Chlorambucil in severe juvenile chronic arthritis: longterm followup with special reference to amyloidosis.

OBJECTIVE: To assess the effectiveness and side effects of chlorambucil therapy and patient outcome in juvenile chronic arthritis (JCA) treated with chlorambucil.
METHODS: 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.
RESULTS: 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).
CONCLUSION: 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.