...
  • 主题
高级搜索  >
历史搜索 清空历史
首页> 外文期刊>Journal of neurology >Multiple sclerosis trial designs for the 21st century: building on recent lessons.
【24h】

Multiple sclerosis trial designs for the 21st century: building on recent lessons.

机译：21世纪的多发性硬化试验设计：以最近的经验为基础。

获取原文
获取原文并翻译 | 示例
           
页面导航
  • 摘要
  • 著录项
  • 相似文献
  • 相关主题

摘要

Starting with the first positive pilot study of glatiramer acetate, trial design in multiple sclerosis has advanced considerably over the past two decades, successively building and improving on previous successes in the implementation and analysis of new clinical trials. Most of these trials have been successful and this has led to the regulatory approval and commercial availability of six agents for the treatment of multiple sclerosis. During this period, outcome measures have been validated to determine the efficacy and safety of such agents, notably those useful in reducing the inflammatory aspects of disease. These include measurements of relapse reduction (annualized relapse rate, time to first relapse, proportion of subjects relapse free), disability (change in EDSS score, change in MSFC score) and MRI metrics (measurements of gadolinium-enhancing lesions, T1 and T2 lesion load). Recent trial design has shown that one can answer some clinical questions after one year on study and that these results may be predictive of more robust two-year trial data. The other important recent lesson involves emergence of rare complications of immunomodulatory therapy, namely progressive multifocal leucoencephalopathy with natalizumab that blocks the access of immune cells to the nervous system. In addition to the increased need for enhanced safety assessment, this issue will have an impact both on the study of combination therapies and on the use of combinations in clinical practice.
机译：从醋酸格拉替雷的第一个积极的先期研究开始，在过去的二十年中，多发性硬化症的试验设计有了长足的发展，并在新的临床试验的实施和分析方面取得了成功，并在此基础上不断发展和完善。这些试验中的大多数都已成功，这已获得监管部门的批准和六种用于治疗多发性硬化症的药物的商业供应。在此期间，已经验证了确定这些药物的疗效和安全性的结果措施，尤其是可用于减少疾病炎症方面的药物。其中包括复发减少量（年复发率，首次复发时间，无复发受试者的比例），残疾（EDSS评分变化，MSFC评分变化）和MRI指标（g增强病变，T1和T2病变的测量）加载）。最近的试验设计表明，研究一年后，人们可以回答一些临床问题，这些结果可能预示了更可靠的两年试验数据。最近的另一个重要教训是出现了免疫调节疗法的罕见并发症，即进行性多灶性白质脑病合并那他珠单抗的治疗会阻止免疫细胞进入神经系统。除了对增强安全性评估的需求日益增加之外，该问题还将对联合疗法的研究以及临床实践中联合疗法的使用产生影响。

著录项

相似文献

  • 外文文献
  • 中文文献
  • 专利
获取原文

客服邮箱：kefu@zhangqiaokeyan.com

京公网安备：11010802029741号 ICP备案号：京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有

  • 客服微信

  • 服务号