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Pipobroman is safe and effective treatment for patients with essential thrombocythaemia at high risk of thrombosis
Author(s) -
Passamonti Francesco,
Malabarba Lucia,
Orlandi Ester,
Pascutto Cristiana,
Brusamolino Ercole,
Astori Cesare,
Baratè Claudia,
Canevari Angelo,
Corso Alessandro,
Bernasconi Paolo,
Cazzola Mario,
Lazzarino Mario
Publication year - 2002
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1046/j.0007-1048.2002.03367.x
Subject(s) - medicine , thrombosis , cumulative incidence , myelofibrosis , cumulative dose , surgery , population , gastroenterology , platelet , cohort , bone marrow , environmental health
Summary. 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.