Individual patient data to allow a more elaborated comparison of trial results with real-world outcomes from second-line immunotherapy in NSCLC

BMC Medical Research Methodology

Research

D. L. Hilarius¹  F. M. N. H. Schramel²  E. M. W. van de Garde³  A. de Boer⁴  M. W. J. M. Wouters⁵  R. K. Ismail⁶  M. van Dartel⁷  A. M. G. Pasmooij⁷  C. M. Cramer-van der Welle⁸ 

[1] Department of Clinical Pharmacy, Rode Kruis Hospital, Beverwijk, The Netherlands;Department of Pulmonary Diseases, St Antonius Hospital, Nieuwegein, The Netherlands;Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht, The Netherlands;Department of Clinical Pharmacy, St Antonius Hospital, Nieuwegein, Utrecht, The Netherlands;Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht, The Netherlands;Medicines Evaluation Board, Utrecht, The Netherlands;Dutch Institute for Clinical Auditing, Leiden, the Netherlands;Department of Biomedical Data Sciences, Leiden University Medical Center, Leiden, The Netherlands;Dutch Institute for Clinical Auditing, Leiden, the Netherlands;Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht, The Netherlands;Medicines Evaluation Board, Utrecht, The Netherlands;Medicines Evaluation Board, Utrecht, The Netherlands;Santeon Hospital Group, Utrecht, The Netherlands;

关键词: NSCLC;    Nivolumab;    Real-world;    Efficacy- effectiveness gap;   

DOI  :  10.1186/s12874-022-01760-0

 received in 2022-03-27, accepted in 2022-10-19,  发布年份 2022

来源: Springer

【 摘 要 】

BackgroundMany studies have compared real-world clinical outcomes of immunotherapy in patients with metastatic non-small cell lung cancer (NSCLC) with reported outcomes data from pivotal trials. However, any differences observed could be only limitedly explored further for causation because of the unavailability of individual patient data (IPD) from trial participants. The present study aims to explore the additional benefit of comparison with IPD.MethodsThis study compares progression free survival (PFS) and overall survival (OS) of metastatic NSCLC patients treated with second line nivolumab in real-world clinical practice (n = 141) with IPD from participants in the Checkmate-057 clinical trial (n = 292). Univariate and multivariate Cox proportional hazards models were used to construct HRs for real-world practice versus clinical trial.ResultsReal-world patients were older (64 vs. 61 years), had more often ECOG PS ≥ 2 (5 vs. 0%) and were less often treated with subsequent anti-cancer treatment (28.4 vs. 42.5%) compared to trial patients. The median PFS in real-world patients was longer (3.84 (95%CI: 3.19-5.49) vs 2.30 (2.20-3.50) months) and the OS shorter than in trial participants (8.25 (6.93-13.2) vs. 12.2 (9.90-15.1) months). Adjustment with available patient characteristics, led to a shift in the hazard ratio (HR) for OS, but not for PFS (HRs from 1.13 (0.88-1.44) to 1.07 (0.83-1.38), and from 0.82 (0.66-1.03) to 0.79 (0.63-1.00), respectively).ConclusionsThis study is an example how IPD from both real-world and trial patients can be applied to search for factors that could explain an efficacy-effectiveness gap. Making IPD from clinical trials available to the international research community allows this.

【 授权许可】

CC BY   
© The Author(s) 2022. corrected publication 2023

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