Comprehensive Patient-reported Outcome Measure Research Articles

Development of a novel Patient-Reported Outcome Measure (PROM) for adults with an Inherited Cardiac Condition (ICC)

Abstract Background Research on the quality-of-life (QoL) among patients with inherited cardiac conditions (ICC) is limited. Existing evidence from a systematic review indicates that most studies rely on generic measures of health-related QoL, which may not adequately capture ICC-specific factors. The current study aims to develop a disease-specific patient-reported outcome measure to address this gap. Methods Using a qualitative study design, we first conducted focus groups until data saturation was achieved (n=15). Additionally, an expert panel of healthcare professionals was interviewed to ascertain their perspectives on patient-centered care and to evaluate face validity. Transcripts of all focus groups and interviews were analyzed thematically using Atlas.ti, qualitative analysis software, to identify concepts and formulate survey items for the initial instrument. We used the ‘Centre for Health Promotion’ QoL model, consisting of three domains- Being, Belonging, and Becoming- as the theoretical framework for our analysis. Results Our analysis revealed alignment with specific QoL domains of the overarching framework and identified a gap in genetic-specific domains. A novel overarching theme, genetic well-being, was identified, encompassing specific constructs related to future uncertainty, reproductive concerns, and the anticipation of disease development, thus enriching the conceptualization of QoL in the context of inherited cardiac conditions. The discussion will include an exploration of the face validity of the draft instrument. Conclusion Patients diagnosed with an inherited cardiac condition (ICC) face a spectrum of challenges originating from both disease-related stressors and hereditary factors alike. Recognizing that relying solely on objective clinical assessment may fail to capture the full impact of the disease, there has been an increasing focus on incorporating patient-reported measures into disease evaluation. This qualitative study contributes to developing a comprehensive patient-reported outcome measure tailored to individuals with ICC.PROM development study

Development and validation of MyCommunication—Adults, a self‐report communicative participation measure

AbstractBackgroundPeople with communication problems experience challenges in participation. Optimizing communicative participation for this population is an important outcome of speech and language therapy. Participation experiences are best assessed from the patient's perspective, using a patient‐reported outcome measure (PROM). The Communicative Participation Item Bank (CPIB) has been developed to identify perceived problems in communicative participation. However, previous research has shown that the item bank does not cover all domains and concepts of communicative participation, contains general communication items, and is not suitable for all people with communication problems.AimsTo develop a comprehensive PROM aimed at measuring communicative participation for all adults with communication problems and test its content validity.Methods & ProceduresAn initial pool of 242 items was based on three sources: relevant content from an earlier literature review and concept elicitation study, and the translation of the items of the CPIB. This item pool was pilot‐tested for comprehensibility and comprehensiveness using cognitive debriefing interviews in adults with different communication problems (speech, language, voice, hearing and cognitive communication disorders). This resulted in a second version of the item bank. The content validity of this version was tested in a group of professionals. The last (third) version of 133 items was tested for content validity in a second, new group of adults with various communication problems.Outcomes & ResultsThe initial pool of 242 items was developed and pilot‐tested in 27 adults with different communication problems. After modifications, 161 items remained, which were tested in a content validity study with 25 people with different communication problems and five professionals (in the field of speech and language therapy and speech and language research). The professionals considered 91.9% of the item bank relevant and comprehensible, and comprehensive by adding two items. The item bank was then considered relevant, comprehensible and comprehensive by the group of adults with communication problems. A total of 133 items remained in the item bank, related to the International Classification of Functioning, Disability and Health (ICF) Activities and Participation domains ‘mobility’, ‘self‐care’, ‘domestic life’, ‘interpersonal interactions and relationships’, ‘major life areas’ and ‘community, social and civic life’.Conclusions & ImplicationsMyCommunication—Adults is an item bank for measuring communicative participation in adults with communication difficulties. The next step is to test the psychometric properties of the item bank, in order to establish a final item bank. The refined description of the construct of communicative participation can already be used for participation‐focused goal‐setting between individuals with communication problems and speech and language therapists.WHAT THIS PAPER ADDSWhat is already known on the subject The CPIB was developed to measure communicative participation in community‐dwelling adults with communication problems. The item bank does not cover all domains and concepts of communicative participation, contains general communication items and not all questions seem to be relevant for specific target groups (e.g., hearing population).What this paper adds to the existing knowledge This study presents a refined description of the construct of communicative participation and the development of a new comprehensive PROM aimed at measuring this construct for all adults with communication difficulties. The development of the PROM is based on a previously conducted literature review and concept elicitation study. This study describes the development process of this new PROM, called MyCommunication‐Adults, and the assessment of its content validity.What are the potential or actual clinical implications of this work? The refined description of the construct of communicative participation can be used for participation‐focused goal‐setting between individuals with communication problems and speech and language therapists.

