THERAPEUTIC TRIALS IN IDIOPATHIC INFLAMMATORY MYOPATHIES

为了找到更好的治疗肌炎的方法，该组织 探索以新的方式使用既有的药物， 联合用药或新疗法。这些审判实际上是唯一的 治疗这种非常罕见的疾病的试验，因此也 吸引患者到临床中心进行其他研究 小组开展的关于肌炎基本机制的项目， 相关或类似疾病（在ZO 1 AR 41074-08和ZO 1 AR中概述） 41099-03）。 大剂量甲氨蝶呤静脉注射与 甲酰四氢叶酸救援和口服甲氨蝶呤和 硫唑嘌呤已经完成了在过去的一年中， 研究结果正在准备发表。 我们已经开始了一项嘌呤类似物氟达拉滨的试验， 肌炎一种评估肌肉炎症的定量方法， 肌炎已经被开发出来，并将在当前和未来使用 治疗试验计划的16名患者中有13名已经进入 到目前为止，其中近一半已经完成了六个月的审判， 治疗阶段。 一项抗甲状腺药物甲巯咪唑的试验将于明年开始。 年选择这种药剂是因为它可以改变肌肉的能力， 细胞作为免疫攻击的目标。在过去的一年里， 评估药物对肌肉和血细胞作用的方法 与NIDDK和NCI合作开发。

In an attempt to find better ways to treat myositis, the group has explored the use of both established medications used in new ways or combinations or new therapies. These trials are virtually the only such trials of the treatment of this very uncommon condition and thus also serve to draw patients to the Clinical Center for the other research projects carried out by the group on the basic mechanisms of myositis and related or mimicking diseases (outlined in ZO1 AR 41074-08 and ZO1 AR 41099-03). A randomized, crossover trial of high dose intravenous methotrexate with leucovorin rescue and of a combination of oral methotrexate and azathioprine has been completed in the past year and an analysis of the results is being prepared for publication. We have begun a trial of the purine analogue, fludarabine, in the therapy of myositis. A quantitative method of assessing muscle inflammation in myositis has been developed and will be used in current and future therapeutic trials. Thirteen of the planned sixteen patients have entered the trial so far, of whom almost half have completed the six-month treatment phase. A trial of the anti-thyroid drug, methimazole, will be begun in the next year. This agent was chosen because it may alter the ability of muscle cells to serve as a target for immunologic attack. In this past year, the methods for assessing the effect of the drug on muscle and blood cells have been developed in collaboration with NIDDK & NCI.