Parides Michael K, Moskowitz Alan J, Ascheim Deborah D, Rose Eric A, Gelijns Annetine C
International Center for Health Outcomes and Innovation Research, Columbia University, New York, New York 10032, USA.
Ann Thorac Surg. 2006 Sep;82(3):1140-6. doi: 10.1016/j.athoracsur.2006.05.123.
New left ventricular assist devices promise fewer adverse events but, currently, only minor improvements in survival. Small (survival) treatment effects, limited patient populations, and the increasing number of left ventricular assist devices in development challenge the efficient conduct of premarketing trials (especially in destination therapy) and, maybe more importantly, hamper innovation. Novel trial designs would facilitate this process. Among a range of trial designs, we opt for small randomized trials, which would preserve the advantages of randomization and also allow for a shorter enrollment period. We also advocate an evidence shift toward postmarketing studies, with the Interagency Registry of Mechanically Assisted Circulatory Support providing a robust infrastructure.
新型左心室辅助装置有望减少不良事件，但目前仅能在生存率上带来微小改善。（生存率方面的）治疗效果微小、患者群体有限以及越来越多处于研发阶段的左心室辅助装置，给上市前试验（尤其是在终末期治疗中）的高效开展带来了挑战，或许更重要的是，阻碍了创新。新颖的试验设计将有助于这一过程。在一系列试验设计中，我们选择小型随机试验，其既能保留随机化的优势，又能缩短入组时间。我们还主张将证据重心转向上市后研究，机械辅助循环支持跨部门注册机构可提供强大的基础设施。