Development and Validation of the Facial Line Distress Scale for Lateral Canthal Lines: FINE-LCL.

Background: Currently available patient-reported outcome measures (PROM) have limited ability to assess unique issues related to lateral canthal lines (LCL). This study aimed to develop a PROM to assess the severity and psychosocial impact of LCL in afflicted patients. Methods: We conducted a cross-sectional survey at a tertiary hospital and two local clinics in Korea. Exploratory factor analysis was conducted to identify the underlying factor structure of the Facial Line Distress Scale for LCL (FINE-LCL), and the internal consistency and test-retest reliability were also examined. Results: We developed a questionnaire comprising 20 items in four domains. Coefficient alphas ranged from 0.94 to 0.97 for subdomains and 0.97 for the total questionnaire. The test-retest intraclass correlation coefficient ranged from 0.77 to 0.90. The FINE-LCL was moderately correlated with the appearance appraisal score and body image. Analysis of the accuracy of the FINE-LCL score in predicting severe LCL was characterized by an area under the curve of 0.79. Conclusions: FINE-LCL is a reliable, valid, and comprehensive PROM for the assessment of the severity of LCL and their associated distress.

How Comprehensive and Efficient Are Patient-Reported Outcome Measures for Individuals with Lower Extremity Amputation Undergoing Implantation of Osseointegrated Bone Anchored Limbs?

» Patient-reported outcome measures (PROMs) are essential for measuring quality and functional outcomes after implantation of osseointegrated bone anchored limbs for patients with lower extremity amputation.» Using a novel assessment criterion with 8 domains, this study assessed all commonly used PROMs for their efficiency and comprehensiveness.» Comprehensiveness was scored according to the presence or absence of PROM questions related to these 8 domains (maximum score = 60): mobility (15 items), prosthesis (14 items), pain (10 items), psychosocial status (10 items), independence/self-care (4 items), quality of life/satisfaction (4 items), osseoperception (1 item), general information (1 item), and vitality (1 item).» The efficiency scores were calculated by dividing the comprehensiveness score by the total number of questions answered by the patients with higher scores being deemed more efficient.» The most comprehensive PROMs were Orthotics and Prosthetics User's Survey-Lower Extremity Functional Status (OPUS-LEFS) (score = 36), Prosthesis Evaluation Questionnaire (PEQ) (score = 31), and Questionnaire for Persons with a Transfemoral Amputation (score = 27).» The most efficient PROMs were the OPUS-LEFS (score = 1.8) and European Quality of Life (score = 1.4).

Patient-Reported Outcome Measures: State of the Art in Patient-Reported Outcome Measure Application in Lower Extremity Orthopaedics.

With an increasing shift toward a value-based and outcome-driven healthcare system, patient-reported outcome measures (PROMs) will continue to play a prominent role in assessing performance, making clinical decisions, shared decision making, and determining the comparative effectiveness of procedures such as total joint arthroplasty for lower extremity conditions, such as ankle, hip, and knee osteoarthritis. As the application of PROMs in evaluating surgical outcomes has evolved from that of a research setting to that of a clinical setting, their use in the decision-making process has become more prevalent. As a result, preoperative optimization, surgical indications, and improved outcomes after surgery have been greatly enhanced. To enable benchmarking, quality reporting, and performance measurement at an aggregate level, it is crucial to have a comprehensive PROM collection system. However, achieving this goal is contingent upon addressing the variability in reported PROMs and the patient-centered benchmarks used to analyze clinical significance.

Operationalizing PROMs at the Musculoskeletal Practice and Policy Levels.

