal disease. This study aimed to assess patients' preferences among a selection of voice-related PROMs (Voice Handicap Index-10 [VHI-10], OMNI-Vocal Effort Scale [OMNI-VES], Communicative Participation Item Bank-General Short Form [CPIB-10], and Visual Analog Scales [VAS]) within the laryngeal movement disorder population and investigate associations between selected instruments. Prior to botulinum toxin A injection, patients with laryngeal dystonia and/or essential tremor of the vocal tract were administered the VHI-10, OMNI-VES, CPIB-10, and three novel VAS questions in a randomized order. Patients rank ordered the four PROMs based on the PROMs' reflection of their voice problems. Pearson's correlation coefficients evaluated pairwise associations among PROM scores. Fisher's exact test compared the preferred PROM rankings. Seventy patients (53 female, mean age=60.7 years) participated. The VHI-10 and CPIB-10 were most preferred at 33.9% and 27.4% respectively. The OMNI-VES and VAS scales were less favored (19.4%, each). When analyzed by age ≥60 years, the CPIB-10 was most favored (33.3%), but for age <60 years, VHI-10 was most preferred (42.3%). There was a strong correlation between scores of all administered PROMs (strongest correlation between OMNI-VES and VAS, r= 0.8, p< 0.001; the weakest correlation between OMNI-VES and VHI-10, r= 0.6, p< 0.001). With an increasing trend in PROMs usage and a strong correlation between all evaluated outcome instruments, insight regarding patients' PROM preferences is an area for further consideration. NA Laryngoscope, 133:1448-1454, 2023.

P9.48: A Systematic Review of the Use of Patient-Reported Outcome Measures (PROMs) in Adults Undergoing Liver Transplantation

Background: Improved survival rates of liver transplantation (LT) (~80% 5-year survival) has shifted the focus towards reducing symptom burden and improving quality of life, which can be assessed using patient-reported outcome measures (PROMs). This study systematically reviewed measurement properties of PROMs to serve as an evidence base for the selection of suitable PROMs and offer new benchmarks for value-based health-care in LT. Methods: MEDLINE, EMBASE, PubMed and COCHRANE databases were searched for relevant articles. Studies were included if they reported PROMs in LT candidates and/or recipients. Articles including patients <16 years only and clinician-assessed instruments were excluded. The COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist was used to asses methodological quality of included studies and measurement properties. Results: The full-texts of 185 out of 2040 studies were reviewed. The Short-Form-36 (SF-36) (n=86 studies) and Hospital Anxiety and Depression Scale (HADS) (n=16 studies) were the most commonly reported generic instruments in this population. Only 15 studies provided original measurement properties for 24 PROMs. Both SF-36 (n=2 studies) and HADS (n=2 studies) showed high quality evidence for sufficient internal consistency (Cronbach’s alpha >7.8 and >7.3, respectively), but indeterminate reliability. The Liver Disease Quality of Life Questionnaire and its short version ((SF-)LDQOL) were the most used disease-specific PROMs reported in LT candidates (n=8 studies), and showed moderate quality evidence for sufficient internal consistency and construct validity in 2 studies. Five individual studies reported newly developed PROMs for LT recipients, often incorporating questions from existing instruments. These showed generally high quality evidence and sufficient internal consistency. Conclusion: In addition to well-established PROMs (SF-36 and HADS), we conclude that the (SF-)LDQOL is the most promising for the detection of disease-specific changes in LT candidates. However, this instrument should be evaluated further for LT recipients.

A Systematic Review of Outcome Measures in Orthopaedic Trauma Trials: What Are We Measuring?

