Melphalan treatment in patients with myelofibrosis with myeloid metaplasia

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  20 × 109/l and/or platelets > 1·0 × 109/l] received low‐dose Melphalan (2·5 mg/3 times/week) to eval...

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Veröffentlicht in:British journal of haematology 2002-03, Vol.116 (3), p.576-581
Hauptverfasser: Petti, M. C., Latagliata, R., Spadea, T., Spadea, A., Montefusco, E., Aloe Spiriti, M. A., Avvisati, G., Breccia, M., Pescarmona, E., Mandelli, F.
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container_end_page 581
container_issue 3
container_start_page 576
container_title British journal of haematology
container_volume 116
creator Petti, M. C.
Latagliata, R.
Spadea, T.
Spadea, A.
Montefusco, E.
Aloe Spiriti, M. A.
Avvisati, G.
Breccia, M.
Pescarmona, E.
Mandelli, F.
description 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  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.
doi_str_mv 10.1046/j.0007-1048.2001.03331.x
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C. ; Latagliata, R. ; Spadea, T. ; Spadea, A. ; Montefusco, E. ; Aloe Spiriti, M. A. ; Avvisati, G. ; Breccia, M. ; Pescarmona, E. ; Mandelli, F.</creator><creatorcontrib>Petti, M. C. ; Latagliata, R. ; Spadea, T. ; Spadea, A. ; Montefusco, E. ; Aloe Spiriti, M. A. ; Avvisati, G. ; Breccia, M. ; Pescarmona, E. ; Mandelli, F.</creatorcontrib><description>Between January 1985 and December 1992, 104 consecutive patients with symptomatic myelofibrosis with myeloid metaplasia (MMM) [splenic enlargement &gt; 5 cm and/or transfusional requirement or Hb &lt; 10 g/dl and/or white blood cell (WBC) count &gt; 20 × 109/l and/or platelets &gt; 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 &gt; 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 &gt; 20 × 109/l (HR = 2·4) and not achieving any type of response, either partial or complete (HR = 3·9). 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C.</creatorcontrib><creatorcontrib>Latagliata, R.</creatorcontrib><creatorcontrib>Spadea, T.</creatorcontrib><creatorcontrib>Spadea, A.</creatorcontrib><creatorcontrib>Montefusco, E.</creatorcontrib><creatorcontrib>Aloe Spiriti, M. 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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 &gt; 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 &gt; 20 × 109/l (HR = 2·4) and not achieving any type of response, either partial or complete (HR = 3·9). 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A.</au><au>Avvisati, G.</au><au>Breccia, M.</au><au>Pescarmona, E.</au><au>Mandelli, F.</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Melphalan treatment in patients with myelofibrosis with myeloid metaplasia</atitle><jtitle>British journal of haematology</jtitle><addtitle>Br J Haematol</addtitle><date>2002-03-01</date><risdate>2002</risdate><volume>116</volume><issue>3</issue><spage>576</spage><epage>581</epage><pages>576-581</pages><issn>0007-1048</issn><eissn>1365-2141</eissn><coden>BJHEAL</coden><abstract>Between January 1985 and December 1992, 104 consecutive patients with symptomatic myelofibrosis with myeloid metaplasia (MMM) [splenic enlargement &gt; 5 cm and/or transfusional requirement or Hb &lt; 10 g/dl and/or white blood cell (WBC) count &gt; 20 × 109/l and/or platelets &gt; 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 &gt; 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 &gt; 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.</abstract><cop>Oxford, UK</cop><pub>Blackwell Science, Ltd</pub><pmid>11849213</pmid><doi>10.1046/j.0007-1048.2001.03331.x</doi><tpages>6</tpages><oa>free_for_read</oa></addata></record>
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subjects Adult
Aged
Aged, 80 and over
Anemia - complications
Antineoplastic agents
Antineoplastic Agents, Alkylating - therapeutic use
Biological and medical sciences
blastic phase
Chemotherapy
Female
Follow-Up Studies
Hematology
Humans
Leukocyte Count
Male
Medical sciences
Melphalan
Melphalan - therapeutic use
Middle Aged
myelofibrosis with myeloid metaplasia
Pharmacology. Drug treatments
Primary Myelofibrosis - blood
Primary Myelofibrosis - drug therapy
Prognosis
Survival Rate
Treatment Outcome
title Melphalan treatment in patients with myelofibrosis with myeloid metaplasia
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