Long-term evaluation of 164 patients with essential thrombocythaemia treated with pipobroman

Occurrence of leukaemic evolution

Vitaliana De Sanctis, Maria Gabriella Mazzucconi, Antonio Spadea, Marco Alfò, Marco Mancini, Luisa Bizzoni, Monica Peraino, Franco Mandelli

Research output: Contribution to journalArticle

26 Citations (Scopus)

Abstract

Essential thrombocythaemia (ET) is usually considered an indolent disease, but it may progress during its natural course into acute leukaemia (AL); however, an influence of myelosuppressive agents in the blastic transformation of ET cannot be excluded. We performed a retrospective study to assess the incidence of AL in ET patients treated with pipobroman (PB) as first-line therapy. One hundred and sixty-four patients with ET were managed with PB at a dose of 1 mg/kg/d until a stable platelet count below 400 x 109/l was achieved. Maintenance therapy was given at a planned dose ranging between 0.2 and 1 mg/kg/d according to platelet count, in all cases, with a median daily dose of 25 mg (range 7-75 mg/d). The median treatment time was 100 months (range 25-243 months). The patients were evaluated for the occurrence of AL and/or secondary malignancies and survival end-points. AL was observed in nine patients (5.5%) after a median treatment time of 153 months (range 79-227 months). The overall survival (OS) and the event-free survival (EFS) at 120 months were 95% and 97%, whereas at 180 months, they were 84% and 76% respectively. In conclusion, this retrospective analysis shows a low incidence of AL in a large group of patients consecutively treated with PB as first-line chemotherapy. Therefore, an investigation of the role of myelosuppressive agents in the blastic transformation of ET would be of interest.

Original languageEnglish
Pages (from-to)517-521
Number of pages5
JournalBritish Journal of Haematology
Volume123
Issue number3
DOIs
Publication statusPublished - Nov 2003

Fingerprint

Pipobroman
Essential Thrombocythemia
Leukemia
Platelet Count
Survival
Incidence
Therapeutics
Disease-Free Survival
Retrospective Studies
Drug Therapy

Keywords

  • Essential thrombocythaemia
  • Leukaemic evolution
  • Myeloproliferative disorders
  • Pipobroman

ASJC Scopus subject areas

  • Hematology

Cite this

Long-term evaluation of 164 patients with essential thrombocythaemia treated with pipobroman : Occurrence of leukaemic evolution. / De Sanctis, Vitaliana; Mazzucconi, Maria Gabriella; Spadea, Antonio; Alfò, Marco; Mancini, Marco; Bizzoni, Luisa; Peraino, Monica; Mandelli, Franco.

In: British Journal of Haematology, Vol. 123, No. 3, 11.2003, p. 517-521.

Research output: Contribution to journalArticle

De Sanctis, Vitaliana ; Mazzucconi, Maria Gabriella ; Spadea, Antonio ; Alfò, Marco ; Mancini, Marco ; Bizzoni, Luisa ; Peraino, Monica ; Mandelli, Franco. / Long-term evaluation of 164 patients with essential thrombocythaemia treated with pipobroman : Occurrence of leukaemic evolution. In: British Journal of Haematology. 2003 ; Vol. 123, No. 3. pp. 517-521.
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