TY - JOUR
T1 - Melphalan treatment in patients with myelofibrosis with myeloid metaplasia
AU - Petti, Maria Concetta
AU - Latagliata, R.
AU - Spadea, T.
AU - Spadea, A.
AU - Montefusco, E.
AU - Aloe Spiriti, M. A.
AU - Avvisati, G.
AU - Breccia, M.
AU - Pescarmona, E.
AU - Mandelli, F.
PY - 2002
Y1 - 2002
N2 - 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.
AB - 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.
KW - Blastic phase
KW - Melphalan
KW - Myelofibrosis with myeloid metaplasia
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U2 - 10.1046/j.0007-1048.2001.03331.x
DO - 10.1046/j.0007-1048.2001.03331.x
M3 - Article
C2 - 11849213
AN - SCOPUS:20244370678
VL - 116
SP - 576
EP - 581
JO - British Journal of Haematology
JF - British Journal of Haematology
SN - 0007-1048
IS - 3
ER -