Assessing prognosis and prediction of treatment response in early rheumatoid arthritis: systematic reviews

Background: Rheumatoid arthritis (RA) is a chronic, debilitating disease associated with reduced quality of life and substantial costs. It is unclear which tests and assessment tools allow the best assessment of prognosis in people with early RA and whether or not variables predict the response of p...

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Main Authors: Rachel Archer, Emma Hock, Jean Hamilton, John Stevens, Munira Essat, Edith Poku, Mark Clowes, Abdullah Pandor, Matt Stevenson
Format: Article
Language:English
Published: NIHR Journals Library 2018-11-01
Series:Health Technology Assessment
Subjects:
Online Access:https://doi.org/10.3310/hta22660
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author Rachel Archer
Emma Hock
Jean Hamilton
John Stevens
Munira Essat
Edith Poku
Mark Clowes
Abdullah Pandor
Matt Stevenson
author_facet Rachel Archer
Emma Hock
Jean Hamilton
John Stevens
Munira Essat
Edith Poku
Mark Clowes
Abdullah Pandor
Matt Stevenson
author_sort Rachel Archer
collection DOAJ
description Background: Rheumatoid arthritis (RA) is a chronic, debilitating disease associated with reduced quality of life and substantial costs. It is unclear which tests and assessment tools allow the best assessment of prognosis in people with early RA and whether or not variables predict the response of patients to different drug treatments. Objective: To systematically review evidence on the use of selected tests and assessment tools in patients with early RA (1) in the evaluation of a prognosis (review 1) and (2) as predictive markers of treatment response (review 2). Data sources: Electronic databases (e.g. MEDLINE, EMBASE, The Cochrane Library, Web of Science Conference Proceedings; searched to September 2016), registers, key websites, hand-searching of reference lists of included studies and key systematic reviews and contact with experts. Study selection: Review 1 – primary studies on the development, external validation and impact of clinical prediction models for selected outcomes in adult early RA patients. Review 2 – primary studies on the interaction between selected baseline covariates and treatment (conventional and biological disease-modifying antirheumatic drugs) on salient outcomes in adult early RA patients. Results: Review 1 – 22 model development studies and one combined model development/external validation study reporting 39 clinical prediction models were included. Five external validation studies evaluating eight clinical prediction models for radiographic joint damage were also included. c-statistics from internal validation ranged from 0.63 to 0.87 for radiographic progression (different definitions, six studies) and 0.78 to 0.82 for the Health Assessment Questionnaire (HAQ). Predictive performance in external validations varied considerably. Three models [(1) Active controlled Study of Patients receiving Infliximab for the treatment of Rheumatoid arthritis of Early onset (ASPIRE) C-reactive protein (ASPIRE CRP), (2) ASPIRE erythrocyte sedimentation rate (ASPIRE ESR) and (3) Behandelings Strategie (BeSt)] were externally validated using the same outcome definition in more than one population. Results of the random-effects meta-analysis suggested substantial uncertainty in the expected predictive performance of models in a new sample of patients. Review 2 – 12 studies were identified. Covariates examined included anti-citrullinated protein/peptide anti-body (ACPA) status, smoking status, erosions, rheumatoid factor status, C-reactive protein level, erythrocyte sedimentation rate, swollen joint count (SJC), body mass index and vascularity of synovium on power Doppler ultrasound (PDUS). Outcomes examined included erosions/radiographic progression, disease activity, physical function and Disease Activity Score-28 remission. There was statistical evidence to suggest that ACPA status, SJC and PDUS status at baseline may be treatment effect modifiers, but not necessarily that they are prognostic of response for all treatments. Most of the results were subject to considerable uncertainty and were not statistically significant. Limitations: The meta-analysis in review 1 was limited by the availability of only a small number of external validation studies. Studies rarely investigated the interaction between predictors and treatment. Suggested research priorities: Collaborative research (including the use of individual participant data) is needed to further develop and externally validate the clinical prediction models. The clinical prediction models should be validated with respect to individual treatments. Future assessments of treatment by covariate interactions should follow good statistical practice. Conclusions: Review 1 – uncertainty remains over the optimal prediction model(s) for use in clinical practice. Review 2 – in general, there was insufficient evidence that the effect of treatment depended on baseline characteristics. Study registration: This study is registered as PROSPERO CRD42016042402. Funding: The National Institute for Health Research Health Technology Assessment programme.
