4Quantitative measures for assessing rheumatoid arthritis in clinical trials and clinical care
Section snippets
Quantitative measures of activity and damage used to assess rheumatoid arthritis
Quantitative measures used to assess patient status in RA include laboratory tests, radiographic scores, formal joint counts, physical measures of functional status, global measures and patient self-report questionnaires (Table 1). These measures may be classified as measures of disease activity, measures of damage to joints and other organs, measures which assess both activity and damage, and long-term outcomes.1
Measures of disease activity, such as joint swelling, are consequences of a
Joint counts
The joint count has been described in many formats. The Glossary Committee of the American Rheumatism Association (ARA) presented a joint count involving 80 joints, each of which was analysed for five variables: swelling, tenderness, pain on motion, limited motion and deformity.19 Swelling was not assessed in the shoulder and hip, in which it is difficult to assess. High correlations were found between tenderness and pain on motion, as well as between deformity and limited motion20, which has
Indices for assessing clinical status and responses to therapy
The classical four-point global scales for assessing functional status and radiographic findings were published in 1949 by Steinbrocker et al.29 These indices are still used today but they are not sufficiently sensitive to changes in clinical status to assess responses to therapy (Table 5).
An early, more detailed, index was ‘a therapeutic scorecard in rheumatoid arthritis’81 (Table 6), which included joint tenderness, joint swelling, joint limited motion, functional status, pain, haemoglobin,
Limitations of randomized clinical trials
The randomized controlled clinical trial is certainly the ‘gold standard’ for evaluating any new therapy89, but is most effective in acute diseases over short periods. In chronic diseases over long periods, important limitations emerge (Table 7); these limitations have been described in reports by several observers89., 90., 91., 92., 93., 94., 95., 96., 97., 98., 99., 100., 101., 102., 103., 104., 105., 106. as well as in our own commentaries.107., 108., 109., 110., 111., 112., 113. Although
Intrinsic limitations of clinical trials
The above pragmatic limitations are potentially surmountable if there were sufficient resources and time to conduct clinical trials, but certain limitations are intrinsic to the method (Table 7).
A rationale for monitoring clinical status in routine clinical care using self-report questionnaires
The limitations of clinical trials, and the strong likelihood that most patients with rheumatic diseases will not be included in clinical trials, indicate that additional approaches are required to obtain optimal information concerning results of clinical care in RA, including the use of new biological agents. As noted above, the question of whether long-term low-dose methotrexate, corticosteroids or new biological agents can change severe long-term outcomes cannot be answered through
A method for collecting data in clinical care using self-report questionnaires
In the clinic of the senior clinical author, a questionnaire has been completed by every patient at every visit135 over the last 20 years. When the patient registers for a visit, he or she is asked by the receptionist to complete a questionnaire mounted on a clipboard, along with a soft pencil or felt-tip pen, while waiting to see the physician. The questionnaire must be distributed in a cheerful and caring manner, so that patients recognize it as an important component of their medical care—to
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