Berk P D, Goldberg J D, Silverstein M N, Weinfeld A, Donovan P B, Ellis J T, Landaw S A, Laszlo J, Najean Y, Pisciotta A V, Wasserman L R
N Engl J Med. 1981 Feb 19;304(8):441-7. doi: 10.1056/NEJM198102193040801.
In studies to determine the optimal treatment for polycythemia vera, 431 previously untreated patients whose disease met established diagnostic criteria were entered into a prospective, randomized controlled trial between 1967 and 1974. Three treatment regimens were used: phlebotomy alone, chlorambucil supplemented by phlebotomy, or radioactive phosphorus supplemented by phlebotomy. Despite minor differences in age and sex, the three groups were comparable in initial hematocrit, white-cell and platelet counts, and disease-related symptoms. The median duration of follow-up is now more than 6 1/2 years. As of February 15, 1980, there were no statistically significant differences in survival among the groups. However, the risk of acute leukemia in patients given chlorambucil was 2.3 times that in patients given radioactive phosphorus and 13 times that in patients treated with phlebotomy alone. The increased incidence of leukemia during chlorambucil treatment is statistically significant (P less than or equal to 0.002); accordingly, the Polycythemia Vera Study Group has discontinued the use of chlorambucil in the treatment of polycythemia vera.
在确定真性红细胞增多症最佳治疗方法的研究中，431例疾病符合既定诊断标准且此前未接受过治疗的患者于1967年至1974年期间进入一项前瞻性随机对照试验。采用了三种治疗方案：单纯放血疗法、苯丁酸氮芥联合放血疗法、放射性磷联合放血疗法。尽管三组患者在年龄和性别上存在细微差异，但在初始血细胞比容、白细胞和血小板计数以及与疾病相关的症状方面具有可比性。目前随访的中位时长超过6年半。截至1980年2月15日，各组之间的生存率无统计学显著差异。然而，接受苯丁酸氮芥治疗的患者发生急性白血病的风险是接受放射性磷治疗患者的2.3倍，是仅接受放血疗法治疗患者的13倍。苯丁酸氮芥治疗期间白血病发病率的增加具有统计学显著性（P小于或等于0.002）；因此，真性红细胞增多症研究组已停止使用苯丁酸氮芥治疗真性红细胞增多症。
