SUPPLEMENTAL INTRAVENOUS IRON THERAPY FOR RLS

我们的核心概念是，RLS与铁利用的一个潜在问题有关。这可能是特定于大脑或大脑某一区域的，也可能是一个普遍的系统性问题，不同的器官具有不同的补偿能力。我们不认为所有人都有一种缺陷；相反，我们认为铁代谢的几个问题最终会被发现导致这种综合征。这项建议的长期目标是通过将中枢神经系统铁状态的变化与RLS患者临床方面的变化联系起来，确定脑铁代谢在RLS中的重要性；并改进RLS的治疗。在这笔赠款中，我们将重点放在静脉用铁作为这种综合征潜在的长期治疗选择上。我们还讨论了铁损失是否增加，以及铁治疗是否可能出现铁超载或过度氧化应激的问题。 目前这项建议的具体目标是： 目的1.评估与安慰剂“补充”疗法相比，1,000毫克静脉注射铁剂与铁剂联合治疗的初期疗效是否能维持2年。 目的2.评估RLS患者静脉铁剂治疗后铁蛋白快速下降的可能机制。 目的3.确定静脉注射铁治疗可能产生的不良反应。 目的4.探讨补充静脉注射铁对初始剂量为1000 mg的静脉注射铁无效患者的疗效。

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.