Exploration, Development, and Validation of Patient-reported Outcomes in Antineutrophil Cytoplasmic Antibody–associated Vasculitis Using the OMERACT Process

Objective.Antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) is a group of linked multisystem life- and organ-threatening diseases. The Outcome Measures in Rheumatology (OMERACT) vasculitis working group has been at the forefront of outcome development in the field and has achieved OMERACT endorsement of a core set of outcomes for AAV. Patients with AAV report as important some manifestations of disease not routinely collected through physician-completed outcome tools; and they rate common manifestations differently from investigators. The core set includes the domain of patient-reported outcomes (PRO). However, PRO currently used in clinical trials of AAV do not fully characterize patients’ perspectives on their burden of disease. The OMERACT vasculitis working group is addressing the unmet needs for PRO in AAV.Methods.Current activities of the working group include (1) evaluating the feasibility and construct validity of instruments within the PROMIS (Patient-Reported Outcome Measurement Information System) to record components of the disease experience among patients with AAV; (2) creating a disease-specific PRO measure for AAV; and (3) applying The International Classification of Functioning, Disability and Health to examine the scope of outcome measures used in AAV.Results.The working group has developed a comprehensive research strategy, organized an investigative team, included patient research partners, obtained peer-reviewed funding, and is using a considerable research infrastructure to complete these interrelated projects to develop evidence-based validated outcome instruments that meet the OMERACT filter of truth, discrimination, and feasibility.Conclusion.The OMERACT vasculitis working group is on schedule to achieve its goals of developing validated PRO for use in clinical trials of AAV.

Download Full-text

OMERACT Endorsement of Patient-reported Outcome Instruments in Antineutrophil Cytoplasmic Antibody–associated Vasculitis

The Journal of Rheumatology ◽

10.3899/jrheum.161139 ◽

2017 ◽

Vol 44 (10) ◽

pp. 1529-1535 ◽

Cited By ~ 13

Author(s):

Joanna C. Robson ◽

Gunnar Tomasson ◽

Nataliya Milman ◽

Sue Ashdown ◽

Annelies Boonen ◽

...

Keyword(s):

Physical Functioning ◽

Working Group ◽

Patient Reported Outcomes ◽

Controlled Trial ◽

Antineutrophil Cytoplasmic Antibody ◽

Pain Interference ◽

Disease Assessment ◽

Measurement Information ◽

Patient Reported ◽

Disease Specific

Objective.The antineutrophil cytoplasmic antibody–associated vasculitides (AAV) are multiorgan diseases. Patients with AAV report impairment in their health-related quality of life (HRQOL) and have different priorities regarding disease assessment compared with physicians. The Outcome Measures in Rheumatology (OMERACT) Vasculitis Working Group previously received endorsement for a core set of domains in AAV. Two approaches to measure patient-reported outcomes (PRO) were presented at OMERACT 2016.Methods.A novel 5-step tool was used to facilitate assessment of the instruments by delegates: the OMERACT Filter 2.0 Instrument Selection Algorithm, with a red-amber-green checklist of questions, including (1) good match with domain (face and content validity), (2) feasibility, (3) do numeric scores make sense (construct validity)?, (4) overall ratings of discrimination, and (5) can individual thresholds of meaning be defined? Delegates gave an overall endorsement. Three generic Patient-Reported Outcomes Measurement Information System (PROMIS) instruments (fatigue, physical functioning, and pain interference) and a disease-specific PRO, the AAV-PRO (6 domains related to symptoms and HRQOL), were presented.Results.OMERACT delegates endorsed the use of the PROMIS instruments for fatigue, physical functioning, and pain interference (87.6% overall endorsement) and the disease-specific AAV-PRO instrument (89.4% overall endorsement).Conclusion.The OMERACT Vasculitis Working Group gained endorsement by OMERACT for use of the PROMIS and the AAV-PRO in clinical trials of vasculitis. These instruments are complementary to each other. The PROMIS and the AAV-PRO need further work to assess their utility in longitudinal settings, including their ability to discriminate between treatments of varying efficacy in the setting of a randomized controlled trial.

