title: Treatment of polycythemia vera and essential thrombocythemia: the role of pipobroman.
abstract: Pipobroman (PB) is a neutral amide of piperazine with a chemical structure close to that of alkylating agents, although the exact mechanism of action of PB has not been demonstrated. PB has well documented clinical activity in polycythemia vera (PV) and essential thrombocythemia (ET). Recent long-term follow-up studies on PV and ET patients receiving PB have facilitated the definition of the risk of late transformation into myelofibrosis with myeloid metaplasia (MMM) or acute leukemia (AL). This report gives an overview of the treatment with PB in patients with PV and ET focusing on clinical activity, administration dose and schedule, toxicity, impact on short- and long-term complications. From our experience and from the data reported in the literature the high clinical activity of PB in both PVand ET becomes evident. This drug allows, within 3 months, to attain a response in more than 90% of patients, without clinically relevant toxicities. The 10-years risk of thrombosis of patients treated with PB is about 15%, similar to that registered with hydroxyurea, the most widely used agent in PVand ET. The antiproliferative activity of PB on bone marrow megakaryocytes seems of particular value in lowering the occurrence of post-PV and post-ET MMM, whose risk (< 4% at 10 years) is the lowest registered with available treatments. The 10-year risk of acute leukemia with PB is 5% in PVand 3% in ET, which is only slightly higher than that expected as a natural evolution of the disease. In conclusion, the use of PB is a definite alternative to hydroxyurea in patients with PV and ET at high risk of thrombosis.
authors.affiliation: Division of Hematology, University of Pavia, IRCCS Policlinico San Matteo, Viale Golgi 19, 27100, Pavia, Italy. f.passamonti@smatteo.pv.it
authors.forename: Francesco
authors.initials: F
authors.lastname: Passamonti
authors.forename: Mario
authors.initials: M
authors.lastname: Lazzarino
mesh_terms: D000022:Abortion, Spontaneous / Q000209:etiology; D000208:Acute Disease; D000477:Alkylating Agents / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D007951:Leukemia, Myeloid / Q000139:chemically induced; D008875:Middle Aged; D010885:Pipobroman / Q000009:adverse effects / Q000494:pharmacology / Q000627:therapeutic use*; D011087:Polycythemia Vera / Q000150:complications / Q000188:drug therapy*; D011247:Pregnancy; D011250:Pregnancy Complications, Hematologic / Q000188:drug therapy; D055728:Primary Myelofibrosis / Q000139:chemically induced; D013920:Thrombocythemia, Essential / Q000150:complications / Q000188:drug therapy*; D013927:Thrombosis / Q000209:etiology / Q000517:prevention & control
chemical_list: D000477:Alkylating Agents; D010885:Pipobroman