How effective are fludarabine and cladribine in the treatment of Waldenström macroglobulinemia?

Updated: Feb 01, 2021
  • Author: Karen Seiter, MD; Chief Editor: Emmanuel C Besa, MD  more...
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The purine nucleoside analogues fludarabine and cladribine have demonstrated activity against Waldenström macroglobulinemia. They are effective therapy for patients who are primarily resistant or who relapse after alkylating agents.

Dhodapkar et al, in a study evaluating fludarabine response in previously untreated and previously treated patients, reported an overall response rate of 36%, with 3% of patients experiencing complete remission; the overall survival period was 84 months. Fludarabine has also demonstrated activity for patients resistant to cladribine. [40]

A phase III study that included 339 patients with Waldenström macroglobulinemia found that fludarabine was superior to initial therapy. Patients receiving fludarabine had significantly longer median progression-free survival than those receiving chlorambucil (37.8 versus 27.1 months, respectively; = 0.015), and median overall survival was not reached with fludarabine but was 69.8 months with chlorambucil (P = 0.014). [41]

Cladribine is also used as initial therapy, and it provides excellent response rates with minimal treatment. The MD Anderson Cancer Center published data from 90 patients treated with either cladribine alone or in combination with prednisone, cyclophosphamide, or rituximab. The overall response was 94% for cladribine alone, 60% for cladribine and prednisone, 84% for cladribine and cyclophosphamide, and 94% for cladribine, cyclophosphamide, and rituximab. [42] The median overall [43] survival was 73 months.

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