Download Full-text

Appraisal of Candidate Instruments for Assessment of the Physical Function Domain in Patients with Psoriatic Arthritis

The Journal of Rheumatology ◽

10.3899/jrheum.191119 ◽

2020 ◽

Vol 48 (1) ◽

pp. 58-66 ◽

Cited By ~ 1

Author(s):

Ying Ying Leung ◽

Ana-Maria Orbai ◽

Alexis Ogdie ◽

Pil Hojgaard ◽

Richard Holland ◽

...

Keyword(s):

Psoriatic Arthritis ◽

Physical Function ◽

Outcome Measures ◽

Physical Functioning ◽

Working Group ◽

Short Form ◽

Outcome Measurement ◽

Patient Reported Outcome Measures ◽

Measurement Information ◽

Patient Reported

Objective.Numerous patient-reported outcome measures (PROM) exist for the measurement of physical function for psoriatic arthritis (PsA), but only a few are validated comprehensively. The objective of this project was to prioritize PROM for measuring physical function for potential incorporation into a standardized outcome measurement set for PsA.Methods.A working group of 13 members including 2 patient research partners was formed. PROM measuring physical function in PsA were identified through a systematic literature review and recommendations by the working group. The rationale for inclusion and exclusion from the original list of existing PROM was thoroughly discussed and 2 rounds of Delphi exercises were conducted to achieve consensus.Results.Twelve PROM were reviewed and discussed. Six PROM were prioritized: Health Assessment Questionnaire (HAQ) and 4 modifications (HAQ-Disability Index, HAQ-Spondyloarthritis, modified HAQ, multidimensional HAQ), Medical Outcomes Study 36-item Short Form survey physical functioning domain, and the Patient-Reported Outcomes Measurement Information System (PROMIS) physical functioning module.Conclusion.Through discussion and Delphi exercises, we achieved consensus to prioritize 6 physical function PROM for PsA. These 6 PROM will undergo further appraisal using the Outcome Measures in Rheumatology (OMERACT) Filter 2.1.

Download Full-text

Developing a Core Set of Outcome Measures for Behçet Disease: Report from OMERACT 2016

The Journal of Rheumatology ◽

10.3899/jrheum.161352 ◽

2017 ◽

Vol 44 (11) ◽

pp. 1750-1753 ◽

Cited By ~ 14

Author(s):

Gulen Hatemi ◽

Alexa Meara ◽

Yesim Ozguler ◽

Haner Direskeneli ◽

Alfred Mahr ◽

...

Keyword(s):

Clinical Trials ◽

Outcome Measures ◽

Working Group ◽

Organ Involvement ◽

Future Research ◽

Behçet Disease ◽

Behcet Disease ◽

Core Set ◽

Patient Reported ◽

Specific Organ

Objective.The Outcome Measures in Rheumatology (OMERACT) Vasculitis Working Group has been working toward developing a data-driven core set of outcome measures for use in clinical trials of Behçet’s syndrome [Behçet disease (BD)]. This paper summarizes the group’s work through OMERACT 2016, discussions during the meeting, and the future research agenda.Methods.Qualitative patient interviews were conducted among 20 patients with BD who have different types of organ involvement. A 3-round Delphi among BD experts and patients was initiated to identify domains, subdomains, and outcomes to be assessed in clinical trials of BD. The results of these studies were discussed during OMERACT 2016 and next steps were planned.Results.Patients’ perspectives and priorities were identified through qualitative interviews that identified candidate domains and subdomains for inclusion in the Delphi and characterized some shortcomings of the currently used patient-reported outcomes in BD. The first round of the Delphi was completed and several domains or subdomains were endorsed by the experts and/or the patients. Because many more items were endorsed than would be feasible to assess during a clinical trial, rating and ranking of items by physicians and patients was planned as a next critical step. The challenges of assessing specific organ system involvement was also discussed.Conclusion.The OMERACT Behçet Syndrome Working Group research program will identify core domains for assessment in BD with the goal of developing a core set of outcome measures for use in all trials of BD with the option to incorporate additional outcomes for specific organ involvement.

Download Full-text

The OMERACT Core Set of Outcome Measures for Use in Clinical Trials of ANCA-associated Vasculitis

The Journal of Rheumatology ◽

10.3899/jrheum.110276 ◽

2011 ◽

Vol 38 (7) ◽

pp. 1480-1486 ◽

Cited By ~ 62

Author(s):

PETER A. MERKEL ◽

SIBEL Z. AYDIN ◽

MAARTEN BOERS ◽

HANER DIRESKENELI ◽

KAREN HERLYN ◽

...

