STUDY OF THALASSEMIA AND TREATMENT

地中海贫血及其治疗的研究

• 批准号: 6760184
• 负责人: NANCY F OLIVIERI
• 金额: $ 32.14万
• 依托单位: UNIVERSITY HEALTH NETWORK
• 依托单位国家: 美国
• 财政年份: 2000
• 资助国家: 美国
• 起止时间: 2000-09-15 至 2005-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/6760184
• 关键词: blood disorder chemotherapy; human therapy evaluation; thalassemia

(Adapted from the applicant's abstract) This research study is designed to compare the effectiveness of the iron-chelating agent deferoxamine, when administered using two different routes: subcutaneous bolus injections, and 8- hour subcutaneous infusions (standard therapy), in patients with thalassemia major. While a few studies to date have measured urinary iron excretion achieved with these two regimens of deferoxamine, their relative effectiveness has not been compared. Despite its effectiveness, the use of deferoxamine is complicated by a requirement for prolonged parenteral infusion and, as a consequence, considerable expense and poor patient compliance. Because most patients affected with thalassemia live in emerging countries which cannot afford this expense associated with infusion pumps, this life-saving therapy is available to a small fraction of the world's thalassemics. The primary hypothesis of this study is that subcutaneous bolus injections of deferoxamine, administered twice daily over a period of 12 months, will reduce or maintain body storage iron at concentrations equivalent to those in a cohort of patients treated with nightly 8-hour subcutaneous infusions of deferoxamine over the same period. The specific aims of the proposed research are to compare: the short-term efficacy of twice-daily bolus injections, and 8-hour infusions of deferoxamine, as determined by urinary iron excretion and reduction in toxic fractions of serum iron and its metabolites; the relative effectiveness of these regimens, as determined by reduction of hepatic iron concentration; and patient compliance with these regimens. The primary endpoint of this trial is hepatic iron concentration, the most quantitative, specific and sensitive method for determining body iron burden, to be determined in each patient at baseline and after 12 months, in all patients. If the effectiveness of twice-daily bolus injections is demonstrated to be equal to that of conventional infusions of deferoxamine, this new regimen will offer to all patients with thalassemia, worldwide, a cost-effective and convenient alternative to prolonged infusions of deferoxamine, currently the only method of administration of this life-saving drug.

（改编自申请人摘要）本研究旨在 比较铁螯合剂去铁胺的有效性，当 使用两种不同的途径施用：皮下推注，和8- 小时皮下输注（标准治疗），地中海贫血患者 少校 虽然迄今为止的一些研究测量了尿铁排泄 这两种去铁胺方案的相对有效性 没有被比较。 尽管它的有效性，使用去铁胺， 由于需要长时间的胃肠外输注而复杂化， 结果，相当大的费用和较差的患者依从性。 因为大多数 地中海贫血患者生活在新兴国家， 支付与输液泵相关的费用， 只适用于世界上一小部分的地中海贫血症患者。 主 本研究的假设是皮下推注 去铁胺，每天两次，为期12个月，将减少 或维持体内储存铁的浓度相当于 每晚8小时皮下输注 去铁胺在同一时期。 拟议研究的具体目标 比较：每日两次推注的短期疗效，以及 8-小时输注去铁胺，通过尿铁排泄测定， 降低血清铁及其代谢产物的毒性部分;相对 这些方案的有效性，通过肝铁的减少来确定 浓度;以及患者对这些方案的依从性。 主 该试验的终点是肝铁浓度，最定量的， 一种特异、灵敏的测定人体铁负荷的方法， 在基线和12个月后，在所有患者中测定每例患者。 如果证明每日两次推注的有效性 与传统的去铁胺注射相同，这种新的治疗方案将 为全球所有地中海贫血患者提供一种具有成本效益和 方便的替代长期输注去铁胺，目前， 这是唯一一种能救命的药物