Please use this identifier to cite or link to this item: https://ahro.austin.org.au/austinjspui/handle/1/30372
Title: Single-agent anti-PD-1 or combined with ipilimumab in patients with mucosal melanoma: an international, retrospective, cohort study.
Austin Authors: Dimitriou, F;Namikawa, K;Reijers, I L M;Buchbinder, E I;Soon, J A;Zaremba, A;Teterycz, P;Mooradian, M J;Armstrong, E;Nakamura, Y;Vitale, M G;Tran, L E;Bai, X;Allayous, C;Provent-Roy, S;Indini, A;Bhave, P;Farid, M;Kähler, K C;Mehmi, I;Atkinson, V;Klein, Oliver ;Stonesifer, C J;Zaman, F;Haydon, A;Carvajal, R D;Hamid, O;Dummer, R;Hauschild, A;Carlino, M S;Mandala, M;Robert, C;Lebbe, C;Guo, J;Johnson, D B;Ascierto, P A;Shoushtari, A N;Sullivan, R J;Cybulska-Stopa, B;Rutkowski, P;Zimmer, L;Sandhu, S ;Blank, C U;Lo, S N;Menzies, A M;Long, G V
Affiliation: Melanoma Institute Australia, The University of Sydney, Sydney, Australia..
Department of Dermatology, University Hospital Zurich (USZ) and University of Zurich (UZH), Zurich, Switzerland..
Faculty of Medicine and Health, The University of Sydney, Sydney, Australia..
Department of Dermatologic Oncology, National Cancer Center Hospital, Tokyo, Japan..
Department of Medical Oncology and Immunology, Netherlands Cancer Institute, Amsterdam, The Netherlands..
Melanoma Disease Center, Dana-Farber Cancer Institute, Boston, USA..
Department of Medical Oncology, Peter MacCallum Cancer Centre, Melbourne, Australia..
Department of Dermatology, University Hospital Essen, Essen, Germany..
Department of Soft Tissue/Bone Sarcoma and Melanoma, Maria Sklodowska-Curie National Research Institute of Oncology, Warsaw, Poland..
Division of Oncology and Hematology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, USA..
Department of Medicine, Melanoma Service, Memorial Sloan Kettering Cancer Center, New York, USA..
Department of Skin Oncology/Dermatology, Saitama Medical University International Medical Center, Saitama, Japan..
Istituto Nazionale Tumori IRCCS Fondazione 'G. Pascale', Napoli, Italy..
Department of Medicine, Vanderbilt University Medical Center, Nashville, USA..
Key Laboratory of Carcinogenesis and Translational Research (Ministry of Education/Beijing), Department of Melanoma and Sarcoma, Peking University Cancer Hospital & Institute, Beijing, China..
APHP Hôpital Saint-Louis, Dermatology Department, DMU ICARE, Paris, France..
Dermatology Service, Department of Medicine, Gustave Roussy and Paris-Saclay University, Villejuif, France..
Unit of Medical Oncology, Ospedale di Circolo e Fondazione Macchi, ASST Sette Laghi, Varese, Italy..
Westmead and Blacktown Hospitals, Sydney, Australia..
Division of Medical Oncology, National Cancer Centre Singapore, Singapore, Singapore..
Department of Dermatology, University Hospital Schleswig-Holstein, Campus Kiel, Germany..
Department of Hematology/Oncology, The Angeles Clinic and Research Institute, a Cedars-Sinai Affiliate, Los Angeles, USA..
Princess Alexandra Hospital, Greenslopes Private Hospital, University of Queensland, Queensland, Australia..
Medical Oncology
Olivia Newton-John Cancer Research Institute
Columbia University Irving Medical Center, New York City, USA..
Alfred Hospital, Melbourne, Australia..
Department of Dermatology, University Hospital Zurich (USZ) and University of Zurich (UZH), Zurich, Switzerland..
Department of Medical Oncology, Royal North Shore and Mater Hospitals, Sydney, Australia..
Unit of Medical Oncology, University of Perugia, Perugia, Italy..
Université de Paris, APHP Hôpital Saint-Louis, Dermatology Department, DMU ICARE, INSERM U-976, Paris, France..
Department of Clinical Oncology, Maria Sklodowska-Curie National Research Institute of Oncology, Cracow, Poland..
Issue Date: 2022
Date: 2022
Publication information: Annals of oncology: Official Journal of the European Society for Medical Oncology 2022; 33(9): 968-980
Abstract: Mucosal melanoma (MM) is a rare melanoma subtype with distinct biology and poor prognosis. Data on the efficacy of immune checkpoint inhibitors (ICIs) is limited. We determined the efficacy of ICIs in MM, analysed by primary site and ethnicity/race. Retrospective cohort study from 25 cancer centres in Australia, Europe, USA and Asia. Patients with histologically confirmed MM were treated with anti-PD1+/-ipilimumab. Primary endpoints were response rate (RR), progression-free survival (PFS), overall survival (OS) by primary site (naso-oral, urogenital, anorectal, other), ethnicity/race (Caucasian, Asian, Other) and treatment. Univariate and multivariate Cox proportional hazard model analyses were conducted. In total, 545 patients were included: 331 (63%) Caucasian, 176 (33%) Asian and 20 (4%) Other. Primary sites included 113 (21%) anorectal, 178 (32%) urogenital, 206 (38%) naso-oral and 45 (8%) other. 348 (64%) received anti-PD1 and 197 (36%) anti-PD1/ipilimumab. RR, PFS and OS did not differ by primary site, ethnicity/race or treatment. RR for naso-oral was numerically higher for anti-PD1/ipilimumab (40%, 95% CI 29-54%) compared with anti-PD1 (29%, 95% CI 21-37%). 35% of patients that initially responded progressed. Median duration of response (mDOR) was 26 months (95% CI 18-NR [Not Reached]). Factors associated with short PFS were ECOG PS ≥3 (p<0.01), LDH >ULN (p=0.01), lung metastases (p<0.01) and ≥1 previous treatments (p<0.01). Factors associated with short OS were ECOG PS ≥1 (p<0.01), LDH >ULN (p=0.03), lung metastases (p<0.01) and ≥1 previous treatments (p<0.01). MM has poor prognosis. Treatment efficacy of anti-PD1+/-ipilimumab was similar and did not differ by ethnicity/race. Naso-oral primaries had numerically higher response to anti-PD1/ipilimumab, without difference in survival. The addition of ipilimumab did not show greater benefit over anti-PD1 for other primary sites. In responders, mDOR was short and acquired resistance was common. Other factors, including site and number of metastases were associated with survival.
URI: https://ahro.austin.org.au/austinjspui/handle/1/30372
DOI: 10.1016/j.annonc.2022.06.004
Journal: Annals of oncology : official journal of the European Society for Medical Oncology
PubMed URL: 35716907
PubMed URL: https://pubmed.ncbi.nlm.nih.gov/35716907/
Type: Journal Article
Subjects: Anti-PD-1
ethnicity/race
immunotherapy
ipilimumab
mucosal melanoma
Appears in Collections:Journal articles

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