Sean P Gavan, Sainan Chang, Felice Rivellese, Zoë Ide, Michael Stadler, Katherine Payne, Darren Plant, Anne Barton, Costantino Pitzalis
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引用次数: 0
Abstract
To map from the health assessment questionnaire disability index (HAQ) to the pain visual analogue scale (VAS) for people with rheumatoid arthritis. The estimation sample comprised adults with rheumatoid arthritis and inadequate response to tumour necrosis factor-α inhibitors in a multicentre phase 4 randomised controlled trial. Beta mixture models were estimated with combinations of HAQ and its square, age and sex as independent variables. Bayesian Information Criteria informed the number of components. Model performance (root mean squared error; mean absolute error; pseudo-R2) was estimated by k-fold cross validation. Graphs illustrated mean observed and predicted pain VAS, and cumulative distribution of observed and simulated pain VAS values. For face validity, a probabilistic analysis simulated 5000 pain VAS values at four HAQ scores. For external validation, the performance of the preferred specification was assessed using the Rheumatoid Arthritis Medication Study cohort. There were 1055 observations from 158 participants in the estimation sample (mean age: 55.8; 81% female; mean HAQ: 1.72). The preferred specification was a two-component beta mixture model (probability variables: HAQ, age, sex; main regression variable: HAQ). Visual plots illustrated good fit across the HAQ distribution, and a similar cumulative distribution of observed and predicted pain VAS values. Probabilistic analysis demonstrated that the preferred specification handled uncertainty appropriately. External validation demonstrated that the preferred specification performed well in an independent dataset. Beta mixture models provide accurate non-linear estimates of pain VAS from HAQ scores to support evidence synthesis and resource allocation decision-making for people with rheumatoid arthritis.
期刊介绍:
RHEUMATOLOGY INTERNATIONAL is an independent journal reflecting world-wide progress in the research, diagnosis and treatment of the various rheumatic diseases. It is designed to serve researchers and clinicians in the field of rheumatology.
RHEUMATOLOGY INTERNATIONAL will cover all modern trends in clinical research as well as in the management of rheumatic diseases. Special emphasis will be given to public health issues related to rheumatic diseases, applying rheumatology research to clinical practice, epidemiology of rheumatic diseases, diagnostic tests for rheumatic diseases, patient reported outcomes (PROs) in rheumatology and evidence on education of rheumatology. Contributions to these topics will appear in the form of original publications, short communications, editorials, and reviews. "Letters to the editor" will be welcome as an enhancement to discussion. Basic science research, including in vitro or animal studies, is discouraged to submit, as we will only review studies on humans with an epidemological or clinical perspective. Case reports without a proper review of the literatura (Case-based Reviews) will not be published. Every effort will be made to ensure speed of publication while maintaining a high standard of contents and production.
Manuscripts submitted for publication must contain a statement to the effect that all human studies have been reviewed by the appropriate ethics committee and have therefore been performed in accordance with the ethical standards laid down in an appropriate version of the 1964 Declaration of Helsinki. It should also be stated clearly in the text that all persons gave their informed consent prior to their inclusion in the study. Details that might disclose the identity of the subjects under study should be omitted.