DIPYRIDAMOLE/MAGNESIUM TO IMPROVE SICKLE CELL HYDRATION

这个子项目是许多研究子项目中的一个 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来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 中心，但不一定是研究者所在的机构。 这是一项多中心试验，招募了患有镰状细胞性贫血（SCA）的成人和儿科患者。本研究的总体目标是确定口服双嘧达莫、口服镁或两种药物联合治疗SCA患者的获益和风险。虽然它们通过阻断不同的途径发挥作用，但双嘧达莫和镁似乎都能阻断或减少致密红细胞（RBC）的形成，而致密红细胞（RBC）是SCA患者血管闭塞的病理生理学驱动因素。我们提出的研究将集中在以下目标和假设：评估对红细胞水合作用的影响，评估对红细胞存活的影响，确定副作用和评估临床结果，如红细胞水合作用与患者临床病程之间的相关性。本研究由两部分组成：第I部分是一项II期随机、双盲治疗研究，主要终点是测量红细胞水合作用，第II部分是一项随机、组间临床试验，主要终点是红细胞存活率（生物素标记技术）。样本量将包括第I部分入组的48例患者和第II部分入组的18例患者。第II部分的患者不必参加第I部分。

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. This is a multi-center trial recruiting both adults and pediatrics with sickle cell anemia (SCA). The overall goal of this study is to determine the benefits and risks of treating patients with SCA with oral dipyridamole, oral magnesium or a combination of both drugs. While they operate by blocking different pathways, both dipyridamole and magnesium appear to block or reduce the formation of dense erythrocytes (RBC) that drive the pathophysiology of vaso-occlusion in patients with SCA. Our proposed study will focus on the following aims and hypotheses: assessing effects on red cell hydration, assessing effects on red cell survival, determining side effects and evaluating clinical outcomes such as correlation between red cell hydration effects and the clinical course of the patients. This study consists of two parts: Part I, is a phase II randomized, double blind treatment study which primary endpoint is to measure red cell hydration and Part II is a randomized, clinical trial between group comparisons with the primary endpoint being red cell survival (biotin label technique). The sample size will consist of forty eight patients enrolled in Part I and eighteen patients will be recruited for Part II. The patients in Part II do not have to have participated in Part I.