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羟基脲治疗真性红细胞增多症:一项对100例患者进行的为期20年的前瞻性研究。

Hydroxyurea in the treatment of polycythemia vera: a prospective study of 100 patients over a 20-year period.

作者信息

West W O

出版信息

South Med J. 1987 Mar;80(3):323-7. doi: 10.1097/00007611-198703000-00012.

DOI:10.1097/00007611-198703000-00012
PMID:3824016
Abstract

From 1963 to 1983, I treated 100 patients with polycythemia vera, using phlebotomy and the adjunctive agent hydroxyurea. These 78 male and 22 female patients ranged in age from 24 to 88 years (mean 55.7). Duration of therapy ranged from three to 216 months (mean 64.9). The mean daily dose was 0.72 gm, and the median dose was 0.64 gm. Hydroxyurea gave adequate control of red cells, platelets, and spleen size. Cytopenia was not observed. Phlebotomy requirements were markedly reduced. Leukocyte alkaline phosphatase scores were generally lowered and several blood chemistry values returned to normal. Side effects were minimal, and there were no drug-related deaths. Infections were not a problem. Hydroxyurea, a metabolic inhibitor of desoxyribonucleic acid, does not interfere with the synthesis of ribonucleic acid or protein and is thus probably less leukemogenic than radioactive phosphorus and alkylating agents. Acute myelogenous leukemia was seen in one patient after five years of continuous hydroxyurea therapy. He had received no other myelosuppressant agent. Because hydroxyurea is safe and effective in the treatment of polycythemia vera, it should be considered as first-line therapy. It probably offers practical and theoretic advantages over present therapy particularly when the disease is not well controlled by phlebotomy alone.

摘要

1963年至1983年期间,我使用放血疗法及辅助药物羟基脲治疗了100例真性红细胞增多症患者。这些患者中,男性78例,女性22例,年龄在24岁至88岁之间(平均55.7岁)。治疗时间为3个月至216个月(平均64.9个月)。平均每日剂量为0.72克,中位剂量为0.64克。羟基脲能充分控制红细胞、血小板及脾脏大小。未观察到血细胞减少。放血需求显著减少。白细胞碱性磷酸酶评分普遍降低,多项血液化学指标恢复正常。副作用极小,且无药物相关死亡病例。感染并非问题。羟基脲作为脱氧核糖核酸的代谢抑制剂,并不干扰核糖核酸或蛋白质的合成,因此其致白血病的可能性可能低于放射性磷和烷化剂。1例患者在接受羟基脲持续治疗5年后发生急性髓性白血病。他未接受过其他骨髓抑制药物治疗。由于羟基脲在治疗真性红细胞增多症方面安全有效,应将其视为一线治疗方法。相较于目前的治疗方法,它可能具有实际和理论上的优势,尤其是当疾病仅通过放血疗法无法得到良好控制时。

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