USE OF HYDROXYUREA IN PATIENTS WITH SICKLE CELL ANEMIA

这是一项合作研究的建议， 羟基脲对HbF产生的影响及临床状况 镰状细胞病。有严重临床表现的患者 在知情同意后，是否会给予少量药物 用于药代动力学研究。然后，他们将被给予15剂 Mg/kg/天，随着剂量的增加，直到最低血液学 观察到了毒性。剂量将会降低，并将 维持20-24个月以确定药物的效果 关于血红蛋白F的产生，关于血液学参数， 以及病人的临床状况。观察到的 会产生毒性和致癌作用。数据将是 分析以确定：1.不同剂量的药物对 镰状细胞病患者的血液学指标。2. 镰刀表型表达差异的影响 细胞疾病对羟基脲的反应3。 用血液学指标测定羟基尿素的反应 以及通过临床状态的变化来衡量临床反应。

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.