Passamonti Francesco, Rumi Elisa, Arcaini Luca, Boveri Emanuela, Elena Chiara, Pietra Daniela, Boggi Sabrina, Astori Cesare, Bernasconi Paolo, Varettoni Marzia, Brusamolino Ercole, Pascutto Cristiana, Lazzarino Mario
M.D., Division of Hematology, University of Pavia, Fondazione IRCCS Policlinico San Matteo, viale Golgi 19, 27100 Pavia, Italy.
Haematologica. 2008 Nov;93(11):1645-51. doi: 10.3324/haematol.13346. Epub 2008 Sep 11.
Essential thrombocythemia is a chronic myeloproliferative disorder; patients with this disorder have a propensity to develop thrombosis, myelofibrosis, and leukemia.
We studied 605 patients with essential thrombocythemia (follow-up 4596 person-years) with the aim of defining prognostic factors for thrombosis, myelofibrosis, and leukemia during follow-up.
Sixty-six patients (11%) developed thrombosis with a 10-year risk of 14%. Age >60 years (p<0.001) and a history of thrombosis (p=0.03) were independent risk factors for thrombosis. Progression to myelofibrosis occurred in 17 patients (2.8%) with a 10-year risk of 3.9%. Anemia at diagnosis of essential thrombocythemia was significantly correlated (p<0.001) with progression to myelofibrosis. Leukemia occurred in 14 patients (2.3%) at a median time of 11 years after the diagnosis of essential thrombocythemia; the risk was 2.6% at 10 years. Age >60 years (p=0.02) was significantly correlated with the development of leukemia. Cytotoxic treatment did not imply a higher risk of leukemia. At the time of the analysis, 64 of the 605 patients (10.6%) had died. The 10-year probability of survival was 88%, with a median survival of 22.3 years. Age >60 years (p<0.001) and history of thrombosis (p=0.001) were independent risk factors for survival.
The findings from this study on a large series of patients treated according to current clinical practice provide reassurance that essential thrombocythemia is an indolent disorder and affected patients have a long survival. The main risk is thrombosis, while myelofibrosis and leukemia are rare and late complications.
原发性血小板增多症是一种慢性骨髓增殖性疾病；患有这种疾病的患者有发生血栓形成、骨髓纤维化和白血病的倾向。
我们研究了605例原发性血小板增多症患者（随访4596人年），目的是确定随访期间血栓形成、骨髓纤维化和白血病的预后因素。
66例患者（11%）发生血栓形成，10年风险为14%。年龄>60岁（p<0.001）和有血栓形成史（p=0.03）是血栓形成的独立危险因素。17例患者（2.8%）进展为骨髓纤维化，10年风险为3.9%。原发性血小板增多症诊断时的贫血与进展为骨髓纤维化显著相关（p<0.001）。14例患者（2.3%）发生白血病，诊断原发性血小板增多症后中位时间为11年；10年风险为2.6%。年龄>60岁（p=0.02）与白血病的发生显著相关。细胞毒性治疗并不意味着白血病风险更高。在分析时，605例患者中有64例（10.6%）死亡。10年生存概率为88%，中位生存期为22.3年。年龄>60岁（p<0.001）和有血栓形成史（p=0.001）是生存的独立危险因素。
这项针对大量按照当前临床实践治疗的患者的研究结果表明，原发性血小板增多症是一种惰性疾病，受影响患者生存期长，这让人放心。主要风险是血栓形成，而骨髓纤维化和白血病是罕见的晚期并发症。
