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

Autor: Ismail RK; Dutch Institute for Clinical Auditing, Leiden, the Netherlands. r.ismail@dica.nl.; Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht, The Netherlands. r.ismail@dica.nl.; Medicines Evaluation Board, Utrecht, The Netherlands. r.ismail@dica.nl., Schramel FMNH; Department of Pulmonary Diseases, St Antonius Hospital, Nieuwegein, The Netherlands., van Dartel M; Medicines Evaluation Board, Utrecht, The Netherlands., Pasmooij AMG; Medicines Evaluation Board, Utrecht, The Netherlands., Cramer-van der Welle CM; Santeon Hospital Group, Utrecht, The Netherlands., Hilarius DL; Department of Clinical Pharmacy, Rode Kruis Hospital, Beverwijk, The Netherlands., de Boer A; Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht, The Netherlands.; Medicines Evaluation Board, Utrecht, The Netherlands., Wouters MWJM; Dutch Institute for Clinical Auditing, Leiden, the Netherlands.; Department of Biomedical Data Sciences, Leiden University Medical Center, Leiden, The Netherlands., van de Garde EMW; 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.
Jazyk: angličtina
Zdroj: BMC medical research methodology [BMC Med Res Methodol] 2023 Jan 03; Vol. 23 (1), pp. 1. Date of Electronic Publication: 2023 Jan 03.
DOI: 10.1186/s12874-022-01760-0
Abstrakt: Background: Many 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.
Methods: This 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.
Results: Real-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).
Conclusions: This 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.
(© 2022. The Author(s).)
Databáze: MEDLINE
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