Development and validation of a patient-reported outcome measure for systemic sclerosis: the EULAR Systemic Sclerosis Impact of Disease (ScleroID) questionnaire

ObjectivesPatient-reported outcome measures (PROMs) are important for clinical practice and research. Given the high unmet need, our aim was to develop a comprehensive PROM for systemic sclerosis (SSc), jointly with patient experts.MethodsThis European Alliance of Associations for Rheumatology (EULAR)-endorsed project involved 11 European SSc centres. Relevant health dimensions were chosen and prioritised by patients. The resulting Systemic Sclerosis Impact of Disease (ScleroID) questionnaire was subsequently weighted and validated by Outcome Measures in Rheumatology criteria in an observational cohort study, cross-sectionally and longitudinally. As comparators, SSc-Health Assessment Questionnaire (HAQ), EuroQol Five Dimensional (EQ-5D), Short Form-36 (SF-36) were included.ResultsInitially, 17 health dimensions were selected and prioritised. The top 10 health dimensions were selected for the ScleroID questionnaire. Importantly, Raynaud’s phenomenon, impaired hand function, pain and fatigue had the highest patient-reported disease impact. The validation cohort study included 472 patients with a baseline visit, from which 109 had a test–retest reliability visit and 113 had a follow-up visit (85% female, 38% diffuse SSc, mean age 58 years, mean disease duration 9 years). The total ScleroID score showed strong Pearson correlation coefficients with comparators (SSc-HAQ, 0.73; Patient’s global assessment, Visual Analogue Scale 0.77; HAQ-Disability Index, 0.62; SF-36 physical score, −0.62; each p<0.001). The internal consistency was strong: Cronbach’s alpha was 0.87, similar to SSc-HAQ (0.88) and higher than EQ-5D (0.77). The ScleroID had excellent reliability and good sensitivity to change, superior to all comparators (intraclass correlation coefficient 0.84; standardised response mean 0.57).ConclusionsWe have developed and validated the EULAR ScleroID, which is a novel, brief, disease-specific, patient-derived, disease impact PROM, suitable for research and clinical use in SSc.

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Assessment of computer adaptive testing version of the Neuro-QOL for people with multiple sclerosis

Multiple Sclerosis Journal ◽

10.1177/1352458518810159 ◽

2018 ◽

Vol 25 (13) ◽

pp. 1791-1799 ◽

Cited By ~ 1

Author(s):

Brian C Healy ◽

Jonathan Zurawski ◽

Cindy T Gonzalez ◽

Tanuja Chitnis ◽

Howard L Weiner ◽

...

Keyword(s):

Multiple Sclerosis ◽

Outcome Measures ◽

Computerized Adaptive Testing ◽

Short Form ◽

Patient Reported Outcome Measures ◽

Adaptive Testing ◽

Patient Reported Outcome ◽

Longitudinal Change ◽

Sf 36 ◽

Patient Reported

Background: To date, the computerized adaptive testing (CAT) version of the Neuro-quality of life (QOL) has not been assessed in a large sample of people with multiple sclerosis (MS). Objective: The aim of this study was to assess the associations between the CAT version of Neuro-QOL and other clinical and patient-reported outcome measures. Methods: Subjects ( n = 364) enrolled in SysteMS completed the CAT version of the Neuro-QOL and the 36-Item Short Form Survey (SF-36) within 4 weeks of a clinical exam that included the Multiple Sclerosis Functional Composite-4 (MSFC-4). The correlations between the Neuro-QOL domains and the MSFC-4 subscores and the SF-36 scores were calculated. The changes over time in the Neuro-QOL and other measures were also examined. Results: The lower extremity functioning score of the Neuro-QOL showed the highest correlations with MSFC-4 components including Timed 25-Foot Walk, 9-Hole Peg Test, and cognitive score. The expected domains of the Neuro-QOL showed high correlations with the SF-36 subscores, and some Neuro-QOL domains were associated with many SF-36 subscores. There was limited longitudinal change on the Neuro-QOL domains over 12 months, and the change was not associated with change on other measures. Conclusion: The CAT version of the Neuro-QOL shows many of the expected associations with clinical and patient-reported outcome measures.

