1. Long-term survival of patients with advanced melanoma treated with BRAF-MEK inhibitors
- Author
-
Ismail, Rawa K, Suijkerbuijk, Karijn P M, de Boer, Anthonius, van Dartel, Maaike, Hilarius, Doranne L, Pasmooij, Anna M J, van Zeijl, Michiel C T, Aarts, Maureen J B, van den Berkmortel, Franchette W P J, Blank, Christian U, Boers-Sonderen, Marye J, de Groot, Jan W B, Haanen, John B, Hospers, Geke A P, Kapiteijn, Ellen, Piersma, Djura, van Rijn, Rozemarijn S, van der Veldt, Astrid A M, Vreugdenhil, Art, Westgeest, Hans, van den Eertwegh, Alfons J, Wouters, Michel W J M, Ismail, Rawa K, Suijkerbuijk, Karijn P M, de Boer, Anthonius, van Dartel, Maaike, Hilarius, Doranne L, Pasmooij, Anna M J, van Zeijl, Michiel C T, Aarts, Maureen J B, van den Berkmortel, Franchette W P J, Blank, Christian U, Boers-Sonderen, Marye J, de Groot, Jan W B, Haanen, John B, Hospers, Geke A P, Kapiteijn, Ellen, Piersma, Djura, van Rijn, Rozemarijn S, van der Veldt, Astrid A M, Vreugdenhil, Art, Westgeest, Hans, van den Eertwegh, Alfons J, and Wouters, Michel W J M
- Abstract
Recent results of patients with advanced melanoma treated with first-line BRAF-MEK inhibitors in clinical trials showed 5-year survival in one-third of patients with a median overall survival (OS) of more than 2 years. This study aimed to investigate these patients' real-world survival and identify the characteristics of long-term survivors. The study population consisted of patients with advanced cutaneous melanoma with a BRAF-V600 mutated tumor who were treated with first-line BRAF-MEK inhibitors between 2013 and 2017. Long-term survival was defined as a minimum OS of 2 years from start therapy. The median progression-free survival (mPFS) and median OS (mOS) of real-world patients ( n = 435) were respectively 8.0 (95% CI, 6.8-9.4) and 11.7 (95% CI, 10.3-13.5) months. Two-year survival was reached by 28% of the patients, 22% reached 3-year survival and 19% reached 4-year survival. Real-world patients often had brain metastases (41%), stage IV M1c disease (87%), ECOG PS ≥2 (21%), ≥3 organ sites (62%) and elevated LDH of ≥250 U/I (49%). Trial-eligible real-world patients had an mOS of 17.9 months. Patients surviving more than 2 years ( n = 116) more often had an ECOG PS ≤1 (83%), normal LDH (60%), no brain metastases (60%), no liver metastases (63%) and <3 organ sites (60%). Long-term survival of real-world patients treated with first-line BRAF-MEK inhibitors is significantly lower than that of trial patients, which is probably explained by poorer baseline characteristics of patients treated in daily practice. Long-term survivors generally had more favorable characteristics with regard to age, LDH level and metastatic sites, compared to patients not reaching long-term survival.
- Published
- 2022