title: Long-term evaluation of 164 patients with essential thrombocythaemia treated with pipobroman: occurrence of leukaemic evolution.
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 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.
authors: Radiotherapy Oncology, Department of Radiology, Università di Roma, Roma, Italy. dsvita@libero.it
authors: Vitaliana
authors: V
authors: De Sanctis
authors: Maria Gabriella
authors: MG
authors: Mazzucconi
authors: Antonio
authors: A
authors: Spadea
authors: Marco
authors: M
authors: Alfò
authors: Marco
authors: M
authors: Mancini
authors: Luisa
authors: L
authors: Bizzoni
authors: Monica
authors: M
authors: Peraino
authors: Franco
authors: F
authors: Mandelli
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000477:Alkylating Agents / Q000627:therapeutic use*; D018450:Disease Progression; D005260:Female; D006801:Humans; D007938:Leukemia / Q000517:prevention & control; D008297:Male; D008875:Middle Aged; D010885:Pipobroman / Q000627:therapeutic use*; D012189:Retrospective Studies; D013920:Thrombocythemia, Essential / Q000188:drug therapy*
chemical_list: D000477:Alkylating Agents; D010885:Pipobroman