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

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

Keywords

  • Essential thrombocythaemia
  • Leukaemic evolution
  • Myeloproliferative disorders
  • Pipobroman

ASJC Scopus subject areas

  • Hematology

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