International patient and physician consensus on a psoriatic arthritis core outcome set for clinical trials
Johns Hopkins University School of Medicine1, VU University Amsterdam2, University of Washington3, University of Pennsylvania4, University of Paris5, Singapore General Hospital6, Royal National Hospital for Rheumatic Diseases7, University College Dublin8, University of Utah9, University of Copenhagen10, University of Leeds11, University of the West of England12, University of Toronto13, Duke University14, Johns Hopkins University15, Hacettepe University16, Ottawa Hospital Research Institute17, Stanford University18
TL;DR: The updated PsA core domain set incorporates patients' and physicians' priorities and evolving PsA research and next steps include identifying outcome measures that adequately assess these domains.
Abstract: Objective To identify a core set of domains (outcomes) to be measured in psoriatic arthritis (PsA) clinical trials that represent both patients9 and physicians9 priorities. Methods We conducted (1) a systematic literature review (SLR) of domains assessed in PsA; (2) international focus groups to identify domains important to people with PsA; (3) two international surveys with patients and physicians to prioritise domains; (4) an international face-to-face meeting with patients and physicians using the nominal group technique method to agree on the most important domains; and (5) presentation and votes at the Outcome Measures in Rheumatology (OMERACT) conference in May 2016. All phases were performed in collaboration with patient research partners. Results We identified 39 unique domains through the SLR (24 domains) and international focus groups (34 domains). 50 patients and 75 physicians rated domain importance. During the March 2016 consensus meeting, 12 patients and 12 physicians agreed on 10 candidate domains. Then, 49 patients and 71 physicians rated these domains9 importance. Five were important to >70% of both groups: musculoskeletal disease activity, skin disease activity, structural damage, pain and physical function. Fatigue and participation were important to >70% of patients. Patient global and systemic inflammation were important to >70% of physicians. The updated PsA core domain set endorsed by 90% of OMERACT 2016 participants includes musculoskeletal disease activity, skin disease activity, pain, patient global, physical function, health-related quality of life, fatigue and systemic inflammation. Conclusions The updated PsA core domain set incorporates patients9 and physicians9 priorities and evolving PsA research. Next steps include identifying outcome measures that adequately assess these domains.
Citations
More filters
TL;DR: The updated recommendations provide stakeholders with an updated consensus on the pharmacological management of PsA, based on a combination of evidence and expert opinion.
Abstract: Objective To update the European League Against Rheumatism (EULAR) recommendations for the pharmacological treatment of psoriatic arthritis (PsA). Methods According to the EULAR standardised operating procedures, a systematic literature review was followed by a consensus meeting to develop this update involving 28 international taskforce members in May 2019. Levels of evidence and strengths of recommendations were determined. Results The updated recommendations comprise 6 overarching principles and 12 recommendations. The overarching principles address the nature of PsA and diversity of both musculoskeletal and non-musculoskeletal manifestations; the need for collaborative management and shared decision-making is highlighted. The recommendations provide a treatment strategy for pharmacological therapies. Non-steroidal anti-inflammatory drugs and local glucocorticoid injections are proposed as initial therapy; for patients with arthritis and poor prognostic factors, such as polyarthritis or monoarthritis/oligoarthritis accompanied by factors such as dactylitis or joint damage, rapid initiation of conventional synthetic disease-modifying antirheumatic drugs is recommended. If the treatment target is not achieved with this strategy, a biological disease-modifying antirheumatic drugs (bDMARDs) targeting tumour necrosis factor (TNF), interleukin (IL)-17A or IL-12/23 should be initiated, taking into account skin involvement if relevant. If axial disease predominates, a TNF inhibitor or IL-17A inhibitor should be started as first-line disease-modifying antirheumatic drug. Use of Janus kinase inhibitors is addressed primarily after bDMARD failure. Phosphodiesterase-4 inhibition is proposed for patients in whom these other drugs are inappropriate, generally in the context of mild disease. Drug switches and tapering in sustained remission are addressed. Conclusion These recommendations provide stakeholders with an updated consensus on the pharmacological management of PsA, based on a combination of evidence and expert opinion.