There continues to be growing interest in patient-reported outcome measures (PROMs), especially as value-based healthcare initiatives gain more traction. Although it is well-established that PROMs can be useful in clinical research, how to operationalize PROMs in clinical care and policy initiatives remains a "work in progress." Following the design of a comprehensive PROM administration and routine collection system, orthopaedic surgeons and their patients can reap the benefits of PROMs in practice through improved shared clinical decision-making discussions at the individual patient level and closer symptom monitoring on a large scale, with improved resource allocation at the population health level. Although certain government and payer incentives exist to collect PROMs at present, it is reasonable to assume that future policy initiatives will begin to use the actual PROM scores to assess clinical outcomes. Orthopaedic surgeons with interest in this area should prioritize their involvement in policy discussion to ensure PROMs are being used appropriately in novel payment models and policy endeavors so that they are both evaluated and compensated fairly. Specifically, orthopaedic surgeons can help ensure appropriate risk adjustment of patients when this is being done. Undoubtedly, PROMs will only become a larger part of musculoskeletal care moving forward.

Psychometric evaluation of Oral Mucositis Daily Questionnaire: A cross-cultural adaptation of the Malay version in multiethnic adult autologous stem cell transplant

ObjectiveMucositis is one of the most feared side effects of cancer treatment. Psychometric analysis of a patient self-assessment score, the oral mucositis daily questionnaire in Malay (OMDQ-Mal) and its construct validity by means of confirmatory factor analysis (CFA) is lacking. This research aimed to test the validity and reliability of OMDQ-Mal. MethodsA total of 114 autologous stem-cell transplantation patients aged ≥ 18 years old at a national hematology center in Malaysia from April 2019 to December 2020 completed OMDQ-Mal concurrently with physician scores. Internal consistency and reproducibility were determined by Cronbach alpha and intraclass correlation coefficient, respectively. Correlations with physician scores were determined by Spearman correlation. Discriminative validity and construct validity were determined by Mann–Whitney U and CFA, respectively. ResultsOMDQ-Mal demonstrated high internal consistency (α ​= ​0.874). Test–retest reliability between paired days were moderate to excellent (95% CI ​= ​0.676–0.953). Items in OMDQ-Mal had moderate to strong correlations with physician scores (ρ ​= ​0.503–0.721). Discriminative validity indicated that the scores of scales were significantly different between participants with severe and mild conditions. Construct validity results of loading factors 0.708–0.952; composite reliability 0.879–0.974; average variant extracted 0.710–0.841; and heterotrait–monotrait ratio 0.528 established the convergent and divergent validity. ConclusionsIn conclusion, the OMDQ-Mal, which captured important quality of life responses, demonstrated adequate validity and reliability. This was supported by a two-component model CFA. The strong correlation of OMDQ-Mal with both physician scores indicated its potential as a comprehensive patient-reported outcome measure of mucositis of the entire alimentary tract.

Patterns of Patient-Reported Chemotherapy-Induced Peripheral Neuropathy in Colorectal Cancer Survivors.

Chemotherapy-induced peripheral neuropathy (CIPN) can be a debilitating toxicity of oxaliplatin used for treatment of colorectal cancer (CRC). We aimed to assess CIPN symptoms and associations in our colorectal survivorship population and review the impact of neurotoxicity on dose delivery of oxaliplatin. Patients attending their first visit to the Sydney Cancer Survivorship Centre following completion of adjuvant treatment for CRC completed comprehensive patient-reported outcome measures, including symptoms, quality of life (QoL), alcohol intake, and exercise habits. Participants scored symptoms of "numbness or pins and needles" in hands or feet from 0 (no trouble at all) to 10 (worst I can imagine). Diagnosis, treatment, and comorbidity details were obtained from medical records. A subset of patients completed serial assessments of PN symptoms at follow-up visits. Data were analyzed from 233 patients (52% male; mean age, 63 years) with CRC attending their first visit at the Sydney Cancer Survivorship Centre. A subset of 104 patients were included in the longitudinal analysis. The odds of patient-reported numbness were significantly higher in patients receiving oxaliplatin (odds ratio, 5.6; 95% CI, 3.2-9.8), with 72.4% of oxaliplatin-treated CRC survivors reporting numbness an average of 5.9 months after chemotherapy. Mean patient-reported numbness was significantly higher in those who received oxaliplatin-containing chemotherapy (mean, 3.31) compared with fluoropyrimidines alone (mean, 1.37) and no chemotherapy (mean, 0.66). Of the patients receiving oxaliplatin, 80% required dose reduction or early cessation, with PN the most common reason reported. QoL in physical, emotional, and functional well-being domains was lower in patients with numbness. We found a weak negative association between numbness score and age, and between (1) numbness and cardiovascular disease and (2) numbers and pain score. CIPN symptoms are common in CRC survivors who have received oxaliplatin and are associated with lower QoL. Neurotoxicity is underreported in clinical trials compared with real-world populations and is a major barrier to oxaliplatin treatment delivery.

