Purpose: To assess the activity and side effects of cladribine (2-CdA)
treatment in patients with advanced Waldenstrom's disease. Patients a
nd methods: Ten symptomatic patients without prior therapy were includ
ed in a prospective multicenter trial, 2-CdA was administered daily at
0.12 mg/kg body weight in a 2-h i.v. infusion over 5 consecutive days
; this was repeated every 25 days for four cycles. Patients achieving
a remission received interferon alfa-2c (IF) 15 mu g s.c. three times
a week for 1 year. Results: All 10 patients responded to 2-CdA (100%;
95% confidence interval, 68-100%), with one complete (CR) and eight pa
rtial responders (PR); one patient had only one 2-CdA cycle and showed
a minor improvement (MR), Patients tolerated the treatment well. Desp
ite considerable immunosuppression, an infection occurred in only two
patients, After a median observation period of 57 weeks, three patient
s had shown progression, including one who died of lymphoma. Conclusio
n: 2-CdA induction and IF maintenance is a well-tolerated therapy for
symptomatic untreated patients with advanced Waldenstrom's disease and
offers excellent palliation.