Sustained improved survival of patients with metastatic melanoma after the introduction of anti-PD-1-based therapies
Menée au Danemark à partir de données portant sur des patients atteints d'un mélanome métastatique, cette étude analyse, dans un contexte de vie réelle et pour les années 2012, 2014, 2016 et 2018, l'effet, sur la survie, des anti-PD-1 en traitement de première ligne
Background: The introduction of modern therapies improved the median survival of patients with metastatic melanoma (MM). Here, we determined the real-world impact of modern treatments on the long-term survival of MM. Methods: In a population-based study, we extracted all cases of MM diagnosed in four non-consecutive years marked by major changes in available 1st line treatments (2012, 2014, 2016, and 2018) from the Danish MM Database. Patients were grouped into “trial-like” and “trial-excluded” based on common trial eligibility criteria. Results: We observed a sustained improved survival of “trial-like” patients diagnosed in 2016 or in 2018, compared to 2012 or 2014, but no major differences in 2018 versus 2016. In contrast, while survival of “trial-excluded” patients in 2016 was better compared to 2014 and 2012, survival in 2018 was improved over all previous years. We then developed a prognostic model based on multivariable stratified Cox regression, to predict the survival of newly diagnosed MM patients. Internal validation showed excellent discrimination and calibration, with a time-area-under-the-curve above 0.79 at multiple time horizons, for up to four years after diagnosis. Conclusions: The introduction of modern treatments such as anti-PD-1 has led to a sustained, improved survival of real-world patients with MM, regardless of their eligibility for clinical trials. We provide an updateable prognostic model that can be used to improve patient information. Overall, these data highlight a positive population-based impact of modern treatments and can help health technology assessment agencies worldwide to evaluate the appropriateness of drug pricing based on known cost-benefit data.