Patient-reported outcome measures (PROMs) after laparoscopic cholecystectomy: systematic review

BackgroundHealthcare requires patient feedback to improve outcomes and experience. This study undertook a systematic review of the depth, variability, and digital suitability of current patient-reported outcome measures (PROMs) in patients undergoing laparoscopic cholecystectomy.MethodsA PROSPERO-registered (registration number CRD42021261707) systematic review was undertaken for all relevant English language articles using PubMed version of MEDLINE, Scopus, and Web of Science electronic databases in June 2021. The search used Boolean operators and wildcards and included the keywords: laparoscopic cholecystectomy AND patient outcome OR patient-reported outcome OR patient-reported outcome measure OR PRO OR PROM. Medical Subjects Heading terms were used to search PubMed and Scopus. Articles published from 1 January 2011 to 2 June 2021 were included.ResultsA total of 4960 individual articles were reviewed in this study, of which 44 were found to evaluate PROMs in patients undergoing laparoscopic cholecystectomy and underwent methodological index for non-randomized studies (MINORS) grading. Twenty-one articles spanning 19 countries and four continents met all inclusion criteria and were included in the qualitative data synthesis. There was significant heterogeneity in PROMs identified with eight different comprehensive PROM tools used in the 21 studies. There was wide variation in the time points at which PROMs were recorded. Fourteen of 21 studies recorded PROMs before and after surgery, and 7 of 21 recorded PROMs only after surgery. Follow-up intervals ranged from 3 days to 2 years after surgery.ConclusionsThis study identified that while post-laparoscopic cholecystectomy PROMs are infrequently measured currently, tools are widely available to achieve this in clinical practice. PROMs may not capture all the outcomes but should be incorporated into future cholecystectomy outcome research. The EQ-5D™ (EuroQoL Group, Rotterdam, the Netherlands) provides a simple platform for the modern digital era.

Qualitative study informing the development and content validity of the HAND-Q: a modular patient-reported outcome measure for hand conditions

ObjectivesThe purpose of this study was to identify and understand the issues that are relevant to patients with hand conditions. The data were used to develop a patient-reported outcome measure...

Living with Chronic Illness Scale: International validation through the classic test theory and Rasch analysis among Spanish-speaking populations with long-term conditions.

BackgroundThe Living with Chronic Illness (LW‐CI) Scale is a comprehensive patient‐reported outcome measure that evaluates the complex process of living with long‐term conditions.ObjectiveThis study aimed to analyse the psychometric properties of the LW‐CI scale according to the classic test theory and the Rasch model among individuals living with different long‐term conditions.DesignThis was an observational, international and cross‐sectional study.MethodsA total of 2753 people from six Spanish‐speaking countries living with type 2 diabetes mellitus, chronic obstructive pulmonary disease, chronic heart failure, Parkinson's disease, hypertension and osteoarthritis were included. The acceptability, internal consistency and validity of the LW‐CI scale were analysed using the classical test theory, and fit to the model, unidimensionality, person separation index, item local independency and differential item functioning were analysed using the Rasch model.ResultsCronbach's α for the LW‐CI scale was .91, and correlation values for all domains of the LW‐CI scale ranged from .62 to .68, except for Domain 1, which showed correlation coefficients less than .30. The LW‐CI domains showed a good fit to the Rasch model, with unidimensionality, item local independency and moderate reliability providing scores in a true interval scale. Except for two items, the LW‐CI scale was free from bias by long‐term condition type.DiscussionAfter some adjustments, the LW‐CI scale is a reliable and valid measure showing a good fit to the Rasch model and is ready for use in research and clinical practice. Future implementation studies are suggested.Patient and Public ContributionPatient and public involvement was conducted before this validation study ‐ in the pilot study phase.

