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Treatment of 72 newly diagnosed Waldenström macroglobulinemia cases with oral melphalan, cyclophosphamide, and prednisone

Results and cost analysis

  1. Ombretta Annibali M.D.1,
  2. Maria Teresa Petrucci M.D.2,
  3. Vincenza Martini M.D.2,
  4. Maria Cristina Tirindelli M.D.1,
  5. Anna Levi M.D.2,
  6. Carolina Fossati M.D.1,
  7. Patrizia Del Bianco B.Sc.2,
  8. Franco Mandelli M.D.2,
  9. Robin Foa M.D.2,
  10. Giuseppe Avvisati M.D., Ph.D.1,†,*

Article first published online: 20 DEC 2004

DOI: 10.1002/cncr.20826

Issue

Cancer

Cancer

Volume 103, Issue 3, pages 582–587, 1 February 2005

Additional Information

How to Cite

Annibali, O., Petrucci, M. T., Martini, V., Tirindelli, M. C., Levi, A., Fossati, C., Bianco, P. D., Mandelli, F., Foa, R. and Avvisati, G. (2005), Treatment of 72 newly diagnosed Waldenström macroglobulinemia cases with oral melphalan, cyclophosphamide, and prednisone. Cancer, 103: 582–587. doi: 10.1002/cncr.20826

Author Information

  1. 1

    Dipartimento di Ematologia, Università Campus Bio-Medico, Rome, Italy

  2. 2

    Sezione di Ematologia, Dipartimento di Biotecnologie Cellulari ed Ematologia, Università La Sapienza, Rome, Italy

  1. †

    Fax: (011) 39 06 22541456

*Dipartimento di Ematologia, Università Campus Bio-Medico, Via Emilio Longoni 83, 00155 Rome, Italy

Publication History

  1. Issue published online: 20 JAN 2005
  2. Article first published online: 20 DEC 2004
  3. Manuscript Accepted: 18 OCT 2004
  4. Manuscript Revised: 6 OCT 2004
  5. Manuscript Received: 6 JUL 2004

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Keywords:

  • macroglobulinemia;
  • Waldenström macroglobulinemia;
  • immunoglobulin M monoclonal gammopathy;
  • melphalan;
  • cyclophosphamide;
  • prednisone;
  • therapy

Abstract

BACKGROUND

Current treatment regimens for Waldenström macroglobulinemia (WM) are based on the use of oral alkylating agents. Recently, however, other more costly agents have been proposed for the treatment of WM. In the current study, the authors report on results obtained using oral melphalan, cyclophosphamide, and prednisone (MCP) to treat 72 patients with WM, and they compare these results (and the associated costs) with those observed using more aggressive protocols.

METHODS

Between July 1973 and April 2002, the authors documented overexpression of the immunoglobulin M paraprotein in 317 consecutive patients. Of these, 100 had newly diagnosed WM, and the 72 who were symptomatic were treated using the MCP protocol. Response rate, overall survival (OS), response duration, freedom from progression (FFP), event-free survival (EFS) duration, toxicity, and cost per course in Euro and U.S. dollars were evaluated for patients receiving this regimen.

RESULTS

Seventy-one of 72 patients (99%) were evaluable. Of these patients, 55 (77%) achieved a response; 7 others (10%) experienced disease stabilization, and the remaining 9 (13%) experienced disease progression. After a median follow-up of 72 months (range, 3–195 months), the median durations of EFS, FFP, response, and OS were 47, 55, 64, and 66 months, respectively. No World Health Organization Grade III or IV toxicities were observed, and side effects were limited to transient nausea, emesis, and mild neutropenia. The cost per course of the MCP regimen was $16, similar to that of standard protocols involving chlorambucil and much less than that of more aggressive protocols (price range, $91–11091) proposed for the treatment of WM.

CONCLUSIONS

Like chlorambucil-based protocols, the MCP regimen is a cost-effective and safe option for the treatment of patients with WM. Furthermore, the results obtained do not appear to be inferior to those yielded by more expensive, aggressive, and toxic intravenous protocols. Cancer 2005. © 2004 American Cancer Society.

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