Alderazi Yazan J, Bomprezzi Roberto
Department of Neurology (YJA), Division of Neurocritical Care, Miller School of Medicine, University of Miami, FL; and Division of Neurology (RB), Maine General Medical Center, Augusta.
Neurol Clin Pract. 2014 Aug;4(4):319-328. doi: 10.1212/CPJ.0000000000000046.
The practice of medicine relies on the patient-physician relationship, knowledge, and clinical judgment. Randomized controlled trials (RCTs) remain the least biased method for studying the effects of interventions in selected populations and are the only method to control adequately for unknown confounders. However, physicians face the limitations of RCTs on a daily basis as they treat relatively unselected populations and individual patients. We explore the benefits and limitations of RCTs for some neurologic disorders, and discuss the difficulties of predicting individualized outcomes and anticipating treatment responses in those heterogeneous conditions. Observational studies and advances in understanding neurologic diseases complement RCTs in decision-making. Considerable challenges remain for personalized medicine; for now, clinicians must rely on their ability to integrate evidence and clinical judgment.
医学实践依赖于医患关系、知识和临床判断。随机对照试验（RCTs）仍然是研究特定人群中干预措施效果偏差最小的方法，也是唯一能充分控制未知混杂因素的方法。然而，医生在日常治疗相对未经筛选的人群和个体患者时，会面临随机对照试验的局限性。我们探讨了随机对照试验对某些神经系统疾病的益处和局限性，并讨论了在这些异质性情况下预测个体化结果和预期治疗反应的困难。观察性研究以及对神经系统疾病认识的进展在决策过程中对随机对照试验起到补充作用。个性化医疗仍然面临相当大的挑战；目前而言，临床医生必须依靠他们整合证据和临床判断的能力。
