Vincristine (1.4 mg/m(2) on day 1, followed by 1 mg on days 4 and 7) was gi
ven to eight patients with thrombotic thrombocytopenic purpura (TTP) who we
re refractory to plasma exchange (n = 4) or plasma infusion (n = 4). Seven
of eight patients (87%) achieved a complete response; one was refractory to
treatment and died within a few weeks. After a median follow-up of 50 mont
hs, all responding patients are alive and well. Two patients relapsed and w
ere successfully retreated with vincristine. Toxicity was mild, consisting
of two episodes of leukopenia and one of autonomic neuropathy leading to pa
ralytic ileus in a patient aged 70 years. We conclude that vincristine is h
ighly effective in the treatment of patients suffering from refractory TTP,
with negligible toxicity.