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Relationship of Preoperative Patient-Reported Outcome Measures (PROMs) to Postoperative Success in End-Stage Ankle Arthritis

Foot & Ankle International ◽

10.1177/1071100719893334 ◽

2019 ◽

Vol 41 (3) ◽

pp. 253-258 ◽

Cited By ~ 2

Author(s):

Feras J. Waly ◽

Eng Meng Nicholas Yeo ◽

Kevin J. Wing ◽

Murray J. Penner ◽

Andrea Veljkovic ◽

...

Keyword(s):

Outcome Measures ◽

Short Form ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Ankle Arthritis ◽

Ankle Osteoarthritis ◽

Sf 36 ◽

Patient Reported ◽

End Stage ◽

Preoperative Patient

Background: Ankle osteoarthritis is a significant cause of pain and disability. Despite the effectiveness of treatments, a subset of patients remains with persistent pain and functional limitations. The purpose of this study was to use preoperative patient-reported outcome measures to predict which ankle osteoarthritis patients would be most likely to experience postoperative improvements in functional outcomes. Methods: A retrospective analysis of prospectively collected data was used to evaluate 427 patients with end-stage ankle arthritis with 5-year follow-up. Demographics, comorbidities, Ankle Osteoarthritis Scale (AOS), Ankle Arthritis Score (AAS), and the physical and mental component scores of the Short Form–36 (SF-36 Physical Components Score [PCS] and Mental Components Score [MCS]) were collected. The minimal clinically important difference (MCID) was then calculated. Receiver operating characteristic (ROC) analysis was used to choose the optimal threshold values of preoperative patient-reported outcome measure and assess the prediction performance. Results: Patients who scored worst at preoperative baseline made the greatest gains in function and pain relief, and they had a high chance of achieving MCID following surgical treatment. ROC curves demonstrated that preoperative AOS, AAS, and SF-36 PCS and MCS scores were predictive of postoperative improvements in physical and mental function. Patients with preoperative AOS score above 45.7 had an 83% probability of achieving an MCID (AUC = 0.67). Similarly, patients with a preoperative AAS score above 25.7 had a 78% probability of achieving an MCID (AUC = 0.63). Patients with a preoperative SF-36 PCS score below 31 had a 62% probability of achieving an MCID (AUC = 0.64). Patients with a preoperative SF-36 MCS below 52.7 had a 47% probability of achieving an MCID (AUC = 0.89). MCIDs for AOS, AAS, SF3-36 PCS, and SF-36 MCS score changes were 12.4, 10.0, 6.43, and 8.1, respectively. Conclusion: Preoperative patient-reported outcomes measures could predict postoperative improvement in ankle arthritis patients. The results of this study may be used to facilitate discussion between physicians and patients regarding the expected benefit of surgery. Level of Evidence: Level III, prognostic comparative study.

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Use and detailed metric properties of patient-reported outcome measures for rheumatoid arthritis: a systematic review covering two decades

RMD Open ◽

10.1136/rmdopen-2021-001707 ◽

2021 ◽

Vol 7 (2) ◽

pp. e001707

Author(s):

Ayşe A Küçükdeveci ◽

Atilla H Elhan ◽

Beyza D Erdoğan ◽

Şehim Kutlay ◽

Derya Gökmen ◽

...

Keyword(s):

Rheumatoid Arthritis ◽

Outcome Measures ◽

Physical Functioning ◽

Short Form ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Personal Factors ◽