492 citations
University of Oxford1, University College Dublin2, Brigham and Women's Hospital3, Medical University of Vienna4, University of Amsterdam5, Geneva College6, University of Utah7, University Health Network8, VU University Amsterdam9, New York Medical College10, University of California, San Diego11, Copenhagen University Hospital12, University of Bath13, University of Washington14, University of Queensland15, University of Pennsylvania16, Stanford University17, Royal National Hospital for Rheumatic Diseases18, University of Leeds19
TL;DR: A meeting was convened by the Group for Research and Assessment of Psoriasis and Psoriatic Arthritis (GRAPPA) and Outcome Measures in Rheumatology (OMERACT) to further the development of consensus among physicians and patients regarding composite disease activity measures and targets in psoriatic arthritis (PsA).
Abstract: Objective
A meeting was convened by the Group for Research and Assessment of Psoriasis and Psoriatic Arthritis (GRAPPA) and Outcome Measures in Rheumatology (OMERACT) to further the development of consensus among physicians and patients regarding composite disease activity measures and targets in psoriatic arthritis (PsA).
Methods
Prior to the meeting, physicians and patients completed surveys on outcome measures. A consensus meeting of 26 rheumatologists, dermatologists, and patient research partners reviewed evidence on composite measures and potential treatment targets plus results of the surveys. The meeting consisted of plenary presentations, breakout sessions, and group discussions. International experts including members of GRAPPA and OMERACT were invited to the meeting, including the developers of all of the measures discussed. After discussions, participants voted on proposals for use, and consensus was established in a second survey.
Results
Survey results from 128 health care professionals and 139 patients were analyzed alongside a systematic literature review summarizing evidence. A weighted vote was cast for composite measures. For randomized controlled trials, the most popular measures were the PsA disease activity score (40 votes) and the GRAPPA composite index (28 votes). For clinical practice, the most popular measures were an average of scores on 3 visual analog scales (45 votes) and the disease activity in PsA score (26 votes). After discussion, there was no consensus on a composite measure. The group agreed that several composite measures could be used and that future studies should allow further validation and comparison. The group unanimously agreed that remission should be the ideal target, with minimal disease activity (MDA)/low disease activity as a feasible alternative. The target should include assessment of musculoskeletal disease, skin disease, and health‐related quality of life. The group recommended a treatment target of very low disease activity (VLDA) or MDA.
Conclusion
Consensus was not reached on a continuous measure of disease activity. In the interim, the group recommended several composites. Consensus was reached on a treatment target of VLDA/MDA. An extensive research agenda was composed and recommends that data on all PsA clinical domains be collected in ongoing studies.
174 citations
Cites background from "International patient and physician..."
...In 2016, a new core set of outcome measures for psoriatic arthritis (PsA) was developed by the Group for Research and Assessment of Psoriasis and Psoriatic Arthritis (GRAPPA) and endorsed by the Outcome Measures in Rheumatology (OMERACT) group (1)....
[...]
TL;DR: This Review addresses the current state of knowledge concerning axial psoriatic arthritis and proposes a research agenda to capture all available comparisons made to date and highlight the similarities and differences between AS and axial PsA.
Abstract: Ankylosing spondylitis (AS) was first identified in the late 17th century. 250 years later, inflammatory spine disease was recognized to be one of the patterns of psoriatic arthritis (PsA). Isolated spondylitis is rare among patients with PsA, occurring in less than 5% of patients; however, many patients with PsA have axial disease that is concurrent with peripheral arthritis. At the other end of the spondyloarthritis spectrum, psoriasis is observed in 10% of patients with AS. Although axial involvement in PsA can be indistinguishable from axial disease in AS, it can also differ in several respects, raising the question of whether axial PsA and AS (with or without psoriasis) are different clinical presentations of the same disease, or whether they are separate diseases that have overlapping features. In this Review, the clinical presentation, metrology, radiographic characteristics, genetic factors, treatment options and axial prognosis of the two diseases are addressed. The aim of this Review is to capture all available comparisons made to date, to highlight the similarities and differences between AS and axial PsA and to propose a research agenda.
