Subcutaneous Bortezomib, Cyclophosphamide and Rituximab (BCR) Versus Fludarabine, Cyclophosphamide and Rituximab (FCR) for Initial Therapy of Waldenstrőm's Macroglobulinaemia (WM): a Randomized Phase II Trial
Waldenstrom macroglobulinaemia (WM) is a low grade nonHodgkin lymphoma characterised by bone
marrow infiltration and the presence of an abnormal protein in the blood (IgM paraprotein.
Most patients require treatment at presentation but there is no agreed standard of first
line therapy. Current treatment is unsatisfactory with responses often incomplete and slow
to attain, while recurrence is inevitable.
The aim of this study is to find out whether a new combination of Bortezomib (Velcade®),
Cyclophosphamide and Rituximab (MabThera), is well tolerated and effective for patients with
WM. R2W is a randomised, noncomparative, phase II trial of subcutaneous bortezomib,
cyclophosphamide, rituximab (BCR, experimental arm) versus fludarabine, cyclophosphamide,
rituximab (FCR, control arm) for initial therapy of WM. This is a two stage trial where six
patients will be treated initially with BCR to assess tolerability. If BCR is considered
tolerable, a further 50 patients will be randomised between BCR and FCR (2:1) in the second
stage of the trial. Patients will receive 3 cycles of treatment and then be reassessed.
Those with evidence of progression will stop trial treatment. All other patients will
continue with a further 3 cycles (to a total of 6) unless there is a clear clinical
contraindication to further treatment.
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Number and percentage of patients who achieve disease response
6 months (end of treatment)
United Kingdom: Medicines and Healthcare Products Regulatory Agency