Abstract

Treatment of Waldenström’s macroglobulinemia (WM) is based on the use of oral alkylating agents, even though recently, more expensive drugs have been proposed for its treatment.The aim of this study was to report the results obtained combining oral melphalan, cyclophosphamide and prednisone (MCP) in 72 WM patients, and to compare the results and related costs with those observed using more aggressive and expensive protocols. Between July 1973 and April 2002, we have observed 100 newly diagnosed WM, of whom 72 were symptomatic and therefore requiring a treatment. The antineoplastic treatment consisted of melphalan (6 mg/m2, days 1–7), cyclofosfamide (125 mg/m2, days 1–7) and prednisone (40 mg/m2, days 1–7) administered orally for a total of 12 courses. Thereafter, responding and stable patients were maintained with chlorambucil (3 mg/m2) and prednisone ( 6 mg/m2) until progression. Levels ≥1.5 g/dL of IgM immunoglobulin associated to 30% of lymphoplasmocytic cells in the bone marrow aspirate were required for diagnosing WM. Among these patients, 46 (63%) were men and 26 (47%) were women. Median age, IgM Immunoglobulin and Hb levels were at diagnosis: 61 years (range 42–87 years), 3.8 gr/dl ( range 1.5–6.5) and 12 gr/dl ( range 6.5 – 16) respectively.

Of these patients, we evaluated response rate, overall survival (OS), response duration (RD), freedom from progression (FFP), event free survival (EFS) duration, toxicities and costs/course in € of the drugs utilized.Of the 72 treated patients,71 (98.6%) were evaluable and 55 (77.5%) achieved a response, 7 (9.9%) showed a disease stabilization and 9 (12.6%) disease progression. After a median follow-up of 72 months (range 3–195 months), median OS, RD, FFP and EFS were: 66, 64, 55 and 47 months, respectively. No grade III or IV WHO toxicities were observed and toxicity was limited to transient nausea, vomiting and mild neutropenia. The cost/course for MCP was € 13, similar to that of standard protocols based on chlorambucil, and much less expensive compared to the costs of more aggressive protocols proposed for the treatment WM, ranging from € 74 to € 3260.90.In conclusion,the MCP is, as chlorambucil based protocols, a cost effective and safe option for treating patients with WM. Moreover, the obtained results do not appear inferior to those so far reported with more expensive, aggressive and toxic intravenous protocols.

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