Waldenström's macroglobulinaemia: results of a combined oral treatment in 34 newly diagnosed patients

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Abstract
Petrucci MT, Awisati G, Tribalto M, Giovangrossi P, Mandelli F. (Section of Hematology, Department of Human Biopathology, University ‘La Sapienza’ and Division of Hematology, S. Eugenio Hospital, University ‘Tor Vergata’, Rome, Italy). Waldenström's macroglobulinaemia: results of a combined oral treatment in 34 newly diagnosed patients. From February 1978 to September 1987, 34 patients with Waldenström's macroglobulinaemia (WM) were treated using an oral combination consisting of melphalan (6 mg m, days−2 1–7) cyclophosphamide (125 mg m−2, days 1–7) and prednisone (40 mg m−2, days 1–7). Courses were repeated every 4–6 weeks for a total of 12 courses. After the completion of 12 courses, responding patients received continuous treatment with chlorambucil (3 mg m−2 d−1) and prednisone (6 mg m−2 d−1) until relapse. Following the induction, 23 of 31 evaluated patients (74%) have responded to induction therapy of whom eight (26% of the 31 evaluated patients) achieved complete remission defined as disappearance of all clinical and laboratory features characteristic of WM. The overall median response duration, as of December 1988, has not yet been reached, while the overall median event-free survival duration for the entire population and for responding patients is 66 months in both groups. However, in responding patients the event-free survival duration after 66 months reaches a plateau. Toxicity was limited to transient nausea and vomiting (grade 2 according to WHO). In conclusion, this study demonstrates the efficacy and safety of an oral polychemotherapeutic treatment in the management of Waldenström's disease.