The objectives of this study are to describe the outcome measures used in orthopaedic fracture care trials, with a particular focus on patient-reported outcome measures (PROMs), and to determine which study characteristics are associated with number of citations. We retrieved randomized clinical trials on fracture care between 2012 and 2017 from Embase, Medline and CENTRAL databases. Data collected included study characteristics (e.g., region, design, setting, sample size) and outcome measures (e.g., primary variable, measurement perspective, use of PROMs, study results and number of citations). We identified a total of 8,580 articles in the initial search. After title screening, abstract screening and full-text review, we included 416 articles for analysis. 58.4% (243) of the studies clearly defined a primary outcome measure and 56.3% (234) reported sample size justifications for outcome selection. The most common primary outcome reported was a visual analogue scale for pain; used in 21 of the 243 (8.6%) studies that defined a primary outcome. At least one PROM was used in 68.5% (285) of the papers included. A large proportion of studies reporting on PROMs for orthopaedic trauma patients do not provide key information on the outcome selection process; a step of utmost importance in and the designing and reporting of RCTs. There is substantial heterogeneity in the selection of PROMs for fracture care trials, which limits the ability to compare and summarize across studies. Future research in fracture care should strive towards improving the reporting of informative PROMs, with rationale that demonstrates understating of the injury, intervention and patient values. The online version contains supplementary material available at 10.1007/s43465-022-00667-8.

Patient Reported Outcome Measures for Rheumatoid Arthritis Disease Activity: a systematic review following COSMIN guidelines.

BackgroundThe current standard of care in rheumatoid arthritis (RA) requires regular assessment of disease activity (DA). All standard RA DA measurement instruments require joint counts to be undertaken by a healthcare professional with/without a blood test. Few healthcare providers have the capacity to assess patients as frequently as stipulated by guidelines. Patient Reported Outcome Measures (PROMs) could be an efficient and informative way to assess RA DA, which is highlighted by the SARS-COV-2 pandemic, as most consultations are remote rather than face-to-face. We aimed to assess all PROMs for RA DA against the internationally recognised COSMIN guidelines to provide evidence‐based recommendations to select the most suitable PROMs.MethodsReview registered on PROSPERO as CRD42020176176. The search strategy was based on a previous similar systematic review and expanded to include all articles up to January 2019. All identified articles were rated by two independent assessors following the COSMIN guidelines.Results668 abstracts were identified, with 10 articles included. A further 21 were identified from a previous review. Ten PROMs were identified. There was insufficient evidence to place any of the identified PROMs into recommendation for use category A due to lack of evidence for content validity, as stipulated by the COSMIN guidelines.ConclusionLack of evidence of content validity limits suitable PROM selection, therefore none can be recommended for use. It is acknowledged that all included PROMs were developed before the COSMIN guidelines were published. Future research on PROMs for RA DA must provide evidence of content validity.

Psychometric properties of patient-reported outcome measures for dysphagia in head and neck cancer: a systematic review protocol using COSMIN methodology

BackgroundDysphagia (swallowing difficulty) is one of the most common and debilitating sequelae of head and neck cancer (HNC). Patient-reported outcome measures (PROMs) are a fundamental component of dysphagia outcomes evaluation, as they inform treatment consequences that cannot be captured by objective clinician measures. Many PROMs for dysphagia in HNC are available, but their validity is unclear. As a consequence, the selection of the most appropriate PROM for dysphagia in HNC is complex and often based on the clinician’s personal preferences, rather than on valid psychometric properties. This protocol describes a systematic review aiming at (1) identifying PROMs specific to dysphagia symptoms, swallowing functional status, swallowing-related health status, and swallowing-related quality of life in HNC, (2) mapping them to our conceptual framework of dysphagia-related PROs, and (3) appraising their psychometric properties using the Consensus Based Standards for the Selection of Health Measurement Instrument (COSMIN) methodology.MethodsSix electronic databases will be searched from inception to December 2020 for all primary studies in any language and design detailing PROM development, reliability, validity, feasibility, interpretability, and/or cross-cultural adaptation. Eligibility criteria will target PROMs for patients with HNC (≥ 90% of the study sample) with ≥ 20% of their items pertaining to swallowing. Two independent raters will screen abstract and full texts and a third rater will resolve discrepancies. Data will be extracted on study, sample and PROM characteristics, and results of psychometric testing. PROMs will be mapped to our conceptual framework. The methodological quality of included PROMs and their psychometric properties will be appraised using the COSMIN risk of bias checklist and evidence will be summarized using a modified Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach.DiscussionThis systematic review will provide a summary of existing dysphagia-related PROMs for people with HNC and a comprehensive account of their psychometric properties. We will provide recommendations on PROMs selection which will aid healthcare professionals to the most appropriate PROM based on its validity, reliability, feasibility, interpretability and suitability for clinical and research settings. Further recommendations will be made on areas of measurement property requiring further testing.Systematic review registrationPROSPERO registration ID: CRD42021237877

