Department of Biostatistics and Computational Biology, Dana-Farber Cancer Institute, 450 Brookline Avenue, Boston, MA 02215, USA; Harvard Medical School, Boston, MA, USA.
Department of Biostatistics and Computational Biology, Dana-Farber Cancer Institute, 450 Brookline Avenue, Boston, MA 02215, USA; Harvard Medical School, Boston, MA, USA.
Breast. 2017 Aug;34 Suppl 1:S10-S12. doi: 10.1016/j.breast.2017.06.020. Epub 2017 Jun 30.
Trials for escalation and de-escalation of treatment aim to improve patient care, but from different sides of the same coin with respect to disease control and burdens of treatment. De-escalation of therapy is inherently a non-inferiority question. A design with random assignment to standard of care versus de-escalated therapy is typically implemented but ordinarily will require a large sample size. Some research questions of treatment de-escalation might be asked in select patient populations using single-arm designs. Trials of therapy escalation may better inform the care of individual patients by posing a clinically-oriented research question in an enriched patient population, or by planning analyses that exploit the heterogeneity of a broadly-defined enrolled population.
治疗升级和降级试验旨在改善患者护理，但在疾病控制和治疗负担方面是同一枚硬币的两面。治疗降级本质上是一个非劣效性问题。通常采用随机分配至标准治疗与降级治疗的设计，但通常需要大样本量。在特定患者人群中，使用单臂设计可能会提出一些治疗降级的研究问题。通过在富集患者人群中提出临床导向的研究问题，或通过计划利用广泛定义的入组人群的异质性进行分析，治疗升级试验可能会更好地为个别患者的护理提供信息。