Sf 36 ◽

Patient Reported ◽

Psychometric Evidence

IntroductionThe importance of patient-reported outcome measures (PROMs) for rheumatoid arthritis (RA) clinical studies has been recognised for many years. The current study aims to describe the RA PROMs used over the past 20 years, and their performance metrics, to underpin appropriate tool selection.MethodsThe study included a systematic search for PROMs that have been in use over the period 2000–2019, with detailed documentation of their psychometric properties, and a user-friendly presentation of the extensive evidence base.Results125 PROMs were identified with psychometric evidence available. The domains of pain, fatigue, emotional functions, mobility, physical functioning and work dominated, with self-efficacy and coping as personal factors. Domains such as stiffness and sleep were poorly served. The most frequently used PROMs included the Health Assessment Questionnaire Disability Index (HAQ), the Short Form 36 (SF-36), the EuroQoL and the Modified HAQ which, between them, appeared in more than 3500 papers. Strong psychometric evidence was found for the HAQ, and the SF-36 Physical Functioning and Vitality (fatigue) domains. Otherwise, all domains except stiffness, sleep, education and health utility, had at least one PROM with moderate level of psychometric evidence.ConclusionThere is a broad range of PROMs for measuring RA outcomes, but the quality of psychometric evidence varies widely. This work identifies gaps in key RA domains according to the biopsychosocial model.

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Patient-Reported Outcome Measures in Colorectal Surgery: Construction of Core Measures Using Open-Source Research Method

Surgical Innovation ◽

10.1177/1553350621998871 ◽

2021 ◽

pp. 155335062199887

Author(s):

Alaa El-Hussuna ◽

Ines Rubio-Perez ◽

Monica Millan ◽

Gianluca Pellino ◽

Ionut Negoi ◽

...

Keyword(s):

Colorectal Surgery ◽

Outcome Measures ◽

Short Form ◽

Wide Spectrum ◽

Core Outcome Set ◽

Well Being ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Patient Reported ◽

Eortc Qlq

Purpose. The primary aim of the study was to review the existing literature about patient-reported outcome measures (PROMs) in colorectal cancer and IBD. The secondary aim was to present a road map to develop a core outcome set via opinion gathering using social media. Method. This study is the first step of a three-step project aimed at constructing simple, applicable PROMs in colorectal surgery. This article was written in a collaborative manner with authors invited both through Twitter via the #OpenSourceResearch hashtag. The 5 most used PROMs were presented and discussed as slides/images on Twitter. Inputs from a wide spectrum of participants including researchers, surgeons, physicians, nurses, patients, and patients’ organizations were collected and analyzed. The final draft was emailed to all contributors and 6 patients’ representatives for proofreading and approval. Results. Five PROM sets were identified and discussed: EORTC QLQ-CR29, IBDQ short health questionnaire, EORTC QLQ-C30, ED-Q5-5L, and Short Form-36. There were 315 tweets posted by 50 tweeters with 1458 retweets. Awareness about PROMs was generally limited. The general psycho-physical well-being score (GPP) was suggested and discussed, and then a survey was conducted in which more than 2/3 of voters agreed that GPP covers the most important aspects in PROMs. Conclusion. Despite the limitations of this exploratory study, it offered a new method to conduct clinical research with opportunity to engage patients. The general psycho-physical well-being score suggested as simple, applicable PROMs to be eventually combined procedure-specific, disease-specific, or symptom-specific PROMs if needed.

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Patient-reported outcome measures in community-acquired pneumonia: a systematic review of application and content validity

BMJ Open Respiratory Research ◽

10.1136/bmjresp-2018-000398 ◽

2019 ◽

Vol 6 (1) ◽

pp. e000398 ◽

Cited By ~ 1

Author(s):

Melanie Lloyd ◽

Emily Callander ◽

Amalia Karahalios ◽

Lucy Desmond ◽

Harin Karunajeewa

Keyword(s):

Outcome Measures ◽

Content Validity ◽

Short Form ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Community Acquired Pneumonia ◽