Patient-Reported Outcome Measures in Symptomatic, Non-Limb-Threatening Peripheral Artery Disease: A State-of-the-Art Review.

Patient-reported outcome measures (PROMs) are health outcomes directly reported by the patient that can be used to measure the effect of disease and treatments on patient perceived well-being. This review summarizes current evidence regarding the validation of PROMs in people with symptomatic, nonlimb-threatening peripheral artery disease. A literature search was conducted to identify studies of symptomatic peripheral artery disease without limb-threatening ischemia that included PROMs and had sample sizes ≥25. PROMs were summarized along a continuum of validation using classical test theory framework and according to whether they fulfilled defined criteria for (1) content validity; (2) psychometric validation; and (3) further validation evidence base expansion. Of 2198 articles identified, 157 (7.1%) met inclusion criteria. Twenty-four PROMs in patients with symptomatic peripheral artery disease were reviewed. Among disease-specific PROMs, 8 of 15 had excellent reliability as measured by a Cronbach alpha ≥0.80. Based on established criteria for PROM responsiveness, 6 of 15 disease-specific PROMs demonstrated excellent sensitivity to change. Of these, the disease-specific peripheral artery questionnaire, vascular quality of life questionnaire, and walking impairment questionnaire met criteria for validation at each stage of the continuum. For generic (nondisease specific) PROMs, the European Quality of Life 5-Dimension and SF-36 had the most extensive evidence of validation. Evidence from this review can inform selection of PROMs aligned with scientific and clinical goals, given the variable degree of validation and potential complementary nature of the measures.

Use of Patient-Reported Outcome Measures and Patient-Reported Experience Measures Within Evaluation Studies of Telemedicine Applications: Systematic Review.

BackgroundWith the rise of digital health technologies and telemedicine, the need for evidence-based evaluation is growing. Patient-reported outcome measures (PROMs) and patient-reported experience measures (PREMs) are recommended as an essential part of the evaluation of telemedicine. For the first time, a systematic review has been conducted to investigate the use of PROMs and PREMs in the evaluation studies of telemedicine covering all application types and medical purposes.ObjectiveThis study investigates the following research questions: in which scenarios are PROMs and PREMs collected for evaluation purposes, which PROM and PREM outcome domains have been covered and how often, which outcome measurement instruments have been used and how often, does the selection and quantity of PROMs and PREMs differ between study types and application types, and has the use of PROMs and PREMs changed over time.MethodsWe conducted a systematic literature search of the MEDLINE and Embase databases and included studies published from inception until April 2, 2020. We included studies evaluating telemedicine with patients as the main users; these studies reported PROMs and PREMs within randomized controlled trials, controlled trials, noncontrolled trials, and feasibility trials in English and German.ResultsOf the identified 2671 studies, 303 (11.34%) were included; of the 303 studies, 67 (22.1%) were feasibility studies, 70 (23.1%) were noncontrolled trials, 20 (6.6%) were controlled trials, and 146 (48.2%) were randomized controlled trials. Health-related quality of life (n=310; mean 1.02, SD 1.05), emotional function (n=244; mean 0.81, SD 1.18), and adherence (n=103; mean 0.34, SD 0.53) were the most frequently assessed outcome domains. Self-developed PROMs were used in 21.4% (65/303) of the studies, and self-developed PREMs were used in 22.3% (68/303). PROMs (n=884) were assessed more frequently than PREMs (n=234). As the evidence level of the studies increased, the number of PROMs also increased (τ=−0.45), and the number of PREMs decreased (τ=0.35). Since 2000, not only has the number of studies using PROMs and PREMs increased, but the level of evidence and the number of outcome measurement instruments used have also increased, with the number of PREMs permanently remaining at a lower level.ConclusionsThere have been increasingly more studies, particularly high-evidence studies, which use PROMs and PREMs to evaluate telemedicine. PROMs have been used more frequently than PREMs. With the increasing maturity stage of telemedicine applications and higher evidence level, the use of PROMs increased in line with the recommendations of evaluation guidelines. Health-related quality of life and emotional function were measured in almost all the studies. Simultaneously, health literacy as a precondition for using the application adequately, alongside proper training and guidance, has rarely been reported. Further efforts should be pursued to standardize PROM and PREM collection in evaluation studies of telemedicine.

