CLINICAL TRIAL: PEDIATRIC HYDROXYUREA PHASE III CLINICAL TRIAL: BABY HUG

该子项目是利用 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得主要资金， 因此可以在其他CRISP条目中表示。列出的机构是 中心，不一定是研究者的机构。 羟基脲（HU）已证明对镰状细胞贫血（SCA）成人的实验室和临床疗效。患有SCA的儿科患者也受益于HU治疗。尽管HU疗法已成为SCA患者令人兴奋且有效的治疗药物，但关于其使用仍存在重要问题，特别是对于那些年龄小于2岁的患者。本研究的主要目的是确定每日口服羟基脲是否可以减少SCA幼儿慢性器官损伤的50%。本研究的次要目的包括：1）确定SCA幼儿中胎儿血红蛋白水平与慢性器官损伤之间的关系; 2）研究HU在以下方面对SCA幼儿的安全性：a）身体生长和发育; B）神经心理发育; c）免疫反应; d）对DNA的致突变作用。

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.