Keyword(s):

Clinical Trials ◽

Outcome Measures ◽

Outcome Measurement ◽

Measurement Instrument ◽

The United States ◽

Anca Associated Vasculitis ◽

Core Set ◽

Patient Reported ◽

Development Evaluation ◽

Multicenter Clinical Trials

There has been a marked increase in the past 15 years in the number and quality of clinical trials in the idiopathic inflammatory vasculitides, especially the small-vessel vasculitides known as antineutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis [AAV; granulomatosis, with polyangiitis (Wegener’s)]. These trials have been conducted by multicenter, international groups in Europe and the United States with financial support provided by government agencies and biopharmaceutical companies. This increased clinical trial activity in vasculitis has been accompanied by the development and validation of new outcome measures — a challenging process for these complex, multiorgan system diseases. The international OMERACT Vasculitis Working Group has developed and implemented an iterative research agenda that has utilized accumulated experience and datasets from several multicenter clinical trials and large cohort studies. This work has led to the development, evaluation, validation, and endorsement, through the OMERACT consensus and validation processes, of a “core set” of outcome measurements for use in clinical trials of AAV. The core set includes domains of disease activity, damage assessment, patient-reported outcomes, and mortality; there is at least one validated outcome measurement instrument available for each domain. This report reviews the domains of illness in AAV included in the OMERACT core set, describes the instruments validated to measure these domains, and presents the approved core set.

Download Full-text

Defining Outcome Measures for Psoriatic Arthritis: A Report from the GRAPPA-OMERACT Working Group

The Journal of Rheumatology ◽

10.3899/jrheum.170150 ◽

2017 ◽

Vol 44 (5) ◽

pp. 697-700 ◽

Cited By ~ 18

Author(s):

Alexis Ogdie ◽

Maarten de Wit ◽

Kristina Callis Duffin ◽

Willemina Campbell ◽

Jeffrey Chau ◽

...

Keyword(s):

Clinical Trials ◽

Psoriatic Arthritis ◽

Outcome Measures ◽

Working Group ◽

Development Process ◽

Outcome Measurement ◽

Core Outcome ◽

Core Domain ◽

Core Set ◽

Core Domain Set

The Group for Research and Assessment of Psoriasis and Psoriatic Arthritis (GRAPPA)-Outcome Measures in Rheumatology (OMERACT) Psoriatic Arthritis (PsA) Core Set working group recently published the updated 2016 psoriatic arthritis (PsA) core domain set, a set of disease features that should be measured in all clinical trials. At the GRAPPA annual meeting in July 2016, the PsA working group presented the updated PsA core domain set endorsed by 90% of participants at OMERACT in May 2016 and drafted a roadmap for the development of the PsA core outcome measurement set. In this manuscript, we review the development process of the PsA core domain set and the ongoing and proposed work streams for development of a PsA core measurement set.

Download Full-text

Longitudinal Analysis of Patient-Reported Outcomes in Clinical Trials: Applications of Multilevel and Multidimensional Item Response Theory

Psychometrika ◽

10.1007/s11336-021-09777-y ◽

2021 ◽

Author(s):

Li Cai ◽

Carrie R. Houts

Keyword(s):

Clinical Trial ◽

Clinical Trials ◽

Latent Variable ◽

Patient Reported Outcomes ◽

Multilevel Models ◽

Latent Variable Models ◽

Trial Data ◽

Regulatory Science ◽

Measurement Information ◽

Patient Reported

AbstractWith decades of advance research and recent developments in the drug and medical device regulatory approval process, patient-reported outcomes (PROs) are becoming increasingly important in clinical trials. While clinical trial analyses typically treat scores from PROs as observed variables, the potential to use latent variable models when analyzing patient responses in clinical trial data presents novel opportunities for both psychometrics and regulatory science. An accessible overview of analyses commonly used to analyze longitudinal trial data and statistical models familiar in both psychometrics and biometrics, such as growth models, multilevel models, and latent variable models, is provided to call attention to connections and common themes among these models that have found use across many research areas. Additionally, examples using empirical data from a randomized clinical trial provide concrete demonstrations of the implementation of these models. The increasing availability of high-quality, psychometrically rigorous assessment instruments in clinical trials, of which the Patient-Reported Outcomes Measurement Information System (PROMIS®) is a prominent example, provides rare possibilities for psychometrics to help improve the statistical tools used in regulatory science.

