Pipobroman is safe and effective treatment for patients with essential thrombocythaemia at high risk of thrombosis

Francesco Passamonti, Lucia Malabarba, Ester Orlandi, Cristiana Pascutto, Ercole Brusamolino, Cesare Astori, Claudia Baratè, Angelo Canevari, Alessandro Corso, Paolo Bernasconi, Mario Cazzola, Mario Lazzarino

Research output: Contribution to journalArticle

Abstract

Essential thrombocythaemia (ET) is a disease associated with an elevated risk of thrombosis. This study evaluated the efficacy and safety of pipobroman (PB) in the long-term control of ET patients who had, at diagnosis, one or more of the following currently known risk factors for thrombosis or haemorrhage (high-risk patients): age > 60 years, history of thrombosis or haemorrhage, platelets > 1000 × 10 9/l. From 1978 to 2000, with a median follow-up of 10 years, 118 previously untreated high-risk ET patients (median age 62 years, range 25-82), were treated with PB at the starting dose of 0.8-1 mg/kg/d. All patients reached a platelet count <600 × 10 9/l and 91% achieved a platelet count <400 × 10 9/l. During follow-up, 13 patients had thrombosis, with a 10-year cumulative risk of 14%. Acute myeloid leukaemia, myelofibrosis and solid tumours occurred in three, two and seven patients with a 10-year cumulative risk of 3%, 2% and 7% respectively. Actuarial survival at 20 years was 64% and the standardized mortality ratio was 1.1 (95% CI: 0.7-1.7), not statistically different from the general population (P = 0.54). Age was associated with a higher risk of death (P = 0.00009) and thrombosis (P = 0.003). The duration of PB treatment did not correlate with the occurrence of second malignancies. This study, with a median follow-up of 10 years, demonstrates that pipobroman is effective and well tolerated. The low cumulative 10-year risk of thrombosis, leukaemia and solid tumours indicates that pipobroman is an adequate treatment for patients with high risk ET.

Original languageEnglish
Pages (from-to)855-861
Number of pages7
JournalBritish Journal of Haematology
Volume116
Issue number4
DOIs
Publication statusPublished - 2002

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Keywords

  • Chronic myeloproliferative disorders
  • Essential thrombocythaemia
  • Pipobroman
  • Second malignancies
  • Thrombosis

ASJC Scopus subject areas

  • Hematology

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