Long-term outcomes to fludarabine and rituximab in Waldenström macroglobulinemia Academic Article uri icon

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  • AbstractWe report the long-term outcome of a multicenter, prospective study examining fludarabine and rituximab in Waldenström macroglobulinemia (WM). WM patients with less than 2 prior therapies were eligible. Intended therapy consisted of 6 cycles (25 mg/m2 per day for 5 days) of fludarabine and 8 infusions (375 mg/m2 per week) of rituximab. A total of 43 patients were enrolled. Responses were: complete response (n = 2), very good partial response (n = 14), partial response (n = 21), and minor response (n = 4), for overall and major response rates of 95.3% and 86.0%, respectively. Median time to progression for all patients was 51.2 months and was longer for untreated patients (P = .017) and those achieving at least a very good partial response (P = .049). Grade 3 or higher toxicities included neutropenia (n = 27), thrombocytopenia (n = 7), and pneumonia (n = 6), including 2 patients who died of non–Pneumocystis carinii pneumonia. With a median follow-up of 40.3 months, we observed 3 cases of transformation to aggressive lymphoma and 3 cases of myelodysplastic syndrome/acute myeloid leukemia. The results of this study demonstrate that fludarabine and rituximab are highly active in WM, although short- and long-term toxicities need to be carefully weighed against other available treatment options. This study is registered at clinicaltrials.gov as NCT00020800.


  • Treon, Steven P
  • Branagan, Andrew R
  • Ioakimidis, Leukothea
  • Soumerai, Jacob D
  • Patterson, Christopher J
  • Turnbull, Barry
  • Wasi, Parveen
  • Emmanouilides, Christos
  • Frankel, Stanley R
  • Lister, Andrew
  • Morel, Pierre
  • Matous, Jeffrey
  • Gregory, Stephanie A
  • Kimby, Eva

publication date

  • April 16, 2009

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