Northern California Consortium for Sickle Cell Disease

北加州镰刀细胞疾病联合会是一个由地区性诊所和 北加州和内华达州的综合医疗中心致力于提供 全面关爱镰状细胞病。此外，该联盟中的捐助中心拥有所有 必要要素，包括医生、护士、临床支持人员、数据协调员和临床 成功参与镰状细胞病临床试验所需的研究基础设施。在以下方面的努力 通过与社区咨询委员会建立伙伴关系，将加强患者招募工作 北加州基于社区的患者倡导组织，通过与患者和 他们的家人对拟议中的临床试验进行了了解。在本申请中建议进行两项临床调查，并 涉及奥克兰该中心在临床研究方面有很强记录的领域。第一个是一个 造血细胞移植治疗镰状细胞病后的长期随访评估 与国际血液和骨髓移植研究中心合作进行 (CIBMTR)。这项研究将对参加合作社的儿童的结果进行比较 研究镰刀细胞病(CSSCD)和多中心儿科羟基尿素试验(HUG-KILDS)，由 Rho生物统计团队的调查人员。第二项研究是第三阶段随机试验。 红细胞输注预防镰状细胞病急性胸部综合征的前瞻性试验。这 调查将针对表现出急性白血病高危特征的镰状细胞病患者进行登记 胸部综合征，表现为分泌型磷脂酶A2(SPLA2)水平升高，这种酶 分解产生脂肪酸和溶血磷脂的磷脂。最近，我们展示了sPLA2 在许多急性冠脉综合征患者中显著升高，sPLA2水平升高在急性冠脉综合征发展之前就预示着它的存在。这个 拟议的临床试验结果很可能对临床管理产生深远的影响 镰状细胞病患者，两者都需要多中心合作临床试验基础设施 以及时和严格的方式完成这些临床调查。

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 clincal 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.