To assess the effectiveness and side effects of chlorambucil therapy and patient outcome in juvenile chronic arthritis (JCA) treated with chlorambucil.
Followup of 79 consecutive patients with JCA refractory to previous therapy, in whom chlorambucil treatment was initiated from 1982 to 1995. Mean treatment duration was 7 months in 72 patients and more than 20 months in 6 patients with amyloidosis and 1 with severe iridocyclitis.
Within 6 months, remission was attained in 41 patients (52%): 29 of these relapsed after a mean period of 2.5 years (range 0.3-8.3). Twenty-one (27%) patients did not respond. Seven out of 11 patients with secondary amyloidosis had proteinuria, which cleared completely in 4 and almost completely in 1. In 16 patients (20%) chlorambucil was stopped because of side effects, and in 1 because of infection. After a mean followup of 8.5 years, 14 patients (18%) were in complete remission without drugs, 34 (43%) had minor symptoms only, but 26 (33%) had limitations in daily activities. Five (6%) had died (2 of leukemia; 2 of infection, 0.5 and 3.3 years after withdrawal of chlorambucil; 1 of amyloidosis).
Chlorambucil was found to be a very potent drug for JCA with acceptable short term, but serious longterm side effects. It may still be useful in JCA complicated by amyloidosis.