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spelling doaj.art-d83c88de61564879944793cb5b83bd672022-12-22T01:07:13ZengNIHR Journals LibraryHealth Technology Assessment1366-52782046-49242018-11-01226610.3310/hta2266014/151/08Assessing prognosis and prediction of treatment response in early rheumatoid arthritis: systematic reviewsRachel Archer0Emma Hock1Jean Hamilton2John Stevens3Munira Essat4Edith Poku5Mark Clowes6Abdullah Pandor7Matt Stevenson8School of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UKSchool of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UKSchool of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UKSchool of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UKSchool of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UKSchool of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UKSchool of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UKSchool of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UKSchool of Health and Related Research (ScHARR), University of Sheffield, Sheffield, UKBackground: Rheumatoid arthritis (RA) is a chronic, debilitating disease associated with reduced quality of life and substantial costs. It is unclear which tests and assessment tools allow the best assessment of prognosis in people with early RA and whether or not variables predict the response of patients to different drug treatments. Objective: To systematically review evidence on the use of selected tests and assessment tools in patients with early RA (1) in the evaluation of a prognosis (review 1) and (2) as predictive markers of treatment response (review 2). Data sources: Electronic databases (e.g. MEDLINE, EMBASE, The Cochrane Library, Web of Science Conference Proceedings; searched to September 2016), registers, key websites, hand-searching of reference lists of included studies and key systematic reviews and contact with experts. Study selection: Review 1 – primary studies on the development, external validation and impact of clinical prediction models for selected outcomes in adult early RA patients. Review 2 – primary studies on the interaction between selected baseline covariates and treatment (conventional and biological disease-modifying antirheumatic drugs) on salient outcomes in adult early RA patients. Results: Review 1 – 22 model development studies and one combined model development/external validation study reporting 39 clinical prediction models were included. Five external validation studies evaluating eight clinical prediction models for radiographic joint damage were also included. c-statistics from internal validation ranged from 0.63 to 0.87 for radiographic progression (different definitions, six studies) and 0.78 to 0.82 for the Health Assessment Questionnaire (HAQ). Predictive performance in external validations varied considerably. Three models [(1) Active controlled Study of Patients receiving Infliximab for the treatment of Rheumatoid arthritis of Early onset (ASPIRE) C-reactive protein (ASPIRE CRP), (2) ASPIRE erythrocyte sedimentation rate (ASPIRE ESR) and (3) Behandelings Strategie (BeSt)] were externally validated using the same outcome definition in more than one population. Results of the random-effects meta-analysis suggested substantial uncertainty in the expected predictive performance of models in a new sample of patients. Review 2 – 12 studies were identified. Covariates examined included anti-citrullinated protein/peptide anti-body (ACPA) status, smoking status, erosions, rheumatoid factor status, C-reactive protein level, erythrocyte sedimentation rate, swollen joint count (SJC), body mass index and vascularity of synovium on power Doppler ultrasound (PDUS). Outcomes examined included erosions/radiographic progression, disease activity, physical function and Disease Activity Score-28 remission. There was statistical evidence to suggest that ACPA status, SJC and PDUS status at baseline may be treatment effect modifiers, but not necessarily that they are prognostic of response for all treatments. Most of the results were subject to considerable uncertainty and were not statistically significant. Limitations: The meta-analysis in review 1 was limited by the availability of only a small number of external validation studies. Studies rarely investigated the interaction between predictors and treatment. Suggested research priorities: Collaborative research (including the use of individual participant data) is needed to further develop and externally validate the clinical prediction models. The clinical prediction models should be validated with respect to individual treatments. Future assessments of treatment by covariate interactions should follow good statistical practice. Conclusions: Review 1 – uncertainty remains over the optimal prediction model(s) for use in clinical practice. Review 2 – in general, there was insufficient evidence that the effect of treatment depended on baseline characteristics. Study registration: This study is registered as PROSPERO CRD42016042402. Funding: The National Institute for Health Research Health Technology Assessment programme.https://doi.org/10.3310/hta22660rheumatoid arthritisprognosisprediction modelstratified medicine
spellingShingle Rachel Archer
Emma Hock
Jean Hamilton
John Stevens
Munira Essat
Edith Poku
Mark Clowes
Abdullah Pandor
Matt Stevenson
Assessing prognosis and prediction of treatment response in early rheumatoid arthritis: systematic reviews
Health Technology Assessment
rheumatoid arthritis
prognosis
prediction model
stratified medicine
title Assessing prognosis and prediction of treatment response in early rheumatoid arthritis: systematic reviews
title_full Assessing prognosis and prediction of treatment response in early rheumatoid arthritis: systematic reviews
title_fullStr Assessing prognosis and prediction of treatment response in early rheumatoid arthritis: systematic reviews
title_full_unstemmed Assessing prognosis and prediction of treatment response in early rheumatoid arthritis: systematic reviews
title_short Assessing prognosis and prediction of treatment response in early rheumatoid arthritis: systematic reviews
title_sort assessing prognosis and prediction of treatment response in early rheumatoid arthritis systematic reviews
topic rheumatoid arthritis
prognosis
prediction model
stratified medicine
url https://doi.org/10.3310/hta22660
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