title: Long-term follow-up of 386 consecutive patients with essential thrombocythemia: safety of cytoreductive therapy.
abstract: Cytotoxic agents like Hydroxyurea, Busulfan and Interferon-alpha are to date the most commonly used therapeutic approaches in Essential Thrombocythemia (ET). However, few data on the efficacy and safety of these agents in the long-term are currently available. We report a retrospective analysis of the long-term outcome of 386 consecutive ET patients, followed at single Institution for a median follow-up of 9.5 years (range, 3-28.5). Cytoreductive therapy was administered to 338 patients (88%), obtaining a response in 86% of cases. Forty-five patients (12%) experienced a thrombosis. Among baseline characteristics, only history of vascular events prior to ET diagnosis predicted a higher incidence of thrombosis. Evolution in acute leukemia/myelofibrosis occurred in 6 (1,5%) and 20 (5%) patients, and was significantly higher in patients receiving sequential cytotoxic agents. Overall survival was 38% at 19 years and was poorer for patients older than 60 years, with higher leukocytes count (>15 x 10(9)/L), hypertension and mellitus diabetes at ET diagnosis and for patients experiencing a thrombotic event during follow-up. Cytoreductive therapy was effective in decreasing platelet number with negligible toxicity; however, thrombocytosis control did not reduce the incidence of thrombosis and, for patients who received sequential therapies, the probability of disease evolution was higher and survival was poorer.
authors.affiliation: Department of Hematology and Medical Oncology L. e A. Seràgnoli, St. Orsola-Malpighi Hospital, University of Bologna, Bologna, Italy.
authors.forename: Francesca
authors.initials: F
authors.lastname: Palandri
authors.forename: Lucia
authors.initials: L
authors.lastname: Catani
authors.forename: Nicoletta
authors.initials: N
authors.lastname: Testoni
authors.forename: Emanuela
authors.initials: E
authors.lastname: Ottaviani
authors.forename: Nicola
authors.initials: N
authors.lastname: Polverelli
authors.forename: Mauro
authors.initials: M
authors.lastname: Fiacchini
authors.forename: Antonio
authors.initials: A
authors.lastname: De Vivo
authors.forename: Federica
authors.initials: F
authors.lastname: Salmi
authors.forename: Alessandro
authors.initials: A
authors.lastname: Lucchesi
authors.forename: Michele
authors.initials: M
authors.lastname: Baccarani
authors.forename: Nicola
authors.initials: N
authors.lastname: Vianelli
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D015897:Comorbidity; D054697:Cytostatic Agents / Q000008:administration & dosage / Q000009:adverse effects* / Q000627:therapeutic use; D003920:Diabetes Mellitus / Q000453:epidemiology; D018450:Disease Progression; D004359:Drug Therapy, Combination; D005260:Female; D005500:Follow-Up Studies; D006470:Hemorrhage / Q000139:chemically induced; D006801:Humans; D006973:Hypertension / Q000453:epidemiology; D015994:Incidence; D008297:Male; D008875:Middle Aged; D016609:Neoplasms, Second Primary / Q000453:epidemiology / Q000209:etiology; D010975:Platelet Aggregation Inhibitors / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D012189:Retrospective Studies; D016019:Survival Analysis; D013920:Thrombocythemia, Essential / Q000150:complications / Q000188:drug therapy* / Q000401:mortality; D019851:Thrombophilia / Q000188:drug therapy / Q000209:etiology; D013927:Thrombosis / Q000453:epidemiology / Q000209:etiology / Q000517:prevention & control
chemical_list: D054697:Cytostatic Agents; D010975:Platelet Aggregation Inhibitors