Development and Psychometric Validation of a Patient-Reported Outcome Measure for Arm Lymphedema: The LYMPH-Q Upper Extremity Module

BackgroundA multiphased mixed-methods study was performed to develop and validate a comprehensive patient-reported outcome measure (PROM) for arm lymphedema in women with breast cancer (i.e., the LYMPH-Q Upper Extremity Module).MethodsQualitative interviews (January 2017 and June 2018) were performed with 15 women to elicit concepts specific to arm lymphedema after breast cancer treatment. Data were audio-recorded, transcribed, and coded. Scales were refined through cognitive interviews (October and Decemeber 2018) with 16 patients and input from 12 clinical experts. The scales were field-tested (October 2019 and January 2020) with an international sample of 3222 women in the United States and Denmark. Rasch measurement theory (RMT) analysis was used to examine reliability and validity.ResultsThe qualitative phase resulted in six independently functioning scales that measure arm symptoms, function, appearance, psychological function, and satisfaction with information and with arm sleeves. In the RMT analysis, all items in each scale had ordered thresholds and nonsignificant chi-square p values. For all the scales, the reliability statistics with and without extremes for the Person Separation Index were 0.80 or higher, Cronbach’s alpha was 0.89 or higher, and the Intraclass Correlation Coefficients were 0.92 or higher. Lower (worse) scores on the LYMPH-Q Upper Extremity scales were associated with reporting of more severe arm swelling, an arm problem caused by cancer and/or its treatment, and wearing of an arm sleeve in the past 12 months.ConclusionsThe LYMPH-Q Upper Extremity Module can be used to measure outcomes that matter to women with upper extremity lymphedema. This new PROM was designed using a modern psychometric approach and, as such, can be used in research and in clinical care.

A systematic review of English language patient-reported outcome measures for use in urogynaecology and female pelvic medicine.

Patient-reported outcome measures (PROMs) are widely used in clinical practice and research in urogynaecology. There is no consensus on which PROMs should be used. No unifying document identifies all available PROMs and compares the psychometric properties of these. Systematic review of the literature following PRISMA guidelines. Studies where women had been administered an English-language PROM which assessed pelvic-floor symptomatology and psychometric properties had been reported were included. 85 PROMs assessing pelvic-floor symptoms in a urogynaecology population were identified. 43 PROMs assessed lower urinary tract symptoms in 95 studies, four PROMS assessed vaginal symptoms in seven studies, 20 PROMs assessed bowel symptoms in 27 studies and three PROMs assessed sexual symptoms in seven studies. 15 PROMs assessed two or more of these symptom areas in 60 studies. PROMs with the with the best available psychometric evidence within these five areas were (urinary symptoms) the Incontinence Quality-of-Life questionnaire (I-QOL aka ICIQ-UIqol) and International Consultation on Incontinence Questionnaire (ICIQ-UI-SF), (bowel symptoms) the Accidental Bowel Leakage Evaluation (ABLE) questionnaire and the International Consultation on Incontinence Bowel questionnaire (ICIQ-B), (vaginal symptoms) the Pelvic Organ Prolapse Symptom Score (POPSS), (sexual symptoms) the Pelvic organ prolapse- urinary Incontinence Sexual function Questionnaire- IUGA revised (PISQ-IR) and (comprehensive PROMs) the Australian Pelvic Floor Questionnaire and the Electronic Personal Assessment Questionnaire-Pelvic-Floor (ePAQ-PF). Multiple PROMs with robust psychometric properties are available. Some widely used PROMs have weak evidence. Formal recommendations on which English-language PROMs to use within clinical practice and research in urogynaecology are required.

CORR Insights®: Mapping and Crosswalk of the Oxford Hip Score and Different Versions of the Hip Disability and Osteoarthritis Outcome Score.

CORR Insights®: Mapping and Crosswalk of the Oxford Hip Score and Different Versions of the Hip Disability and Osteoarthritis Outcome Score.