130 citations
TL;DR: Current knowledge on impairments of health-related quality of life related to PsA, as well as patient priorities and patient-reported outcome measures such as the PsA Impact of Disease or thePsA Quality of Life questionnaires are reviewed.
Abstract: Psoriatic arthritis (PsA) is a multi-organ chronic inflammatory disease which impacts patients both physically and psychologically. The highest priority for patients goes to pain relief, but also to ability to function and participate in social life, fatigue, and psychological distress. Areas covered: In the present article, we will review current knowledge on impairments of health-related quality of life related to PsA, as well as patient priorities and patient-reported outcome measures such as the PsA Impact of Disease or the PsA Quality of Life questionnaires. The impact of PsA appears to be very broad, covering all aspects of life, i.e. activities and participation, physical and emotional aspects, but also domains such as fatigue, coping or sleep disturbance. Some of these aspects which are important for PsA patients have been included in the recently updated PsA Core Domain Set to be reported in clinical studies. Expert commentary: A better understanding of quality of life issues faced by patients with PsA could improve patient-physician communication and ultimately, quality of care. QoL is altered in PsA due both to the physical impact, but also the psychological impact of this disease. Several scores are available to better assess these aspects of PsA.
106 citations
Cites background or methods from "International patient and physician..."
...(OMERACT) group, both in 2006 [15] and 2016 [16]....
[...]
...the Core Domain Set development for PsA [16]....
[...]
...Further on, the domains identified through the international focus groups and the systematic literature review were rated for importance by both patients and physicians in web-based surveys, followed by a face-to-face nominal group discussion including again PRPs and clinicians from around the world, resulting in the final list of domains considerend important to be measured in PsA studies [16]....
[...]
...The recognition of the importance of fatigue translated consequently into including it in the inner circle of the core domain set, making it mandatory to evaluate in all PsA studies [16]....
[...]
...In the qualitative study preceding the development of the GRAPPA/OMERACT updated PsA Core Domain Set [16] fatigue was considered a critical element of life impact by PsA patients, along with participation and emotional well-being, i....
[...]
Johns Hopkins University1, VU University Amsterdam2, University of Washington3, University of Utah4, University College Dublin5, Cleveland Clinic6, University of Pennsylvania7, University Health Network8, University of Toronto9, Duke University10, Royal National Hospital for Rheumatic Diseases11, University of Copenhagen12, Singapore General Hospital13, National Health Service14, Stanford University15, Leiden University16, University of Ottawa17, University of Leeds18, University of Paris19, Hacettepe University20
TL;DR: The updated PsA Core Domain Set endorsed with 90% agreement by OMERACT 2016 participants included musculoskeletal disease activity, skin disease activity), fatigue, pain, patient’s global assessment, physical function, health-related quality of life, and systemic inflammation, which were recommended for all RCT and LOS.
