POLYCYTHEMIA-VERA - THE NATURAL-HISTORY OF 1213 PATIENTS FOLLOWED FOR 20 YEARS
POLYCYTHEMIA-VERA - THE NATURAL-HISTORY OF 1213 PATIENTS FOLLOWED FOR 20 YEARS
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DOI：
10.7326/0003-4819-123-9-199511010-00003
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发表时间：
1995-11-01
影响因子：
39.2
通讯作者：
ROSSIFERRINI, P
中科院分区：
文献类型：
--
作者：
RIUNITI, O;BARBUI, T;ROSSIFERRINI, P
目的：重新评估真性红细胞增多症的自然史，获得血栓发生率和生存率的可靠估计，用于确定治疗性临床试验的样本量。研究设计：对跟踪 20 年的红细胞增多症患者进行回顾性队列研究。地点：11 个意大利血液学机构。患者：1213 名真性红细胞增多症患者，根据真性红细胞增多症研究组制定的标准进行诊断，临床实践中常用。主要指标：全因死亡率、静脉和动脉血栓形成、血液和非血液肿瘤疾病。心肌梗塞和中风被列为主要血栓事件，静脉和外周动脉血栓被视为次要血栓事件。死亡患者人数和发生重大血栓事件（合并终点）的患者人数被用作与使用骨髓抑制剂相关的获益风险比的综合衡量标准。 结果：485 名患者（41%）中记录了 634 例致命和非致命性动脉和静脉血栓；其中 36% 的发作发生在 230 名患者的随访期间 (19%)，64% 发生在就诊时或诊断前。血栓事件在诊断前两年发生的频率更高，表明潜在的骨髓增生性疾病与血管事件之间存在因果关系。随访期间血栓发生率为3.4%/年；老年患者或有血栓病史的患者发生血栓的风险较高。每年总死亡率为 2.9/100 名患者；血栓事件和血液或非血液癌症对死亡率有类似的影响。接受化疗的患者死亡率是未接受化疗患者的三到四倍。接受骨髓抑制剂治疗的患者患癌症的风险在诊断后大约 6 年出现增加。此外，以最难发生的事件（死亡、非致命性心肌梗死或中风）的总和计算的综合终点表明，骨髓抑制剂具有总体不利的影响。 结论：细胞减灭术有利于血栓事件的发生率，但积极的治疗似乎与肿瘤风险增加有关。这些结果为重新评估真性红细胞增多症患者的治疗策略和估计旨在测试新治疗方法的临床试验的规模提供了基础。
Objective: To reassess the natural history of polycythemia vera and to obtain reliable estimates of both incidence of thrombosis and survival for use in defining the sample size for therapeutic clinical trials.Study Design: Retrospective cohort study of patients with polycythemia who had been followed for 20 years.Setting: 11 Italian hematology institutions.Patients: 1213 patients with polycythemia vera, which was diagnosed according to criteria established by the Polycythemia Vera Study Group and commonly used in clinical practice.Main Outcome Measures: All-cause mortality, venous and arterial thrombosis, and hematologic and nonhematologic neoplastic disease. Myocardial infarction and stroke were classified as major thrombotic events, and venous and peripheral arterial thrombosis were considered minor thrombotic events. The number of patients who died and the number of those who had major thrombotic events (combined end point) were used as a comprehensive measure of the benefit-risk ratio associated with the use of myelosuppressive agents.Results: 634 fatal and nonfatal arterial and venous thromboses were recorded in 485 patients (41%); 36% of these episodes occurred during follow-up in 230 patients (19%), and 64% occurred either at presentation or before diagnosis. Thrombotic events occurred more frequently in the 2 years preceding diagnosis, suggesting a causal relation between the latent myeloproliferative disorder and the vascular event. The incidence of thrombosis during follow-up was 3.4%/y; older patients or those with a history of thrombosis had a higher risk for thrombosis. Overall mortality was 2.9/100 patients per year; thrombotic events and hematologic or nonhematologic cancers had similar effects on mortality. Patients receiving chemotherapy died three to four times more frequently than those not receiving chemotherapy. The increased risk for cancer in patients receiving myelosuppressive agents was seen approximately 6 years after diagnosis. In addition, the combined end point, computed as the sum of the hardest available events (death, nonfatal myocardial infarction, or stroke), suggests that myelosuppressive agents have an overall unfavorable effect.Conclusions: Cytoreduction favorably affects the incidence of thrombotic events, but aggressive treatment seems to be associated with increased risk for neoplasm. These results provide a basis for reevaluating the therapeutic strategy in patients with polycythemia vera and for estimating the size of clinical trials aimed at testing new therapeutic approaches.