Northern California Consortium for Sickle Cell Disease

描述（由申请人提供）： 北方加州镰状细胞病联盟是一个有凝聚力的区域诊所网络， 位于北方加州和内华达州的综合医疗中心致力于为镰状细胞病提供全面的护理。此外，该联盟的贡献中心拥有成功参与镰状细胞病临床试验所需的所有必要要素，包括医生、护士、临床支持人员、数据协调员和临床研究基础设施。通过与北方加州的一个以社区为基础的患者倡导组织社区咨询委员会建立伙伴关系，将加强患者招募工作，与患者及其家属就拟议的临床试验进行沟通。本申请中提出了两项临床研究，涉及奥克兰中心具有良好临床研究记录的领域。首先是镰状细胞病造血细胞移植后的长期随访评估，将与国际血液和骨髓移植研究中心（CIBMTR）合作进行。这项研究将包括对参加镰状细胞病合作研究（CSSCD）和多中心儿科羟基脲试验（HUG-KIDS）的儿童的结局进行比较，该试验由Rho生物统计学团队的研究人员领导。第二项研究是一项关于红细胞输注预防镰状细胞病急性胸部综合征的III期随机前瞻性试验。本研究将针对表现出急性胸部综合征高风险特征的镰状细胞病患者进行招募，如分泌型磷脂酶A2（sPLA2）水平升高所证明，sPLA2是一种切割磷脂产生脂肪酸和溶血磷脂的酶。最近，我们证明了sPLA2在许多ACS病例中显著升高，并且sPLA2水平升高可在ACS发展之前预测ACS。拟议临床试验的结果很可能对镰状细胞病患者的临床管理产生深远影响，并且两者都需要多中心合作临床试验基础设施，以便及时和严格地完成这些临床研究。(End摘要）

DESCRIPTION (provided by applicant): The Northern California Consortium for Sickle Cell Disease is a cohesive network of regional clinics and comprehensive medical centers in Northern California and Nevada that are committed to providing comprehensive care for sickle cell disease. In addition, contributing centers in this Consortium have all the necessary elements that include physicians, nurses, clinical support staff, data coordinators, and clinical research infrastructure necessary to participate successfully in clinical trials for sickle cell disease. Efforts at patient recruitment will be enhanced by a partnership with the Community Advisory Committee, a community-based patient advocate organization in Northern California, by communicating with patients and their families about proposed clinical trials. Two clinical investigations are proposed in this application and involve areas in which the center in Oakland has a strong track record of clinical research. The first is a long-term follow-up evaluation after hematopoietic cell transplantation for sickle cell disease, which will be conducted in collaboration with the Center for International Blood and Marrow Transplantation Research (CIBMTR). This study will include a comparison of outcomes among children enrolled in the Cooperative Study of Sickle Cell Disease (CSSCD) and the multicenter pediatric hydroxyurea trial (HUG-KIDS), led by investigators in the Biostatistics team at Rho. The second investigation is a phase III randomized prospective trial of RBC transfusions to prevent acute chest syndrome in sickle cell disease. This investigation will target enrollment of sickle cell disease patients who exhibit high-risk features for acute chest syndrome, as evidenced by elevated levels of secretory phospholipase A2 (sPLA2), an enzyme which cleaves phospholipids generating fatty acids and lysophospholipids. Recently, we demonstrated that sPLA2 is dramatically elevated in many cases of ACS and rising sPLA2 levels predict ACS before it develops. The results of the proposed clinical trials are very likely to have a profound effect on the clinical management of patients with sickle cell disease, and both require a multicenter cooperative clinical trial infrastructure in order to complete these clinical investigations in a timely and rigorous manner. (End of Abstract)