116 Cross-cultural Translation, Adaptation and Validation of the Burnt Hand Outcome Tool (BHOT) from English to French Canadian

Abstract Introduction The Burnt Hand Outcome Tool (BHOT) is a comprehensive patient-reported outcome measure to assess the multiple impacts of hand burn injuries. However, this tool is currently only available in English. The aim of this study was to create a French Canadian, cross-cultural translation and adaptation of the BHOT and to investigate its reliability and validity. Methods The BHOT was translated and culturally adapted following published good practice principles for patient-reported outcome measures. The steps included translation to French, backward translation, expert committee review, and cognitive debriefing with 5 adults having burn injuries excluding their hands. Then, 39 adults with hand burn injuries tested the pre-final French version of the questionnaire (BHOT-F) in order to determine its clinimetric properties. Reliability was investigated by determining the Cronbach’s alpha internal consistency coefficient. Construct convergent validity was assessed by comparing the BHOT-F to the shortened version of the Disabilities of the Arm, Shoulder and Hand questionnaire (QuickDASH). Content validity was evaluated based on comments extracted from interviews with the participants and a committee of burn care experts. Results The BHOT-F was modified during the adaption process to ensure its clarity. Cronbach’s alpha was 0.94 indicating excellent internal consistency and was > 0.75 for all sub-domains. The BHOT-F and the QuickDASH were strongly correlated (rs = .86; p < 0.01). Content validity was deemed satisfying. Conclusions The French-Canadian version of the BHOT is a reliable and valid tool that can confidently be used in clinical practice for adults with hand burn injuries and compared to data generated with Anglophone populations.

OP0251 THE EULAR SYSTEMIC SCLEROSIS IMPACT OF DISEASE (SCLEROID) SCORE – A NEW PATIENT-REPORTED OUTCOME MEASURE FOR PATIENTS WITH SYSTEMIC SCLEROSIS

Background:Patient reported outcome measures (PROM) are important for clinical practice and research. Given the unmet need for a comprehensive PROM for systemic sclerosis (SSc), the ScleroID questionnaire was developed by a joint team of patients with SSc and medical experts. This is intended as a brief, specific, patient-derived, disease impact score for research and clinical use in SSc.Objectives:Here, we present the validation and final version of the ScleroID.Methods:This EULAR-endorsed project involves 9 European expert SSc centers. Patients fulfilling the ACR/EULAR 2013 criteria were prospectively included since 05/16 in a large observational cohort study. Patients completed the ScleroID and comparators SHAQ, EQ5D, SF36. They also weighted the 10 dimensions of the ScleroID by distributing 100 points according to the perceived impact on their health. The final score calculation is based on the ranking of the weights. The validation study included a reliability arm and a longitudinal arm, looking at sensitivity to change at follow-up.Results:Of the 472 patients included at baseline, 109 patients also had a reliability visit and 113 patients a follow-up visit. 84.5% of patients were female, 29.8% had diffuse SSc, mean age was 54.6 years, and mean disease duration 9.5 years. The highest weights were assigned by the patients to Raynaud`s phenomenon, fatigue, hand function and pain, confirming our previous results. The total ScleroID score showed good Spearman correlation coefficients with the comparators (SHAQ, 0.73; EQ5D -0.48; Patient’s global assessment, VAS 0.77; HAQ-DI 0.62; SF36 physical score -0.62; each p<0.001). The internal consistency was good: Crohnbach’s alpha 0.866, similar to SS-HAQ (0.88) and higher than EQ5D (0.77). The ScleroID had a very good reliability: intra-class correlation coefficient 0.839 (ranging 0.608 to 0.788 for the individual items), superior to all comparators. Twenty of 113 patients reported a change in their disease status at follow up. Sensitivity to change: the standardized response mean was 0.34 for the total ScleroID score and highest for lower GI (0.633) and life choices domains (0.521), superior to all other PROM. Figure 1 shows the final ScleroID.Figure 1.Conclusion:The EULAR ScleroID is a novel PROM designed for use in clinical practice and clinical trials to reflect the disease impact of SSc, showing good performance in the validation study. Importantly, Raynaud syndrome, impaired hand function, pain and fatigue were the main patient reported drivers of disease impact.Disclosure of Interests:Mike O. Becker: None declared, Rucsandra Dobrota: None declared, Kim Fligelstone: None declared, Annelise Roennow: None declared, Yannick Allanore Grant/research support from: BMS, Inventiva, Roche, Sanofi, Consultant of: Actelion, Bayer AG, BMS, BI, Patricia Carreira Grant/research support from: Actelion, Roche, MSD, Consultant of: GlaxoSmithKline, VivaCell Biotechnology, Emerald Health Pharmaceuticals, Boehringer Ingelheim, Roche, Speakers bureau: Actelion, GlaxoSmithKline, Roche, László Czirják Consultant of: Actelion, BI, Roche-Genentech, Lilly, Medac, Novartis, Pfizer, Bayer AG, Christopher Denton Grant/research support from: GlaxoSmithKline, CSL Behring, and Inventiva, Consultant of: Medscape, Roche-Genentech, Actelion, GlaxoSmithKline, Sanofi Aventis, Inventiva, CSL Behring, Boehringer Ingelheim, Corbus Pharmaceuticals, Acceleron, Curzion and Bayer, Roger Hesselstrand: None declared, Gunnel Sandqvist: None declared, Otylia Kowal-Bielecka Consultant of: Bayer, Boehringer Ingelheim, Inventiva, MSD, Medac, Novartis, Roche and Sandoz, Speakers bureau: Bayer, Boehringer Ingelheim, Inventiva, MSD, Medac, Novartis, Roche and Sandoz, Cosimo Bruni Speakers bureau: Actelion, Eli Lilly, Marco Matucci Cerinic: None declared, Carina Mihai: None declared, Ana Maria Gheorghiu: None declared, Ulf Müller-Ladner Speakers bureau: Biogen, Joe Sexton: None declared, Turid Heiberg: None declared, Oliver Distler Grant/research support from: Grants/Research support from Actelion, Bayer, Boehringer Ingelheim, Competitive Drug Development International Ltd. and Mitsubishi Tanabe; he also holds the issued Patent on mir-29 for the treatment of systemic sclerosis (US8247389, EP2331143)., Consultant of: Consultancy fees from Actelion, Acceleron Pharma, AnaMar, Bayer, Baecon Discovery, Blade Therapeutics, Boehringer, CSL Behring, Catenion, ChemomAb, Curzion Pharmaceuticals, Ergonex, Galapagos NV, GSK, Glenmark Pharmaceuticals, Inventiva, Italfarmaco, iQvia, medac, Medscape, Mitsubishi Tanabe Pharma, MSD, Roche, Sanofi and UCB, Speakers bureau: Speaker fees from Actelion, Bayer, Boehringer Ingelheim, Medscape, Pfizer and Roche

