MULTICENTER STUDY OF HYDROXYUREA 5 YEARS FOLLOW-UP STUDY

一项确定羟基脲治疗镰状细胞贫血有效性的试验。镰状细胞病是一种复杂的综合征，通过遗传、体液、血管和环境因素的相互作用引起多器官系统紊乱。疾病的过程可以是突然和逐渐的发作和缓解，从而损害功能，永久损害器官，并最终导致死亡。本研究的目的是确定用羟基脲治疗镰状细胞性贫血是否有任何长期不良反应。 贫血患者人群长期随访的具体目的将是：确定新出现的严重医学状况和特定器官或系统衰竭的进行性损害;评估全因死亡率并对死亡原因进行分类;对入组研究以来患者后代发生的出生缺陷进行分类;评估患者的生活质量;并将特定器官或症状的功能、所有原因和原因特异性死亡率、出生缺陷的频率和生活质量与相关定义的患者人群（如镰状细胞病临床研究）中观察到的结果进行比较。如果患者有入组羟基脲研究的病史，则有资格进入本研究。 将入组约267例患者。他们将完成五次年度门诊。他们将根据自己的医生的指导服用药物，并将知道药物是什么。他们将帮助记录他们的药物。他们将每年访问研究中心一次，为期四年，以看到研究医生。每次访视时，将抽取血液样本，以了解受试者的一般健康状况并研究血细胞。部分样本将在当天使用，部分将冷冻备用。抽取的一部分血液将用于寻找细胞中基因的变化。

A trial to determine the effectiveness of hydroxyurea in Sickle Cell Anemia. Sickle cell disease is a complex syndrome with multiple organ system disturbances brought about through the interplay of genetic, humoral, vascular, and environmental factors. The course of the disease can be one of abrupt and gradual attacks and remissions, thus impairing function, permanently damaging organs, and ultimately causing death. The objectives of the study will be to determine whether there are any long-term adverse effects of treatment of sickle cell anemia with hydroxyurea. The specific objectives in the long-term follow-up of the anemia patient population will be: to identify newly arising, serious medical conditions and progressive impairment of failure of specific organs or systems; to assess all cause mortality and classify causes of death; to classify birth defects occurring in the offspring of patients since enrollment in the study; to assess quality of life for the patients; and to compare the function of specific organs or symptoms, all cause and cause specific mortality, frequency of birth defects and quality of life to those observed in relevant defined patient populations such as the Clinical Study of Sickle Cell Disease. Patients are eligible to enter this study if they have had a history of enrollment in a hydroxyurea study. Approximately 267 patients will be enrolled. They will have completed five annual visits to a clinic. They will be taking medication according to the direction of their own physicians and will know what the medications are. They will help keep records of their medications. They will visit the research center once a year for four years to see a research doctor. At each visit, a blood sample will be drawn to find out about the general health of the subject and to study the blood cells. Part of the sample will be used on that day and part will be frozen for further use. A part of the blood taken will be used to look for changes in the genes in the cells.