Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105-2794, USA.
Hematology Am Soc Hematol Educ Program. 2009:62-9. doi: 10.1182/asheducation-2009.1.62.
Clinical experience with hydroxyurea for patients with sickle cell disease (SCD) has been accumulating for the past 25 years. The bulk of the current evidence suggests that hydroxyurea is well-tolerated, safe, and efficacious for most patients with SCD. Hydroxyurea has proven clinical efficacy for reducing acute vaso-occlusive events including pain episodes and acute chest syndrome. Salutary effects on hematological parameters include increases in fetal hemoglobin (HbF), hemoglobin, and MCV; also significant decreases occur in WBC, ANC, reticulocytes, LDH, and bilirubin. Treatment with hydroxyurea is usually considered for patients with recurrent vaso-occlusive events, but additional indications for treatment may include laboratory markers of disease severity and evidence of chronic organ dysfunction. Ten years ago, the US Food and Drug Administration approved hydroxyurea for adult patients with clinically severe SCD; however, its use in children remains off-label. Despite the large body of evidence regarding its efficacy and safety, hydroxyurea is currently prescribed only sparingly for patients with SCD and therefore has only limited effectiveness for this disorder; barriers to its use need to be identified and overcome.
羟基脲治疗镰状细胞病（SCD）患者的临床经验已积累了 25 年。目前的大量证据表明，羟基脲对大多数 SCD 患者具有良好的耐受性、安全性和疗效。羟基脲已被证明对减少急性血管阻塞性事件（包括疼痛发作和急性胸部综合征）具有临床疗效。对血液学参数的有益影响包括胎儿血红蛋白（HbF）、血红蛋白和 MCV 的增加；白细胞计数、中性粒细胞计数、网织红细胞计数、乳酸脱氢酶和胆红素也显著降低。对于经常发生血管阻塞性事件的患者，通常会考虑使用羟基脲进行治疗，但治疗的其他适应证可能包括疾病严重程度的实验室标志物和慢性器官功能障碍的证据。十年前，美国食品和药物管理局批准羟基脲用于临床严重 SCD 的成年患者；然而，其在儿童中的使用仍属超适应证用药。尽管有大量关于其疗效和安全性的证据，但羟基脲目前仅在 SCD 患者中有限地使用，因此对这种疾病的疗效有限；需要确定并克服其使用的障碍。
