Biometry Unit, Institut Curie, PSL Research University, Paris, 75005, France.
Klinikum der Landeshauptstadt Stuttgart gKAöR, Olgahospital, Stuttgart Cancer Center, Zentrum für Kinder-, Jugend- und Frauenmedizin, Pädiatrie 5 (Pädiatrische Onkologie, Hämatologie, Immunologie), Stuttgart, 70174, Germany.
Future Oncol. 2023 Aug;19(24):1645-1653. doi: 10.2217/fon-2023-0114. Epub 2023 May 3.
The EPI VITRAKVI study is a retrospective study designed to place the results of the single-arm Phase I/II larotrectinib SCOUT trial into context by comparison with external historical controls. Its primary objective is to compare the time to medical treatment failure between larotrectinib and the historical standard of care (chemotherapy) in patients with infantile fibrosarcoma. External historical cohorts have been selected by using objective criteria. The Inverse Probability of Treatment Weighting method will be used to adjust for potential confounding. The current publication illustrates how an external control arm study can complement data from a single-arm trial and addresses uncertainties encountered in the assessment of therapies targeting rare abnormalities where randomized controlled trials are considered not feasible. Clinical Trial Registration: NCT05236257 (ClinicalTrials.gov).
EPI VITRAKVI 研究是一项回顾性研究，旨在通过与外部历史对照进行比较，将单臂 I/II 期 larotrectinib SCOUT 试验的结果置于背景中。其主要目的是比较 larotrectinib 与婴儿纤维肉瘤患者的历史标准治疗（化疗）之间的医疗失败时间。外部历史队列是通过使用客观标准选择的。将使用逆概率治疗加权法（Inverse Probability of Treatment Weighting method）来调整潜在的混杂因素。本出版物说明了外部对照臂研究如何补充单臂试验的数据，并解决了在评估针对罕见异常的治疗方法时遇到的不确定性，这些异常被认为不适合进行随机对照试验。临床试验注册：NCT05236257（ClinicalTrials.gov）。
