4.7 Article

Surrogacy analysis of intermediate end-points for overall survival in randomized controlled trials of rhabdomyosarcoma

Journal

SCIENTIFIC REPORTS
Volume 12, Issue 1, Pages -

Publisher

NATURE PORTFOLIO
DOI: 10.1038/s41598-022-23944-w

Keywords

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Funding

  1. National Cancer Center Research and Development Fund [2020-J-3]
  2. Japan Agency for Medical Research and Development (AMED) [JP21ck0106614, JP21ck0106507]

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In this study, a systematic review and meta-analysis were conducted to assess the surrogacy of intermediate end-points for overall survival (OS) in patients with rhabdomyosarcoma (RMS). The results showed a marginal correlation between event-free survival (EFS) and OS, but the surrogacy of EFS for OS cannot be confirmed.
Treatment of malignant tumors, such as rhabdomyosarcoma (RMS), can improve overall survival (OS). It is time-consuming and expensive for patients to obtain benefits from randomized controlled trials (RCTs) with OS as the primary end-point. Therefore, another surrogate end-point is necessary; however, there is no report on the surrogacy analysis of RMS. In this study, we performed a systematic review of RCTs, involving patients with newly diagnosed RMS, and 11 RCTs were identified. We performed a meta-analysis to assess the surrogacy of intermediate end-points for OS. The correlations between surrogate end-points and OS were investigated using Spearman's rank correlation coefficient (rho). The coefficient of determination (R-2) was employed to measure the strength of the association. A total of 5183 patients were randomly allocated to 34 treatment groups. A marginal correlation (R-2 = 0.281, rho = 0.445) between the hazard ratios (HRs) for event-free survival (EFS) and OS was observed. In patients with localized RMS, the EFS HR had a weaker correlation with OS HR in the sensitivity analysis than that in the primary analysis. Overall, the surrogacy of EFS for OS cannot be confirmed.

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