Current Evidence ◽

Measurement Instruments ◽

Health Measurement ◽

Patient Reported

IntroductionPatient-reported outcome measures (PROMs) are a vital component of patient-centred care. Community-acquired pneumonia (CAP) is a significant contributor to morbidity, mortality and health service costs globally, but there is a lack of consensus regarding PROMs for this condition.MethodsWe searched MEDLINE, EMBASE and Cochrane Collaboration for studies, both interventional and observational, of adult recovery from CAP that applied at least one validated PROM instrument and were published before 31 December 2017. The full text of included studies was examined and data collected on study design, PROM instruments applied, constructs examined and the demographic characteristics of the populations measured. For all CAP-specific PROM instruments identified, content validity was assessed using the COnsensus based Standards for selection of health Measurement INstruments guidelines (COSMIN).ResultsForty-two articles met the inclusion criteria and applied a total of 17 different PROM instruments including five (30%) classified as CAP specific, six (35%) as generic and six (35%) that measured functional performance or were specific to another disease. The 36-Item Short Form Survey (SF-36) was the most commonly used instrument (15 articles). Only one of 11 (9%) patient cohorts assessed using a CAP-specific instrument had a mean age ≥70 years. The CAP-Sym and CAP-BIQ questionnaires had sufficient content validity, though the quality of evidence for all CAP-specific instruments was rated as very low to low.DiscussionPROM instruments used to measure recovery from CAP are inconsistent in constructs measured and have frequently been developed and validated in highly selective patient samples that are not fully representative of the hospitalised CAP population. The overall content validity of all available CAP-specific instruments is unclear, particularly in the context of elderly hospitalised populations. Based on current evidence, generic health instruments are likely to be of greater value for measuring recovery from CAP in this group.

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Development of a crosswalk for the bidirectional mapping of two commonly used condition-specific patient-reported outcome measures, the oswestry disability index (ODI) and the core outcome measures index (COMI)

Brain and Spine ◽

10.1016/j.bas.2021.100042 ◽

2021 ◽

Vol 1 ◽

pp. 100042

Author(s):

Anne F. Mannion ◽

Achim Elfering ◽

Frank S. Kleinstück ◽

Markus Loibl ◽

Tamás Fekete ◽

...

Keyword(s):

Outcome Measures ◽

Oswestry Disability Index ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Core Outcome ◽

Specific Patient ◽

The Core ◽

Patient Reported ◽

Core Outcome Measures ◽

Bidirectional Mapping

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Development and Validation of a Novel Knee-Specific Patient-Reported Outcomes Measure

Journal of Sport Rehabilitation ◽

10.1123/jsr.2019-0264 ◽

2020 ◽

pp. 1-11

Author(s):

James L. Farnsworth ◽

Todd Evans ◽

Helen Binkley ◽

Minsoo Kang

Keyword(s):

Outcome Measures ◽

Patient Reported Outcomes ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Measurement Properties ◽

Partial Credit Model ◽

Partial Credit ◽

Specific Patient ◽

Patient Reported ◽

Full Study

Context: Previous research suggests that several knee-specific patient-reported outcome measures have poor measurement properties. The patient-reported outcomes knee assessment tool (PROKAT) was created to improve assessment of knee-specific function. Examination of the measurement properties of this new measure is critical to determine its clinical value. Objective: Examine the measurement properties of the PROKAT. Design: Cross-sectional study. Setting: Clinical athletic training setting. Patients or Other Participants: The pilot study included 32 student-athletes (mean age = 20.78 [1.01], males = 56.30%). The full study included 203 student-athletes (mean age = 21.46 [4.64], males = 54.70%) from 3 separate institutions. The participants were recruited for both the pilot and full study using face-to-face and electronic (eg, email and social media sites) communications. Intervention(s): Evaluation of the measurement properties of the PROKAT occurred using the Rasch partial-credit model. Main Outcome Measures: Infit and outfit statistics, item step difficulties, person ability parameters, category function, item and test information functions, and Cronbach alpha. An independent samples t test was used to evaluate the differences in injured and noninjured athletes’ scores. Results: The Rasch partial-credit model analysis of pilot test items and qualitative participant feedback were used to modify the initial PROKAT. Evaluation of the revised PROKAT (32 items) indicated 27 items had acceptable model–data fit. The injured athletes scored significantly worse than the noninjured athletes (t188 = 12.89; P < .01). The ceiling effects for the PROKAT were minimal (3.9%). Conclusions: A major advantage of this study was the use of the Rasch measurement and the targeted population. Compared with alternative knee-specific patient-reported outcome measures (eg, Knee Injury Osteoarthritis Outcome Score, International Knee Documentation Committee Subjective Knee Form), the PROKAT has low ceiling effects in athletic populations. In addition, evidence suggests the measure may be capable of distinguishing between injured and noninjured athletes.

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