Download Full-text

Adaptação transcultural dos Bancos de Itens de Ansiedade e Depressão do Patient-Reported Outcomes Measurement Information System (PROMIS) para língua portuguesa

Cadernos de Saúde Pública ◽

10.1590/0102-311x00096113 ◽

2014 ◽

Vol 30 (4) ◽

pp. 879-884 ◽

Cited By ~ 2

Author(s):

Natália Fontes Caputo de Castro ◽

Carlos Henrique Alves de Rezende ◽

Tânia Maria da Silva Mendonça ◽

Carlos Henrique Martins da Silva ◽

Rogério de Melo Costa Pinto

Keyword(s):

Information System ◽

Patient Reported Outcomes ◽

Outcome Measurement ◽

Patient Reported Outcome ◽

Measurement Information ◽

Outcomes Measurement ◽

Patient Reported ◽

Measurement Information System

O Patient-Reported Outcome Measurement Information System (PROMIS), estruturado em domínios físicos e psicossociais, superou lacunas ao propor nova ferramenta de avaliação de resultados aplicáveis às doenças crônicas com base em técnicas avançadas de estatística (TRI) e testes adaptativos computadorizados (CAT). O objetivo do estudo foi adaptar culturalmente os Bancos de Itens de Ansiedade e Depressão do PROMIS para a língua portuguesa. O processo seguiu rigorosas recomendações do FACIT por meio da tradução avançada, reconciliação, retrotradução, revisão do FACIT, revisores independentes, finalização das etapas pelo FACIT, pré-teste e incorporação dos resultados do pré- teste. A versão traduzida foi pré-testada em dez pacientes, sendo necessária a modificação nos itens 3, 46 e 53 de Ansiedade e no item 46 de Depressão. As alterações alcançaram a equivalência de significado e a versão final foi compatível com as habilidades linguísticas e culturais da população brasileira. Concluiu-se que a versão traduzida é semântica e conceitualmente equivalente aos originais.

Download Full-text

Generic Patient-Reported Outcomes and Outcome Measures: A Systematic Review Study Protocol

10.1101/2021.05.28.21257980 ◽

2021 ◽

Author(s):

Yuki Seidler ◽

Erika Mosor ◽

Margaret R Andrews ◽

Carolina Watson ◽

Nick Bott ◽

...

Keyword(s):

Systematic Review ◽

Outcome Measures ◽

Systematic Reviews ◽

Patient Reported Outcomes ◽

Outcome Measurement ◽

Meta Analysis ◽

Patient Reported Outcome ◽

Cochrane Library ◽

Screening Process ◽

Patient Reported

Background: Patient-reported outcomes (PROs) are an essential part of health outcome measurement and vital to patient-centricity and valued-based care. Several international consortia have developed core outcome sets and many of them include PROs. PROs are measured by patient-reported outcome measures (PROMs). PROs and PROMs can be generic or specific to certain diseases or conditions. While the characteristics of generic PROs and PROMs are well recognised as widely relevant and applicable across different domains, diseases and conditions, there is a lack of knowledge on the types of PROs measured by generic PROMs. We also do not know in which disease areas generic PROs and PROMs are commonly used. To date, there has been no systematic review solely focusing on generic PROMs, what they measure and their areas of application. Objectives: This systematic review will identify core PROs measured by generic PROMs used in adult populations and the areas in which they are applied. Methods: We will conduct a systematic review of reviews. The screening process and the reporting will comply with the PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analysis) 2020 Statement. We will use four databases, Medline [PubMed], CINHAL [Ebsco], Cochrane [Cochrane Library], and PsycINFO [Ovid], and reports from international consortia. Inclusion criteria are systematic reviews, meta-analysis or patient-reported outcome sets developed by international consortia reporting on generic PROMs in adult populations. Articles primarily focusing on patient-reported experience measures (PREMs), children or adolescents, or those not written in English will be excluded. Risk of bias will be assessed by checking if the included articles comply with established guidelines for systematic reviews such as the PRISMA statement. We will extract generic PROMs and PROs measured by these PROMs, and the areas applied from the selected articles and reports. Extracted data and information will be quantitatively and qualitatively synthesised without statistical interference. The quality of the synthesised evidences will be assessed by clarifying the strengths, limitations and possible biases in our review.

Download Full-text