Patient-reported outcome measures for systemic lupus erythematosus clinical trials: a review of content validity, face validity and psychometric performance

Objective Definitions of remission in systemic lupus erythematosus (SLE; DORIS (1A/1B/2A/2B)), disease activity assessments and patient-reported outcome measures (PROMs) are useful in shared decision making between patients with SLE and physicians. We used longitudinal registry data from well-characterized Swedish patients with recent-onset SLE to explore potential correlations between DORIS status or disease activity, and PROMs. Methods Patients from the Clinical Lupus Register in North-Eastern Gothia, Sweden, who fulfilled the 1982 American College of Rheumatology and/or the 2012 Systemic Lupus International Collaborating Clinics classification criteria without prior organ damage, were enrolled at diagnosis. Data on treatments, serology, remission status (DORIS), disease activity (SLE Disease Activity Index-2000 (SLEDAI-2K)) and PROMs (quality of life: EuroQoL-5 Dimensions (EQ-5D); pain intensity, fatigue and well-being: visual analog scale (VAS) 0–100 mm) were collected during rheumatology clinic visits at months 0 (diagnosis), 6, 12, 24, 36, 48 and 60. Correlations were assessed using Pearson correlation and/or beta regression coefficients. Results A total of 41 patients were enrolled (median age = 39 years, 80% female, 85% white). Achievement of DORIS 1A and 2A (neither of which includes serology) significantly correlated with all PROMs (EQ-5D: p ≤ 0.02; pain: p = 0.0001; fatigue: p = 0.0051; well-being: p < 0.0001). Disease activity measures were correlated with VAS pain intensity ( p < 0.03) and VAS well-being ( p < 0.04). Conclusions Our findings illustrate the importance of the interplay between remission, disease activity assessments and PROMs. PROMs may be a useful tool in clinical practice, being administered prior to patient visits to streamline clinical care.

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52528 Implementation of Web-Based Patient-Reported Outcome Measures (PROMs) in the Clinical Care of Systemic Lupus Erythematosus (SLE): A Multi-Center Prospective Cohort Study

Journal of Clinical and Translational Science ◽

10.1017/cts.2021.770 ◽

2021 ◽

pp. 1-2

Author(s):

Shanthini Kasturi ◽

Lori Lyn Price ◽

David Curtis ◽

W. Benjamin Nowell ◽

Norma Terrin ◽

...

Keyword(s):

Systemic Lupus Erythematosus ◽

Cohort Study ◽

Lupus Erythematosus ◽

Prospective Cohort ◽

Clinical Care ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Systemic Lupus ◽

Web Based ◽

Patient Reported

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Patient-reported outcome measures in systemic lupus erythematosus by a web-based application: A randomized, crossover, agreement study

Lupus ◽

10.1177/09612033211051641 ◽

2021 ◽

pp. 096120332110516

Author(s):

Line Uhrenholt ◽

Simone Høstgaard ◽

Julie F Pedersen ◽

Robin Christensen ◽

Lene Dreyer ◽

...

Keyword(s):

Systemic Lupus Erythematosus ◽

Global Health ◽

Lupus Erythematosus ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Systemic Lupus ◽

Patient Reported ◽

Web App ◽

The Difference ◽

The Web

Objectives Patient-reported outcome measures (PROMs) are evaluated in randomized controlled trials (RCTs) in patients with systemic lupus erythematosus (SLE), but not widely used in clinical practice. However, interest in incorporating PROMs into the management of SLE is increasing as PROMs provide a unique insight into the patient’s perception of lupus disease activity. The objective was to assess agreement in PROMs answered using a web app versus an outpatient touchscreen among patients with SLE. Methods In a crossover RCT, SLE patients answered the following PROMs in a random order using the web app and the outpatient touchscreen: Systemic Lupus Erythematosus Activity Questionnaire (SLAQ) Global Health, SLAQ Symptom, SLAQ Total, SLAQ Worsening, Pain Visual Analog Scale (VAS), Fatigue VAS, Patient Global Health VAS, Health Assessment Questionnaire Disability Index (HAQ-DI), Patient Acceptable Symptom State (PASS), and an Anchoring Question. Equivalence between the two device types was demonstrated if the 95% confidence interval (95% CI) of the difference in PROM scores was within the prespecified equivalence margin. Agreement between the two device types was assessed using mixed linear models. Results Thirty-four patients with SLE were included. Equivalence was demonstrated between the two device types for SLAQ Global Health with a difference of −0.21 (95% CI: −0.65 to 0.23). Moreover, equivalence was also found for HAQ-DI, Pain VAS, and Fatigue VAS whereas only comparability within the limits of the Minimal Clinically Important Difference (MCID) was demonstrated for VAS Patient Global Health. Statistical comparability was demonstrated for SLAQ Total, SLAQ Worsening, PASS, and Anchoring Question (no predefined MCID/equivalence margins available). However, a statistically significant difference between device types was observed for the SLAQ Symptom of −0.56 (95% CI: −1.10 to −0.01). The difference was, however, very small when considering the scale range of 0–24; thus, it was not judged to be of clinical relevance. Preference for the web app was very high (91.2%). Conclusion For the first time ever, equivalence and comparability between two electronic device types for various PROMs were demonstrated among patients with SLE. Implementation of the device is expected to improve the management of SLE.

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Enabling patient-reported outcome measures in clinical trials, exemplified by cardiovascular trials

Health and Quality of Life Outcomes ◽

10.1186/s12955-021-01800-1 ◽

2021 ◽

Vol 19 (1) ◽

Author(s):

Theresa M. Coles ◽

Adrian F. Hernandez ◽

Bryce B. Reeve ◽

Karon Cook ◽

Michael C. Edwards ◽

...

Keyword(s):

Clinical Trials ◽

Outcome Measures ◽

Contextual Factors ◽

The United States ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Think Tank ◽

Level Of Evidence ◽

Patient Reported ◽

Evidentiary Standards

Abstract Objectives There has been limited success in achieving integration of patient-reported outcomes (PROs) in clinical trials. We describe how stakeholders envision a solution to this challenge. Methods Stakeholders from academia, industry, non-profits, insurers, clinicians, and the Food and Drug Administration convened at a Think Tank meeting funded by the Duke Clinical Research Institute to discuss the challenges of incorporating PROs into clinical trials and how to address those challenges. Using examples from cardiovascular trials, this article describes a potential path forward with a focus on applications in the United States. Results Think Tank members identified one key challenge: a common understanding of the level of evidence that is necessary to support patient-reported outcome measures (PROMs) in trials. Think Tank participants discussed the possibility of creating general evidentiary standards depending upon contextual factors, but such guidelines could not be feasibly developed because many contextual factors are at play. The attendees posited that a more informative approach to PROM evidentiary standards would be to develop validity arguments akin to courtroom briefs, which would emphasize a compelling rationale (interpretation/use argument) to support a PROM within a specific context. Participants envisioned a future in which validity arguments would be publicly available via a repository, which would be indexed by contextual factors, clinical populations, and types of claims. Conclusions A publicly available repository would help stakeholders better understand what a community believes constitutes compelling support for a specific PROM in a trial. Our proposed strategy is expected to facilitate the incorporation of PROMs into cardiovascular clinical trials and trials in general.

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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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