HEREDITARY ANGIONEUROTIC EDEMA (HAE)

遗传性血管神经性水肿 (HAE)

• 批准号: 7952134
• 负责人: J. ANDREW GRANT
• 金额: $ 1.79万
• 依托单位: UNIVERSITY OF TEXAS MED BR GALVESTON
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-04-01 至 2009-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7952134
• 关键词: Abdomen; Adverse event; Angioneurotic Edema; Blood; Clinical Research; Coagulation Process; Complement 1 Inactivators; Complement Inactivators; Computer Retrieval of Information on Scientific Projects Database; Europe; Face; Funding; Grant; Human; Infusion procedures; Inherited; Institution; Limb structure; Literature; Patients; Prophylactic treatment; Pruritus; Research; Research Personnel; Resources; Safety; Schedule; Secondary to; Serine Proteinase Inhibitors; Serpins; Severities; Source; Swelling; System; Time; Toxic effect; Trauma; United States National Institutes of Health; Viscera; alternative treatment; autosomal dominant trait; base; hereditary angioneurotic edema; open label; prevent; prophylactic; protein function; response

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. C1 Esterase Inhibitor (C1INH) is a normal constituent of human blood and is one of the serine protease inhibitors (serpins). The protein functions as an inhibitor of the complement and contact systems (intrinsic clotting). Hereditary angioedema (HAE) is manifested by attacks of non-itching swelling of the extremities, face, trunk, airway, or abdominal viscera, occurring spontaneously or secondary to trauma. It is inherited as an autosomal dominant trait and is due to deficient activity of C1INH . Treatment of HAE patients with C1INH has been documented in the scientific literature since the concentrate became available in Europe in the early 1970's. There was no evidence of toxicity. This is a multi-center open-label study that will evaluate the efficacy and safety of C1INH-nf as prophylaxis to prevent HAE attacks. Subjects will be given prophylactic infusions of C1INH-nf every 3-7 days (approximately 1-2 times a week). The infusion schedule will be determined by the investigator based on the subject's response to the therapy. The efficacy will be analyzed by the number of all angioedema attacks that occur during the treatment irrespective of whether the subject obtained open label C1INH-nf or not and will be summarized. For analysis of safety, the number and severity of adverse experiences will be summarized. This study may help develop alternative treatment option for HAE patients.

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 研究中心，而研究中心不一定是研究者所在的机构。 C1酯酶抑制剂（C1 Esterase Inhibitor，C1 INH）是一种丝氨酸蛋白酶抑制剂（Serpin），是人体血液中的一种正常成分。 该蛋白质作为补体和接触系统（内源性凝血）的抑制剂发挥作用。 遗传性血管性水肿（HAE）表现为自发或继发于创伤的四肢、面部、躯干、气道或腹部内脏的非瘙痒性肿胀的发作。 它是作为常染色体显性性状遗传的，是由于C1 INH活性不足。自从20世纪70年代早期浓缩物在欧洲上市以来，用C1 INH治疗HAE患者已被记录在科学文献中。 没有毒性证据。这是一项多中心开放标签研究，将评估C1 INH-nf作为预防HAE发作的预防措施的疗效和安全性。受试者将每3-7天接受一次C1 INH-nf预防性输注（约每周1-2次）。研究者将根据受试者对治疗的反应确定输注时间表。将通过治疗期间发生的所有血管性水肿发作次数分析疗效，无论受试者是否获得开放标签C1 INH-nf，并将进行总结。对于安全性分析，将总结不良事件的数量和严重程度。 这项研究可能有助于为HAE患者开发替代治疗方案。