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。 小儿羟基脲III期临床试验;一项在极年幼儿童中进行的羟基脲治疗的随机、双盲、安慰剂对照试验。在研究之前，将进行为期两年的内部试点研究。 入组内部初步研究和主研究的所有儿童将接受为期104周的研究治疗。将对内部初步研究中的所有儿童进行随访，直至最后一名儿童的研究治疗结束，并观察停止研究治疗的不良反应。研究治疗结束后，将在研究治疗结束后3、6、9、12、16、20、24和30个月以及此后每6个月一次，持续5年，要求重新签署同意书以继续进行随访门诊访视。 目的： 内部初步研究的目的： 1.确定BABY HUG在招募、随访、研究治疗依从性和研究程序时间表依从性方面的可行性。 2.评估尿素氢的毒性、对生长发育的影响以及重度/非预期不良事件的发生率。 3.根据肝脾扫描的双重独立读数，确定脾脏功能的分布以及观察者间和观察者内变异性。 4.评价通过血清肌酐和身高（Schwartz公式）估计的肾小球滤过率（GFR）与“金标准”（如DTPA清除率）相比的有效性和变异性。 5.评估在婴儿HUG年龄组中，羟基脲的某些药代动力学参数可降低。 主要研究目的： 1.确定每日口服羟基脲是否可以减少镰状细胞贫血幼儿发生的慢性器官损伤≥ 50%。 2.探讨镰状细胞贫血患儿胎儿血红蛋白（HbF）水平与慢性器官损害的关系。 3.探讨羟基脲治疗婴幼儿镰状细胞性贫血的安全性， a.身体的生长和发育， B.神经心理学发展， C.免疫反应，和 D.对DNA的诱变作用。

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. Pediatric Hydroxyurea Phase III Clinical Trial; a Randomized Double-blind Placebo Controlled Trial of Hydroxyurea Therapy in Very Young Children. The study will be preceded by a two-year Internal Pilot Study. All the children enrolled in the Internal Pilot Study and the main study will receive study treatment for the duration of 104 weeks. All children in the Internal Pilot Study will be followed until the last child's study treatment has ended with observation for untoward effects of discontinuing study treatment. After study treatment ends, renewed consent will be requested for continued follow-up clinic visits at 3, 6, 9, 12, 16, 20, 24, and 30 months after the end of study treatment, and every six months thereafter for up to five years. OBJECTIVES: Objectives of the Internal Pilot Study: 1. To determine the feasibility of BABY HUG in terms of recruitment, follow-up, adherence to study treatment, and compliance with the study schedule of procedures. 2. To assess Hydroyurea toxicity, effect on growth and development, and occurrence of severe/unexpected adverse events. 3. To establish the distribution and inter-observer and intra-observer variability of spleen function based on dual, independent readings of liver-spleen scans. 4. To evaluate the validity and variability of glomerular filtration rate (GFR) as estimated by serum creatinine and height (the Schwartz formula) compared with a "gold standard" such as DTPA clearance. 5. to assess some pharmacokinetics parametes of Hydroxyurea can reduce in the BABY HUG age group. Objective of the Main Study: 1. To determine whether daily oral Hydroxyurea can reduce by /=50% chronic organ damage that develops in young children with sickle cell anemia. 2. To determine the relationship between fetal hemoglobin (HbF) levels and chronic organ damage in young children with sickle cell anemia. 3. To investigate the safety of Hydroxyurea for young children with sickle cell anemia regarding a. physcial growth and development, b. neuropsychological development, c. immunological responses, and d. mutagenic effects on DNA.