The Patients' Perspective of Important Glucocorticoid Effects: A Nominal Group Study Among Patients With Systemic Lupus Erythematosus and Myositis.

The objective of this cohort study was to understand the positive and negative effects of glucocorticoids (GCs) in patients with systemic lupus erythematosus and myositis from the patients' perspective with the aim of developing a patient-reported outcome measure. Included patients were asked to participate in 1 of 5 nominal groups where demographic information and a quality-of-life questionnaire were collected. Patients were asked 2 open-ended questions on (1) benefits and (2) harms related to GC use. We used the Nominal Group Technique, a highly structured consensus method in which responses are generated, shared, and ranked. Descriptive statistics were used to summarize the results. Nominal group sessions took place from April to May 2019. Of 206 patients who were approached, 21 patients participated, 17 with systemic lupus erythematosus and 4 with myositis, predominantly women with more than 10 years of steroid use. The domains ranked highest for GC benefits were disease control (55 votes), fast onset of action (30 votes), increased energy (10 votes), and pain relief (10 votes). The highest-ranked negative effects were bone loss (38 votes) and weight gain (16 votes); psychological effects and damaged internal organs each received 12 votes. The top-ranked GC effects-both benefits and harms-among patients with systemic rheumatic disease are consistent with the top domains associated with GC use reported with other inflammatory diseases. This study informs the development of a comprehensive patient-reported outcome measure that can be used across inflammatory diseases.