Abstract 11270: Including the Patient’s Perspective in the Planning of Data Collection in the Recurrent Pericarditis Registry, RESONANCE

Introduction: In conjunction with pericardial disease physician specialists, industry experts, and recurrent pericarditis (RP) patients the RESONANCE Registry was designed to collect retrospective and prospective, longitudinal, observational data in real-world clinical practice across the United States (US). We aimed to collect data that were complete and meaningful in terms of patient experience and health related quality of life (HRQoL). Herein, we describe a novel patient-centric research approach to obtain feedback on evaluation tools for incorporation in the RESONANCE Registry (NCT04687358). Methods: Ten adult patients with a self-reported duration of disease ranging from 8 months to 25 years participated in an advisory board during which they provided feedback through a virtual platform. Their feedback informed development of multiple registry aspects. These included selection of patient reported outcomes measures (PROMs), to be collected every three months at prospective data collection timepoints, and design of several questionnaires, including evaluation of specific RP episode characteristics. Results: Prior to finalization, registry case report forms were optimized based on feedback provided by patients. Areas of data-capture informed by patient input included selection of PROMs to be assessed and patient-centric tools used for self-assessment of disease experience and HRQoL. For example, sections of a patient questionnaire were modified to include documentation of the presenting symptom, breathlessness, fear of episodes, and patient management of each episode. Conclusions: This patient-centered research collaboration resulted in enhancements to the RESONANCE Registry that enabled capture of information meaningful to patients’ daily experiences, preferences, and expectations. Patient participation in tangible aspects of research, including registry tools and design, can increase engagement with the communities that have a vested interest in improving patient care. In addition, patient involvement can help to ensure research efforts reflect the needs of not only the healthcare community but of patients alike, with an ultimate goal of improving clinical outcomes.

P06.03 Child, parent, and clinician selection of patient-reported outcome measures to use in pediatric neuro-oncology outpatient follow-up clinics

Abstract BACKGROUND Survivors of childhood brain tumours are at risk of poor health-related quality of life (HRQoL). Appropriate and relevant measures can be used to monitor HRQoL so that timely interventions may be made for issues thus identified. We had previously selected patient-reported outcome measures (PROMs) with good psychometric properties that had emerged as well-suited for this use in two systematic reviews. The aim of the present study was to ascertain the views of families regarding the suitability of the selected measures for use in paediatric neuro-oncology follow-up clinics. These views were then used to inform the choice of PROMs for use on the KLIK PROM portal, a website designed to gather HRQoL information from families. MATERIAL AND METHODS As part of the PROMOTE study, we used a multi-centre, multi-informant, cross-sectional, qualitative methods research design. Using ‘think aloud’ audio-recorded interviews, children aged 8–17 years diagnosed within the previous five years with a brain tumour, off treatment and receiving outpatient care, and their parents, were shown a total of nine PROMs and asked to express their views on which they preferred. Detailed notes were made of all audio-recordings by two independent researchers. The final choice of PROMs to be included on the KLIK PROM portal was agreed through discussion of the PROMs selected by families with an expert panel of clinicians, researchers, and parent representatives. RESULTS 16 children and 17 parents participated and of these 2 children and 2 parents did not express a preference. The Pediatric Quality of Life Inventory (PedsQL) Core module was the most popular among the children and parents with 7/14 (50%) of children selecting it as either their 1st or 2nd choice citing that they liked the questions and felt them to be most relevant to them, and 8/15 (53%) of parents citing that it was easy, quick and simple, the wording was easy, and they liked the questions about emotional, social, and school functioning. The least popular questionnaires, with no participants selecting them as first choices, were the Kidscreen-10 and the Health Utilities Index. The latter 2 were selected only as 2nd or lower choices by 1 child and 3 parents respectively. CONCLUSION The PROMs that were finally selected for the KLIK PROM portal were the parent- and child-report PedsQL-Core measures of HRQoL due to their good psychometric properties, family and clinician preference and perceived relevance to follow-up care, and for clinical utility on the KLIK PROM portal.

