STUDY OF THALASSEMIA AND TREATMENT

地中海贫血及其治疗的研究

• 批准号: 6605000
• 负责人: NANCY F OLIVIERI
• 金额: $ 36.06万
• 依托单位: UHN TORONTO GENERAL HOSPITAL
• 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/6605000
• 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.

（根据申请人的摘要改编）该研究旨在 比较当铁螯合剂脱氧胺的有效性 使用两种不同的途径进行管理：皮下注射注射和8- 小时皮下输注（标准疗法），患有丘脑的患者 主要的。 迄今为止的一些研究测量了尿铁排泄 通过这两种deferoxamine的方案实现，它们的相对有效性 尚未被比较。 尽管有效，脱氧胺的使用是 由于需要长时间的肠胃外输注而复杂 结果，大量费用和差的患者依从性。 因为大多数 患有丘脑贫血影响的患者生活在新兴国家 提供与输液泵有关的费用，这种挽救生命的疗法 可用于世界上thalalsemics的一小部分。 主要 这项研究的假设是皮下注射 脱氧胺，每天两次在12个月内服用两次，将减少 或以等效于浓度的浓度保持人体存储铁 通过每晚8小时皮下注入治疗的患者队列 在同一时期的脱脂氧胺。 拟议研究的具体目的 要比较：每天两次注射的短期功效，以及 由尿铁排泄和 减少血清铁及其代谢产物的有毒部分；亲戚 这些方案的有效性，由肝铁的减少确定 专注;并遵守这些方案。 主要 该试验的终点是肝铁浓度，最定量的， 确定身体铁负担的特定和敏感的方法 在基线和12个月后，在所有患者中确定。 如果证明每天两次注射的有效性是 等于脱铁胺的常规输注，这种新方案将 向所有thalassybia，全球，具有成本效益的患者提供报价 长期输注脱氧胺的方便替代方案，目前是 唯一的挽救生命药物的方法。