Sensitivity to change of the Health Assessment Questionnaire (HAQ) and other clinical and health status measures in rheumatoid arthritis results of short‐term clinical trials and observational studies versus long‐term observational studies

Abstract

To obtain evidence concerning short‐term and longterm efficacy of clinical and health status measures in rheumatoid arthritis (RA), we conducted two observational studies—a 6‐month study of 233 patients receiving methotrexate and a 10‐year study of 157 patients receiving multiple treatments in a rheumatic disease clinic. Results of the 6‐month study yielded effect sizes for treatment similar to the metaanalyses reported by Weinblatt et al. (Arthritis Rheum 33:1449–1461, 1990) and the controlled trials of methotrexate reported by Weinblatt et al. (Arthritis Rheum 33:330–338, 1990), suggesting that observational studies provide valid measurements of treatment effect. The effect size for the Health Assessment Questionnaire (HAQ) was 0.5. By contrast, the 10‐year study suggested that standard clinical variables changed little and were not useful in assessing RA outcome, while the effect size of the HAQ was − 2.39. These data continue to underscore the differences between short‐term trials and the long‐term outcome of RA, and suggest an important place for the HAQ or similar instruments in all phases of RA evaluation and assessment.

Measures of activity and damage in rheumatoid arthritis: Depiction of changes and prediction of mortality over five years

“…Proposed criteria for improvement (92) include a 20% improvement in tender and swollen joint counts and in 3 of 5 remaining measures-patient and physician global assessments, pain, patient questionnaire, and ESR from the "Core Data Set" recommended for use in clinical trials (93)(94)(95). Evidence presented here and in other reports (30)(31)(32) suggests that patients may meet improvement criteria and nonetheless show disease progression according to measures of damage, although further direct analyses of short-term improvement are needed. It may be that more aggressive strategies, toward remission, may be required to control RA (83,85,96,97).…”

“…A repeat assessment was performed in 130 of the 169 survivors, 100 of whom had a complete ACR glossary 70-joint count at baseline. The other 39 surviving patients did not return for a comprehensive evaluation: 5 had moved, 30 indicated they were too infirm to travel to the clinic (many lived at least 50 miles away), and 4 refused to return for an evaluation. A patient questionnaire was completed by telephone or mail by each of these patients, except the 4 who refused a second evaluation (who were recognized as survivors in analyses over 5 years).…”

“…Reports by Hawley and Wolfe (30), Paimela et a1 (31), and Mulherin et a1 (32) suggest that many measures of inflammatory activity may indicate improvement or no change over long periods, while measures of damage indicate disease progression in the same RA patients.…”

The influence of medication beliefs and other psychosocial factors on early discontinuation of disease‐modifying anti‐rheumatic drugs

“…Duration of morning stiffness (Mallya and Mace, 1981), pain (on a 100 mm visual analogue scale; VAS) (Mallya and Mace, 1981), patient global disease activity VAS (Guillemin et al, 1997), grip strength (Harkenonen et al, 1993), and swollen and tender joint count (Ritchie et al, 1968;Smolen et al, 1995) were measured at both visits, and a patient record of DMARD discontinuation and the reason for it was collected at the second interview (also see Callahan, 1992a, 1992b (Mallya and Mace, 1981;van der Heijde et al, 1992). Patients completed a range of questionnaires at both visits, including the modifi ed Stanford Health Assessment Questionnaire (HAQ) (Kirwan et al, 1986;Hawley and Wolfe, 1992), the Beck Depression Inventory-1 (BDI-1) (Beck et al, 1988) and some further self-reported measures of psychological health.…”

Life events and disability in rheumatoid arthritis: a European cohort

“…Few long-term studies have been carried out in this area. Hawley and Wolfe [27] followed a cohort for 10 yr, collecting clinical data on a regular basis and recording the patients' overall [30] found a HAQ score of 1.06 in a group of 79 women (duration of disease < 10 yr). Taking into account these scores and the fact that our patients were volunteers, one could expect a selection of severe cases in the constitution of the EURIDISS cohort.…”