Abstract: Objective To include the patient perspective in accordance with the Outcome Measures in Rheumatology (OMERACT) Filter 2.0 in the updated Psoriatic Arthritis (PsA) Core Domain Set for randomized controlled trials (RCT) and longitudinal observational studies (LOS). Methods At OMERACT 2016, research conducted to update the PsA Core Domain Set was presented and discussed in breakout groups. The updated PsA Core Domain Set was voted on and endorsed by OMERACT participants. Results We conducted a systematic literature review of domains measured in PsA RCT and LOS, and identified 24 domains. We conducted 24 focus groups with 130 patients from 7 countries representing 5 continents to identify patient domains. We achieved consensus through 2 rounds of separate surveys with 50 patients and 75 physicians, and a nominal group technique meeting with 12 patients and 12 physicians. We conducted a workshop and breakout groups at OMERACT 2016 in which findings were presented and discussed. The updated PsA Core Domain Set endorsed with 90% agreement by OMERACT 2016 participants included musculoskeletal disease activity, skin disease activity, fatigue, pain, patient’s global assessment, physical function, health-related quality of life, and systemic inflammation, which were recommended for all RCT and LOS. These were important, but not required in all RCT and LOS: economic cost, emotional well-being, participation, and structural damage. Independence, sleep, stiffness, and treatment burden were on the research agenda. Conclusion The updated PsA Core Domain Set was endorsed at OMERACT 2016. Next steps for the PsA working group include evaluation of PsA outcome measures and development of a PsA Core Outcome Measurement Set.
90 citations
References
More filters
TL;DR: Research electronic data capture (REDCap) is a novel workflow methodology and software solution designed for rapid development and deployment of electronic data Capture tools to support clinical and translational research.
29,988 citations
TL;DR: Qualitative research produces large amounts of textual data in the form of transcripts and observational fieldnotes, and the systematic and rigorous preparation and analysis of these data is time consuming and labour intensive.
Abstract: This is the second in a series of three articles
Contrary to popular perception, qualitative research can produce vast amounts of data. These may include verbatim notes or transcribed recordings of interviews or focus groups, jotted notes and more detailed “fieldnotes” of observational research, a diary or chronological account, and the researcher's reflective notes made during the research. These data are not necessarily small scale: transcribing a typical single interview takes several hours and can generate 20–40 pages of single spaced text. Transcripts and notes are the raw data of the research. They provide a descriptive record of the research, but they cannot provide explanations. The researcher has to make sense of the data by sifting and interpreting them.
#### Summary points
Qualitative research produces large amounts of textual data in the form of transcripts and observational fieldnotes
The systematic and rigorous preparation and analysis of these data is time consuming and labour intensive
Data analysis often takes place alongside data collection to allow questions to be refined and new avenues of inquiry to develop
Textual data are typically explored inductively using content analysis to generate categories and explanations; software packages can help with analysis but should not be viewed as short cuts to rigorous and systematic analysis
High quality analysis of qualitative data depends on the skill, vision, and integrity of the researcher; it should not be left to the novice
In much qualitative research the analytical process begins during data collection as the data already gathered are analysed and shape the ongoing data collection. This sequential analysis1 or interim analysis2 has the advantage of allowing the researcher to go back and refine questions, develop hypotheses, and pursue emerging avenues of inquiry in further depth. Crucially, it also enables the researcher to look for deviant or negative cases; that is, …
7,637 citations
TL;DR: The CASPAR criteria are simple and highly specific but less sensitive than the Vasey and Espinoza criteria and are confirmed as the validity of clinical diagnosis as the gold standard definition of "case"-ness.
Abstract: Objective
To compare the accuracy of existing classification criteria for the diagnosis of psoriatic arthritis (PsA) and to construct new criteria from observed data.
Methods
Data were collected prospectively from consecutive clinic attendees with PsA and other inflammatory arthropathies. Subjects were classified by each of 7 criteria. Sensitivity and specificity were compared using conditional logistic regression analysis. Latent class analysis was used to calculate criteria accuracy in order to confirm the validity of clinical diagnosis as the gold standard definition of “case”-ness. Classification and Regression Trees methodology and logistic regression were used to identify items for new criteria, which were then constructed using a receiver operating characteristic curve.
