Premium
Long‐term evaluation of 164 patients with essential thrombocythaemia treated with pipobroman: occurrence of leukaemic evolution
Author(s) -
De Sanctis Vitaliana,
Mazzucconi Maria Gabriella,
Spadea Antonio,
Alfò Marco,
Mancini Marco,
Bizzoni Luisa,
Peraino Monica,
Mandelli Franco
Publication year - 2003
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.1365-2141.2003.04542.x
Subject(s) - medicine , incidence (geometry) , retrospective cohort study , gastroenterology , chemotherapy , surgery , neutropenia , physics , optics
Summary. 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 × 10 9 /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.