Melphalan treatment in patients with myelofibrosis with myeloid metaplasia

Maria Concetta Petti, R. Latagliata, T. Spadea, A. Spadea, E. Montefusco, M. A. Aloe Spiriti, G. Avvisati, M. Breccia, E. Pescarmona, F. Mandelli

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Between January 1985 and December 1992, 104 consecutive patients with symptomatic myelofibrosis with myeloid metaplasia (MMM) [splenic enlargement > 5 cm and/or transfusional requirement or Hb <10 g/dl and/or white blood cell (WBC) count > 20 × 109/l and/or platelets > 1.0 × 109/l] received low-dose Melphalan (2.5 mg/3 times/week) to evaluate the efficacy and toxicity of this approach. Among 99 evaluable patients, 66 (66.7%) achieved a response after a median time of 6.7 months: 26 (26.3%) had a normalization of all clinical and haematological parameters (complete response, CR) and 40 (40.4%) showed an improvement > 50% (partial response, PR). Thirty-three patients (33.3%) were resistant. Reversible haematological toxicity was the most common complication. Median durations of CR and PR were 28.4 and 26 months respectively: median survival of CR + PR patients was 71.2 months (95%CI: 33.8-108.7) versus 36.5 months (95%CI: 24.5-48.5) for the non-responders (log-rank test, P = 0.002). In the multivariate analysis, the following variables were significantly associated with a shorter survival: anaemia [hazard risk (HR) = 2.7], WBC count > 20 × 109/l (HR = 2.4) and not achieving any type of response, either partial or complete (HR = 3.9). In conclusion, Melphalan could be a promising first-line option for MMM patients with clinical or haematological symptoms requiring treatment.

Original languageEnglish
Pages (from-to)576-581
Number of pages6
JournalBritish Journal of Haematology
Issue number3
Publication statusPublished - 2002


  • Blastic phase
  • Melphalan
  • Myelofibrosis with myeloid metaplasia

ASJC Scopus subject areas

  • Hematology


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