Kiladjian Jean-Jacques, Gardin Claude, Renoux Michel, Bruno Franck, Bernard Jean-François
Service d'Hematologie Clinique, Hopital Beaujon, 100 Boulevard du General Leclerc, 92110 Clichy, France.
Hematol J. 2003;4(3):198-207. doi: 10.1038/sj.thj.6200250.
From 1968 to 1993, 179 newly diagnosed patients with polycythemia vera (PV) were enrolled in a prospective study using pipobroman as first chemotherapy. Among them, 140 fulfilled the Polycythemia Vera Study Group criteria for PV, and 39 patients (22%) can be considered as idiopathic erythrocytosis (IE). Vascular events occurred in 10% of IE and 20% of PV patients and solid tumors in 7.7% of IE and 12.8% of PV patients. There were no differences between PV and IE patients with regard to progression to myelofibrosis (MF), leukemic events and overall survival. Overall, 98.3% of patients initially responded to pipobroman, with very mild toxicity. A total of 164 PV patients who received more than 1 year of pipobroman were analyzed for long-term evolution. The actuarial risk of thrombosis was 15.6 and 23.8% at 10 and 18 years, respectively. In all, 21 patients developed a solid tumor during follow-up, added and/or switched drugs being a risk factor. Actuarial risk of MF was as low as 4.9 and 9.4% at 10 and 15 years, respectively. Actuarial risk of leukemia was 14.4 and 18.7% at 10 and 15 years, respectively. Hyperleukocytosis at diagnosis was the only variable significantly associated with higher risk of leukemia. The median survival was 15.5 years, with two initial adverse prognostic factors: age above 60 years and hyperleukocytosis. Despite an increasing risk of leukemia with time, survival was not lower when compared to the French matched population. Only age and hyperleukocytosis at diagnosis were found to have a prognostic value in PV.
1968年至1993年，179例新诊断的真性红细胞增多症（PV）患者被纳入一项前瞻性研究，使用马利兰作为一线化疗药物。其中，140例符合真性红细胞增多症研究组的PV标准，39例患者（22%）可被视为特发性红细胞增多症（IE）。血管事件在10%的IE患者和20%的PV患者中发生，实体瘤在7.7%的IE患者和12.8%的PV患者中发生。PV和IE患者在进展为骨髓纤维化（MF）、白血病事件和总生存期方面没有差异。总体而言，98.3%的患者最初对马利兰有反应，毒性非常轻微。对164例接受马利兰治疗超过1年的PV患者进行了长期演变分析。血栓形成的精算风险在10年和18年时分别为15.6%和23.8%。总共有21例患者在随访期间发生了实体瘤，加用和/或更换药物是一个危险因素。MF的精算风险在10年和15年时分别低至4.9%和9.4%。白血病的精算风险在10年和15年时分别为14.4%和18.7%。诊断时白细胞增多是与白血病高风险显著相关的唯一变量。中位生存期为15.5年，有两个初始不良预后因素：年龄超过60岁和白细胞增多。尽管随着时间的推移白血病风险增加，但与法国匹配人群相比，生存期并不更低。在PV中，仅发现诊断时的年龄和白细胞增多具有预后价值。