Development and initial validation of an electronic personal assessment questionnaire for menstrual, pelvic pain and gynaecological hormonal disorders (ePAQ-MPH)

ObjectiveMenstrual disorders, pelvic-pain and gynaecological hormonal conditions in women can have a significant impact on quality-of-life. Reliable assessment and monitoring of these intimate conditions is challenging. Patient reported outcome measures (PROMs) can be invaluable in providing objective assessment, but no comprehensive PROM assessing all of these conditions and their impact on quality of life is currently available. The purpose of this study was to develop and undertake initial psychometric testing of a comprehensive interactive electronic patient reported outcome measure for these conditions. Study designA prototype electronic PROM (ePAQ-MPH) was developed following systematic literature review, semi structured interviews with 25 patients and expert panel review. Exploratory factor analysis was undertaken in 291 women attending a menstrual-disorders clinic; establishing a domain structure and enabling item reduction. Two validated PROMS (Women’s Health Questionnaire and Menstrual Distress Questionnaire) were completed to assess criterion validity in 213 patients. Test-retest reliability was carried out in 30 women completing ePAQ-MPH at least one week apart. Patients' views on ‘Value’ and ‘Burden’ were assessed in 278 women using a validated 10-item survey measuring questionnaire utility (QQ-10). Confirmatory factor analysis (CFA) of the revised version of ePAQ-MPH following item reduction was undertaken in a different sample of 254 women. ResultsExploratory factor analysis identified 18 domains (Cronbach’s α > 0.7) and 30 redundant items. Test-retest analysis found acceptable intra-class correlations of 0.6–0.9 (p < 0.05). Eight domains were compared with Menstrual Distress Questionnaire showing moderate or strong correlation in seven domains. Ten domains were compared with Women’s Health Questionnaire, six of which showed moderate correlation. Mean QQ-10 Value and Burden scores were 76 and 25, respectively (SD=15.8 and 15.5). The mean completion time for ePAQ-MPH was 31 min. CFA of the revised version 2 instrument with 15 domains showed good model fit. ConclusionsWhilst wider psychometric testing of the revised version of ePAQ-MPH is required, including in different settings and in assessments of data quality and responsiveness, initial analysis provides some evidence for reliability, validity and acceptability of this multi-dimensional electronic PROM. ePAQ-MPH shows potential for both patient assessment and roles in service evaluation and research.

Danish and Dutch linguistic validation and cultural adaptation of the WOUND-Q, a PROM for chronic wounds

Chronic wounds represent a significant problem to patients, healthcare professionals, and healthcare systems alike. The inclusion of patient-reported outcome measures (PROMs) into wound care outcome assessment will provide important information. The WOUND-Q is a new PROM developed to measure outcomes for people with chronic wounds. This study aimed to perform a linguistic validation/cultural adaption of the WOUND-Q from English into Danish and Dutch. The field-test version of the WOUND-Q is a comprehensive PROM composed of 222 items in 16 independently functioning scales. We followed the rigorous guidelines set forth by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the World Health Organization (WHO). These guidelines involved two forward translations, one back translation, an expert panel meeting, and cognitive debriefing interviews with patients. In the forward translation, the psychological function scale showed the greatest number of discrepancies from the English version. Comparison of the back translation of the Danish and Dutch versions with the English version identified 19 and 24 items respectively where the meaning had changed and required re-translation. A total of 38 cognitive debriefing interviews resulted in minor adjustments and demonstrated that the questionnaire had sufficient comprehensibility. The linguistic validation and cultural adaptation process is an essential step to adapting PROMs for use in other languages and cultures. The described method of translation and linguistic validation can be recommended for future translations of any PROM into any target language. The translation process led to conceptually equivalent Danish and Dutch versions of the WOUND-Q.Level of Evidence: Not ratable.

Patient-Reported Outcome Measures in Urethral Reconstruction.

Treatment of anterior urethral stricture disease (USD) has shifted from endoscopic approaches to urethroplasty with significantly higher success rates among reconstructive urologists. This academic stance has led to a critical evaluation of "success" and developing disease-specific instruments to assess surgical outcomes focusing on patients' satisfaction rather than the historical goal of avoiding secondary procedures. Many disease non-specific and/or non-validated patient-reported outcome measures (PROMs) have been utilized to evaluate the voiding symptoms and sexual of function of patients after urethroplasty in the literature. Urethral Stricture Surgery PROM (USS PROM) is the first validated, disease-specific PROM for anterior USD which has been designed in 2001. Urethral Stricture Symptoms and Impact Measure (USSIM) is a comprehensive PROM and is currently being validated at multiple institutions. This article reviews the tools used to assess success after urethroplasty and elaborates the need to develop a comprehensive USD-specific PROM.