Fludarabine (FLU) is a fluorinated purine analogue with a promising an
tineoplastic activity in lymphoproliferative disorders. In this study,
we evaluated the efficacy of FLU in 12 previously treated (primary re
fractory and refractory relapse) patients with Waldenstrom's macroglob
ulinemia. All patients were treated at a dosage of 25 mg/m(2) per day
for 5 consecutive days for a total of six courses. Of the 12 patients,
5 (41%) achieved partial response (PR), and the remaining 7 showed no
benefit from the treatment. An increased response rate was obtained i
n the 4 primary refractory patients in which 2 PR were documented. Tre
atment was well-tolerated and there were no Fludarabine-related fatali
ties. With a mean follow-up of 10 months, only 1 PR patient has relaps
ed, Fludarabine is an interesting new salvage agent effective against
recurrent/resistant Waldenstrom's macroglobulinemia and should be eval
uated in further studies in untreated patients with Fludarabine in mon
ochemotherapy or in combination with other active modalities.