USE OF HYDROXYUREA IN PATIENTS WITH SICKLE CELL ANEMIA

这是一个建议，以合作研究的影响， 羟基脲对血红蛋白F产生的影响及临床状况 镰状细胞病 临床表现严重的患者 在知情同意后，将给予少量药物 用于药代动力学研究。 然后他们将被给予15 mg/Kg/天，剂量递增至最低血液学检查 观察到毒性。 剂量会降低， 维持20-24个月，以确定药物的效果 对血红蛋白F的产生，对血液学参数， 以及患者的临床状态。 观测 毒性和致癌性。 数据将 分析以确定：1.药物剂量的差异 镰状细胞病患者的血液学参数。 2. 镰状细胞表型表达差异的影响 细胞疾病对羟基脲的反应3.相关性 通过血液学变量测量的对羟基脲的反应 以及通过临床状态的变化测量的临床反应。

This is a proposal to study collaboratively the effects of hydroxyurea on hemoglobin F production and the clinical state in sickle cell disease. Patients with severe clinical manifestations will, after informed consent, be given small amounts of the drug for pharmacokinetic studies. They will then be given a dose of 15 mg/Kg/day with escalation of the dose until minimal hematologic toxicity is observed. The dose will be lowered and will be maintained for 20-24 months to determine the effect of the drug on the production of hemoglobin F, on hematologic parameters, and on the clinical status of the patient. Observations for toxicity and carcinogenesis will be made. The data will be analyzed to determine: 1. differences in dose of the drug on the hematological parameters in patients with sickle cell disease. 2. the effect of differences in the phenotypic expression of sickle cell disease on the response to hydroxyurea 3. Correlation of the response to hydroxyurea as measured by hematologic variables and the clinical response as measured by changes in clinical state.