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- Title
Long-Term Survival in Patients With Advanced Melanoma.
- Authors
van Not, Olivier J.; van den Eertwegh, Alfons J. M.; Jalving, Hilde; Bloem, Manja; Haanen, John B.; van Rijn, Rozemarijn S.; Aarts, Maureen J. B.; van den Berkmortel, Franchette W. P. J.; Blank, Christian U.; Boers-Sonderen, Marye J.; de Groot J. W. B., Jan Willem; Hospers, Geke A. P.; Kapiteijn, Ellen; Leeneman, Brenda; D., Piersma; Stevense-den Boer, Marion; van der Veldt, Astrid A. M.; Vreugdenhil G., Gerard; Wouters, Michel W. J. M.; Blokx, Willeke A. M.
- Abstract
This cohort study evaluates long-term survival in patients with advanced melanoma treated with immune checkpoint inhibitors outside clinical trials. Key Points: Question: What are the long-term outcomes of patients with advanced melanoma who are treated in daily clinical practice, and do they have a chance of sustaining long-term survival? Findings: In this cohort study including 2490 patients with advanced melanoma treated with first-line immune checkpoint inhibitors, survival curves flattened over time, suggesting long-term survival. Meaning: These findings suggest patients treated in clinical practice for advanced melanoma may reach a plateau phase in their survival after treatment with immune checkpoint inhibitors, as is seen in clinical trials. IMPORTANCE: Long-term survival data from clinical trials show that survival curves of patients with advanced melanoma treated with immune checkpoint inhibitors (ICIs) gradually reach a plateau, suggesting that patients have a chance of achieving long-term survival. OBJECTIVE: To investigate long-term survival in patients with advanced melanoma treated with ICIs outside clinical trials. DESIGN, SETTING, AND PARTICIPANTS: Cohort study using prospectively collected data from the nationwide Dutch Melanoma Treatment Registry, including patients in the Netherlands with advanced melanoma treated with first-line ICIs from 2012 to 2019. Data were analyzed from January to September 2023. EXPOSURES: Patients were treated with first-line ipilimumab-nivolumab, antibodies that target programmed cell death (anti–PD-1), or ipilimumab. MAIN OUTCOMES AND MEASURES: Progression-free survival (PFS) and melanoma-specific survival were analyzed, and a Cox proportional hazards model was used to investigate factors associated with PFS after reaching partial response (PR) or complete response (CR). RESULTS: A total of 2490 patients treated with first-line ICIs were included (median [IQR] age, 65.0 [55.3-73.0] years; 1561 male patients [62.7%]). Most patients had an Eastern Cooperative Oncology Group Performance Status of 1 or lower (2202 patients [88.5%]) and normal lactate dehydrogenase levels (1715 patients [68.9%]). PFS for all patients was 23.4% (95% CI, 21.7%-25.2%) after 3 years and 19.7% (95% CI, 18.0%-21.4%) after 5 years. Overall survival for all patients was 44.0% (95% CI, 42.1%-46.1%) after 3 years and 35.9% (95% CI, 33.9%-38.0%) after 5 years. Patients with metastases in 3 or more organ sites had a significantly higher hazard of progression after reaching PR or CR (adjusted hazard ratio, 1.37; 95% CI, 1.11-1.69). CONCLUSIONS AND RELEVANCE: This cohort study of patients with advanced melanoma treated with ICIs in clinical practice showed that their survival reached a plateau, comparable with patients participating in clinical trials. These findings can be used in daily clinical practice to guide long-term surveillance strategies and inform both physicians and patients regarding long-term treatment outcomes.
- Subjects
NETHERLANDS; THERAPEUTIC use of antineoplastic agents; PEARSON correlation (Statistics); T-test (Statistics); DEATH; PROGRAMMED death-ligand 1; CLINICAL trials; BRAIN; REPORTING of diseases; CANCER patients; LACTATE dehydrogenase; DESCRIPTIVE statistics; CHI-squared test; MANN Whitney U Test; MULTIVARIATE analysis; IMMUNE checkpoint inhibitors; LONGITUDINAL method; KAPLAN-Meier estimator; METASTASIS; NIVOLUMAB; PROGRESSION-free survival; CONFIDENCE intervals; GENETIC mutation; LIVER; CUTANEOUS malignant melanoma; IPILIMUMAB; PROPORTIONAL hazards models; OVERALL survival; MEDICAL practice; REGRESSION analysis; PATIENT aftercare; EVALUATION
- Publication
JAMA Network Open, 2024, Vol 7, Issue 8, pe2426641
- ISSN
2574-3805
- Publication type
Article
- DOI
10.1001/jamanetworkopen.2024.26641