SUPPLEMENTAL INTRAVENOUS IRON THERAPY FOR RLS

不宁腿综合征的补充静脉铁剂治疗

• 批准号: 7260478
• 负责人: CHRISTOPHER J EARLEY
• 金额: $ 5.46万
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/7260478
• 关键词: abnormal involuntary movement; blood chemistry; brain metabolism; clinical trials; dosage; electromyography; human subject; human therapy evaluation; interview; intravenous administration; iron; iron metabolism; iron storage disorder; leg; limb movement; longitudinal human study; magnetic resonance imaging; neuromuscular disorder chemotherapy; outcomes research; oxidative stress; patient oriented research; polysomnography; questionnaires; substantia nigra; therapy adverse effect; urinalysis

Our core concept is that RLS is related to an underlying problem in iron utilization. This may be specific to the brain or a region of the brain, or may be a general systemic problem with different organs having different abilities to compensate. We do not believe that there is one defect for all; rather, we believe that several problems in iron metabolism will eventually be found to contribute to this syndrome. The long-term goal of this proposal is to establish the importance of brain iron metabolism in RLS by relating changes in the CNS iron status to changes in the clinical aspects of RLS patients; and to provide improved treatment for RLS. In this grant we focus on the use of intravenous iron as a potential long-term treatment option for this syndrome. We also address the question of whether there is increased iron loss and whether there are likely to be problems of iron overload or excessive oxidative stress with iron treatment. The specific aims of the current proposal are: Aim 1. To evaluate the ability to sustain for 2 years the initial therapeutic benefit of 1,000 mg of IV iron with iron versus placebo "supplemental" therapy. Aim 2. To evaluate putative mechanisms behind the rapid decrease in ferritin following intravenous (IV) iron treatment of RLS patients. Aim 3. To determine possible adverse effects from the IV iron treatment. Aim 4. To explore the effects of supplemental IV iron on patients who are non-responders to the initial 1000-mg IV iron dose.

我们的核心概念是，RLS与铁利用中的一个根本问题有关。这可能是特定于大脑或大脑的一个区域，或者可能是一个一般的系统性问题，不同的器官具有不同的补偿能力。我们并不认为所有的缺陷都是一个;相反，我们认为铁代谢中的几个问题最终会被发现导致这种综合征。该提案的长期目标是通过将CNS铁状态的变化与RLS患者临床方面的变化相关联来确定脑铁代谢在RLS中的重要性;并为RLS提供改进的治疗。在这项资助中，我们专注于静脉铁剂的使用，作为这种综合征的潜在长期治疗选择。我们还解决了是否有增加铁的损失，是否有可能与铁治疗铁过载或过度氧化应激的问题。 本提案的具体目标是： 目标1.评价与安慰剂“补充”治疗相比，1，000 mg IV铁剂+铁剂的初始治疗获益持续2年的能力。 目标2.评价RLS患者静脉（IV）铁剂治疗后铁蛋白快速降低的假定机制。 目标3。确定IV铁剂治疗可能产生的不良反应。 目标4。探索补充IV铁剂对初始1000 mg IV铁剂无应答患者的影响。