PEDIATRIC HYDROXYUREA PHASE III CLINICAL TRIAL: BABY HUG

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 羟基脲(HU)已证实对成人镰状细胞性贫血(SCA)的实验室和临床疗效。儿童SCA患者也从HU治疗中受益。虽然HU疗法已经成为治疗SCA患者的一种令人兴奋和有效的药物，但关于它的使用仍然存在重要的问题，特别是对那些不到2岁的儿童。这项研究的主要目的是确定每天口服羟基脲是否可以减少50%的慢性器官损害，这种损害发生在患有SCA的幼儿身上。本研究的次要目标包括：1)确定胎儿血红蛋白水平与SCA儿童慢性器官损害的关系；2)研究HU对SCA儿童的安全性，包括a)身体生长发育，b)神经心理发育，c)免疫反应，d)DNA突变效应。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Hydroxyurea (HU) has demonstrated laboratory and clinical efficacy for adults with sickle cell anemia (SCA). Pediatric patients with SCA also benefit from HU therapy. Although HU therapy has emerged as an exciting and efficacious therapeutic agent for patients with SCA, important issues remain regarding its use, especially for those less than 2 years old. The primary objective of this study is to determine whether daily oral hydroxyurea can reduce by¿50% chronic organ damage that develops in young children with SCA. Secondary objectives of this study include: 1) to determine the relationship between fetal hemoglobin levels and chronic organ damage in young children with SCA and 2) investigate the safety of HU for young children with SCA regarding a) physical growth and development, b) neuropsychological development, c) immunological responses, and d) mutagenic effects on DNA.