Effects of the American College of Rheumatology systemic sclerosis trial guidelines on the nature of systemic sclerosis patients entering a clinical trial

Abstract

Objectives. To compare the systemic sclerosis (SSc) patients entered into the D-penicillamine trial with SSc patients entered into previous controlled SSc trials. It was hypothesized that the D-penicillamine trial patients, who conformed to the American College of Rheumatology (ACR) guidelines for clinical trials in SSc were different from patients entered into previous trials. Methods. Patients entering a double-blind, randomized trial of low- vs high-dose D-penicillamine were described carefully and completely. Their characteristics were then compared with previously published data on SSc and its treatment. Results. One hundred and thirty-four patients had early [mean duration 9.5 (S.D. 4.2) months], diffuse [skin score 21 (8)] disease. Organ involvement in the patients was as follows: pulmonary 54%, cardiac 20%, joints 38%, muscular 20%. Thirty-three per cent had mild proteinuria and 13% were hypertensive when first seen. Compared with patients in most previous studies, these SSc patients had earlier disease and uniformly had diffuse disease. They had less muscular involvement, less dyspnoea, less abnormal pulmonary function and less cardiac and less renal involvement than patients in earlier studies. Conclusions. The use of the new ACR guidelines for SSc trials may change the nature of patient populations entering future studies.