TY - JOUR
T1 - Long-term survival of patients with advanced melanoma treated with BRAF-MEK inhibitors
AU - Ismail, Rawa K.
AU - Suijkerbuijk, Karijn P.M.
AU - de Boer, Anthonius
AU - van Dartel, Maaike
AU - Hilarius, Doranne L.
AU - Pasmooij, A. M.G.
AU - van Zeijl, Michiel C.T.
AU - Aarts, Maureen J.B.
AU - van den Berkmortel, Franchette W.P.J.
AU - Blank, Christian U.
AU - Boers-Sonderen, Marye J.
AU - de Groot, Jan W.B.
AU - Haanen, John B.A.G.
AU - Hospers, Geke A.P.
AU - Kapiteijn, Ellen
AU - Piersma, Djura
AU - van Rijn, Rozemarijn S.
AU - van der Veldt, Astrid A.M.
AU - Vreugdenhil, Art
AU - Westgeest, Hans
AU - van den Eertwegh, Alfons J.
AU - Wouters, Michel W.J.M.
N1 - Publisher Copyright: Copyright © 2022 The Author(s). Published by Wolters Kluwer Health, Inc.
PY - 2022/12/1
Y1 - 2022/12/1
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=85141004079&partnerID=8YFLogxK
U2 - 10.1097/CMR.0000000000000832
DO - 10.1097/CMR.0000000000000832
M3 - Article
C2 - 35703270
AN - SCOPUS:85141004079
SN - 0960-8931
VL - 32
SP - 460
EP - 468
JO - Melanoma Research
JF - Melanoma Research
IS - 6
ER -
