Efficacy of axitinib in a US cohort of patients with programmed cell death protein 1-resistant mucosal melanoma Journal Article


Authors: Lochrin, S. E.; Cugliari, M. K.; Yeh, R.; Shoushtari, A. N.
Article Title: Efficacy of axitinib in a US cohort of patients with programmed cell death protein 1-resistant mucosal melanoma
Abstract: Mucosal melanoma is a rare melanoma subtype, accounting for about 1% of all diagnosed melanomas. It is characterized by an aggressive phenotype with a poor prognosis and a low response rate to approved treatments. We retrospectively analyzed the clinical features, treatments, and outcomes of patients diagnosed with mucosal melanoma treated with axitinib ± anti-programmed cell death protein 1 (PD-1) therapy at a single US referral center between 2018 and 2021. Radiologic response was assessed according to Response Evaluation Criteria in Solid Tumors (RECIST), v1.1. Twenty-three patients were included in this study. In all, 78% were females with a median age of 62 years. The originating site of mucosal melanoma was the sinonasal (35%), genitourinary (35%), and gastrointestinal (30%) tracts. Sixty-five percent of patients had M1c or M1d disease and 0% had BRAF V600 mutations detected. The majority (96%) had prior treatment inclusive of anti-PD-1, with a median of 2 prior lines, and 78% of patients received a combination of axitinib and PD-1 and the median duration of treatment was 3.2 months. The overall response rate was 13% and the disease control rate was 26%. The median progression-free survival was 3.2 months, and the median overall survival was 8.2 months. Overall, the regimen was well tolerated with 39% of patients requiring dose reduction and 9% requiring treatment cessation. Axitinib with anti-PD-1 therapy has modest clinical activity in heavily pretreated patients with mucosal melanoma outside of Asia, including some with long-term benefits. This data supports the worldwide clinical trials evaluating this combination and the role of incorporating vascular endothelial growth factor-based therapy in the therapeutic paradigm for patients with mucosal melanoma. © Copyright 2024 Wolters Kluwer Health, Inc. All rights reserved.
Keywords: adult; cancer chemotherapy; cancer survival; clinical article; aged; aged, 80 and over; middle aged; retrospective studies; unclassified drug; overall survival; clinical feature; constipation; disease course; fatigue; neutropenia; cancer recurrence; cytotoxic agent; advanced cancer; diarrhea; drug dose reduction; drug efficacy; hypertension; monotherapy; side effect; skin toxicity; systemic therapy; treatment duration; united states; cancer patient; follow up; anorexia; ipilimumab; melanoma; progression free survival; pain; cohort studies; anemia; bleeding; leukopenia; mucosa inflammation; thrombocytopenia; cohort analysis; pathology; retrospective study; urogenital tract cancer; cancer resistance; arthralgia; gastrointestinal toxicity; hyponatremia; vascular endothelial growth factor; nausea and vomiting; cholesterol; triacylglycerol; inoperable cancer; cancer control; axitinib; drug therapy; b raf kinase; amylase; mucous membrane; triacylglycerol lipase; venous thromboembolism; programmed death 1 receptor; molecularly targeted therapy; posterior reversible encephalopathy syndrome; antiangiogenesis; clinical outcome; mucosa; head and neck melanoma; mucosal melanoma; germline mutation; overall response rate; acute heart failure; body weight loss; immune checkpoint inhibitor; response evaluation criteria in solid tumors; mapk signaling; nivolumab; very elderly; real-world; humans; human; male; female; article; median survival time; programmed cell death 1 receptor; sinonasal melanoma; programmed cell death protein 1 antibody; tumor mutational burden; programmed cell death protein 1; checkpoint inhibitor therapy; central nervous system melanoma; gastrointestinal melanoma
Journal Title: Melanoma Research
Volume: 34
Issue: 5
ISSN: 0960-8931
Publisher: Lippincott Williams & Wilkins  
Date Published: 2024-10-01
Start Page: 450
End Page: 456
Language: English
DOI: 10.1097/cmr.0000000000000988
PUBMED: 38953532
PROVIDER: scopus
DOI/URL:
Notes: The MSK Cancer Center Support Grant (P30 CA008748) is acknowledge in the PDF -- Corresponding authors is MSK author: Sarah E. Lochrin -- Source: Scopus
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  1. Randy Yeh
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