MULTICENTER STUDY OF HYDROXYUREA 5 YEARS FOLLOW-UP STUDY

羟基尿素治疗镰刀细胞性贫血的疗效观察镰状细胞病是一种由遗传、体液、血管和环境因素相互作用而导致的多器官系统紊乱的复杂综合征。这种疾病的病程可以是突然和渐进的发作和缓解，从而损害功能，永久损害器官，并最终导致死亡。这项研究的目的将是确定羟基尿素治疗镰状细胞性贫血是否有任何长期不良影响。对贫血患者群体的长期随访的具体目标将是：确定新出现的严重疾病和特定器官或系统衰竭的渐进性损害；评估所有原因的死亡率并对死亡原因进行分类；对自纳入研究以来患者子女中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、所有原因和原因的特定死亡率、出生缺陷的频率和生活质量与相关确定的患者群体中观察到的情况进行比较，如镰刀细胞病的临床研究。如果患者有参加羟基脲研究的历史，他们就有资格参加这项研究。将有大约267名患者入选。他们将完成对一家诊所的五次年度访问。他们将根据自己医生的指示服用药物，并将知道药物是什么。他们将帮助保存他们的药物记录。他们将在四年内每年访问研究中心一次，去看研究医生。在每次就诊时，都会抽取血液样本，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，部分样本将被冷冻以供进一步使用。采集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.