Results
Data were collected on 588 cases and 536 controls with rheumatoid arthritis (n = 384), ankylosing spondylitis (n = 72), undifferentiated arthritis (n = 38), connective tissue disorders (n = 14), and other diseases (n = 28). The specificity of each set of criteria was high. The sensitivity of the Vasey and Espinoza method (0.97) was similar to that of the method of McGonagle et al (0.98) and greater than that of the methods of Bennett (0.44), Moll and Wright (0.91), the European Spondylarthropathy Study Group (0.74), and Gladman et al (0.91). The CASPAR (ClASsification criteria for Psoriatic ARthritis) criteria consisted of established inflammatory articular disease with at least 3 points from the following features: current psoriasis (assigned a score of 2; all other features were assigned a score of 1), a history of psoriasis (unless current psoriasis was present), a family history of psoriasis (unless current psoriasis was present or there was a history of psoriasis), dactylitis, juxtaarticular new bone formation, rheumatoid factor negativity, and nail dystrophy. These criteria were more specific (0.987 versus 0.960) but less sensitive (0.914 versus 0.972) than those of Vasey and Espinoza.
Conclusion
The CASPAR criteria are simple and highly specific but less sensitive than the Vasey and Espinoza criteria.
2,797 citations
01 Jan 2000
TL;DR: In this article, the authors present a method for sifting and interpreting large-scale qualitative research data, such as verbatim notes or transcribed recordings of interviews or focus groups, jotted notes and more detailed fieldnotes.
Abstract: Contrary to popular perception, qualitative research can produce vast amounts of data. These may include verbatim notes or transcribed recordings of interviews or focus groups, jotted notes and more detailed “fieldnotes” of observational research, a diary or chronological account, and the researcher’s reflective notes made during the research. These data are not necessarily small scale: transcribing a typical single interview takes several hours and can generate 20-40 pages of single spaced text. Transcripts and notes are the raw data of the research. They provide a descriptive record of the research, but they cannot provide explanations. The researcher has to make sense of the data by sifting and interpreting them.
2,509 citations
TL;DR: If the journal does publish papers for education and debate it follows that they should be understandable to all of the readers of the journal, including such lowly students as surgeons, and that it has to be remembered by educators that an essential part of the educational process is good communication.
Abstract: Editor—The first Education and debate section of the new millennium was very educational in a way that was almost certainly not anticipated or intended by either the staff of the journal (unless they were being very mischievous) or the authors of the papers concerned.1,2 In the paper by Lilford et al1 the study under discussion was clearly defined, but unfortunately in the paper by Mays et al2 I was not able, after reading the paper three times, to find a definition of the type of research being discussed anywhere.
The style of the paper by Lilford et al allowed an easy understanding of the thesis being developed, but the same could not be said of the paper by Mays et al, which seemed to lack a clearly discernible logic in relation to the case being made. The paper was replete with jargon and many strangely unscientific terms, which made it difficult to read—such as “epistemological,” “extreme relativists,” “antirealist,” “reflexivity,” “inductive inquiries,” and “subtle realism.” No such problem seemed to exist in relation to the paper by Lilford et al. As one of the “researchers from other traditions,” I was appalled to read of research trying to “derive . . . unequivocal insights.” I thought in my “naive realism” that we sought facts. Should not all research “be concerned to develop theory?” The need to develop a hypothesis to be tested is surely not “arguable.” I was taught by my research mentors that the truth, rather than subtle realism, was what we were trying to attain. It would have been unthinkable to omit a clear account of the process of data collection and analysis.
In this double blind (I had no idea prior to publication of the content or style), randomised (by chance I chose to read the “unintelligible paper” first) controlled (the papers were controls for each other) trial, not intended by the journal (?), I found a significant difference (I could not even understand one of the papers) in favour of tracker studies. Perhaps this was because of my only admitted bias or conflict of interest, that of being a surgeon and an educator. I am not really sure what all of this means except that if the journal does publish papers for education and debate it follows that they should be understandable to all of the readers of the journal, including such lowly students as surgeons, and that it has to be remembered by educators that an essential part of the educational process is good communication. Quality in qualitative research is a mystery to many health services researchers, and, sadly, it is an even greater mystery to me now. I am left pondering the simple question “Who should be responsible for educating the educators?”
1,135 citations