Sedaghat-Hamedani F, Katus H A, Meder B
Institute for Cardiomyopathies Heidelberg, Department of Medicine III, University of Heidelberg, Im Neuenheimer Feld 410, 69120, Heidelberg, Germany.
DZHK (German Centre for Cardiovascular Research), Berlin, Germany.
Herz. 2018 Mar;43(2):123-130. doi: 10.1007/s00059-017-4667-x.
Evidence-based medicine has considerably advanced the treatment of highly prevalent cardiovascular diseases. Its implementation was driven by multicenter interventional trials in treatment and placebo cohorts, propelling numerous biomedical innovations toward standard of care. While a uniform treatment can be effective in such disease cohorts ("one size fits all"), it neglects the genetic and phenotypic individuality of a single patient and his or her disease. Accordingly, a recent observation was made that several newer "mega" trials, demanding considerable resources for their execution, showed statistically significant differences in outcome, however, with small overall efficacies that render implementation in the clinics unlikely. To overcome this concerning development, new methods for individualized treatment of cardiovascular disease are required. Rarer conditions, such as distinct cardiomyopathies, may deliver the blueprint for a paradigm shift: deep and precise phenotyping of individual patients by a multimodal approach and development of targeted treatments for smaller groups ("one treatment for many") or even for single patients ("one treatment of some").
循证医学极大地推动了高发性心血管疾病的治疗。其实施是由治疗组和安慰剂组的多中心干预试验推动的，促使众多生物医学创新成为护理标准。虽然统一治疗在这类疾病群体中可能有效（“一刀切”），但它忽略了单个患者及其疾病的遗传和表型个体性。因此，最近有人观察到，几项执行需要大量资源的较新的“大型”试验在结果上显示出统计学上的显著差异，然而，总体疗效较小，使得在临床中实施不太可能。为了克服这一令人担忧的发展趋势，需要用于心血管疾病个体化治疗的新方法。罕见病症，如特定的心肌病，可能为范式转变提供蓝图：通过多模式方法对个体患者进行深入精确的表型分析，并为较小群体（“一人一方”）甚至单个患者（“一人一剂”）开发靶向治疗方法。
