CRL 5861 (PURIFIED POLOXAMER 188) IN SICKLE CELL DISEASE & VASO OCCLUSIVE CRISIS

CRL 5861（纯化的泊洛沙姆 188）治疗镰状细胞病

• 批准号: 6263443
• 负责人: BRIAN K ADLER
• 金额: $ 2.95万
• 依托单位: UNIVERSITY OF ALABAMA AT BIRMINGHAM
• 依托单位国家: 美国
• 财政年份: 1998
• 资助国家: 美国
• 起止时间: 1998-12-01 至 1999-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6263443
• 关键词: blood disorder chemotherapy; blood vessel occlusion; clinical research; ethylene oxide; human subject; human therapy evaluation; pathologic process; sickle cell anemia

Sickle Cell Disease (SCD) is the most common human hereditary hematologic disorder. This disease varies in its clinical manifestation and severity. Persons with this debilitating disease are often plagued with acute pain crises requiring hospitalization as well as assaults on various organ systems leading to impairment and/or failure of those organs. This study focuses on the painful crisis. The average crisis lasts for approximately 7-10 days. In results from the natural history study (CSSCD-Cooperative Study of SCD), Platt et al documented that approximately one-third of SCD patients die during a sickle cell crisis, usually after complications associated with this event, i.e. stroke, seizure or acute chest syndrome. The purpose of this study is to assess the efficacy of FLOCOR compared to placebo control in reducing the duration of vaso-occlusive crisis in patients with SCD.

镰状细胞病（SCD）是人类最常见的遗传性血液病。 这种疾病的临床表现和严重程度各不相同。患有这种使人衰弱的疾病的人经常受到需要住院治疗的急性疼痛危机以及对各种器官系统的攻击的困扰，导致这些器官的损伤和/或衰竭。 本研究的重点是痛苦的危机。 平均危机持续约7-10天。 在自然史研究（CSSCD-SCD合作研究）的结果中，Platt et al记录了大约三分之一的SCD患者在镰状细胞危象期间死亡，通常是在与该事件相关的并发症（即中风、癫痫发作或急性胸部综合征）之后。本研究的目的是评估与安慰剂对照相比，FLOCOR在缩短SCD患者血管闭塞危象持续时间方面的疗效。