Can the Knee Outcome and Osteoarthritis Score (KOOS) Function Subscale Be Linked to the PROMIS Physical Function to Crosswalk Equivalent Scores?

An increased focus on patient-reported outcome measures (PROMs) has led to a proliferation of these measures in orthopaedic surgery. Mandating a single PROM in clinical and research orthopaedics is not feasible given the breadth of data already collected with older measures and the emergence of psychometrically superior measures. Creating crosswalk tables for scores between measures allows providers to maintain control of measure choice. Furthermore, crosswalk tables permit providers to compare scores collected with older outcome measures with newly collected ones. Given the widespread use of the newer Patient-reported Outcome Measure Information System Physical Function (PROMIS PF) and the established Knee Outcome and Osteoarthritis Score (KOOS), it would be clinically useful to link these two measures. Can the KOOS Function in Activities of Daily Living (ADL) subscale be robustly linked to the PROMIS PF to create a crosswalk table of equivalent scores that accurately reflects a patient's reported physical function level on both scales? We sought to establish a common standardized metric for collected responses to the PROMIS PF and the KOOS ADL to develop equations for converting a PROMIS PF score to a score for the KOOS-ADL subscale and vice versa. To do this, we performed a retrospective, observational study at two academic medical centers and two community hospitals in an urban and suburban healthcare system. Patients 18 years and older who underwent TKA were identified. Between January 2017 and July 2020, we treated 8165 patients with a TKA, 93% of whom had a diagnosis of primary osteoarthritis. Of those, we considered patients who had completed a full KOOS and PROMIS PF 10a on the same date as potentially eligible. Twenty-one percent (1708 of 8165) of patients were excluded because no PROMs were collected at any point, and another 67% (5454 of 8165) were excluded because they completed only one of the required PROMs, leaving 12% (1003 of 8165) for analysis here. PROMs were collected each time they visited the health system before and after their TKAs. Physical function was measured by the PROMIS PF version 1.0 SF 10a and KOOS ADL scale. Analyses to accurately create a crosswalk of equivalent scores between the measures were performed using the equipercentile linking method with both unsmoothed and log linear smoothed score distributions. Crosswalks were created, and adequate validation results supported their validity; we also created tables to allow clinicians and clinician scientists to convert individual patients' scores easily. The mean difference between the observed PROMIS PF scores and the scores converted by the crosswalk from the KOOS-ADL scores was -0.08 ± 4.82. A sensitivity analysis was conducted, confirming the effectiveness of these crosswalks to link the scores of two measures from patients both before and after surgery. The PROMIS PF 10a can be robustly linked to the KOOS ADL measure. The developed crosswalk table can be used to convert PROMIS PF scores from KOOS ADL and vice versa. The creation of a crosswalk table between the KOOS Function in ADL subscale and PROMIS PF allows clinicians and researchers to easily convert scores between the measures, thus permitting greater choice in PROM selection while preserving comparability between patient cohorts and PROM data collected from older outcome measures. Creating a crosswalk, or concordance table, between the two scales will facilitate this comparison, especially when pooling data for meta-analyses.