Vemurafenib in patients with BRAF(V600) mutated metastatic melanoma: An open-label, multicentre, safety study

James Larkin*, Michele Del Vecchio, Paolo A. Ascierto, Ivana Krajsova, Jacob Schachter, Bart Neyns, Enrique Espinosa, Claus Garbe, Vanna Chiarion Sileni, Helen Gogas, Wilson H. Miller, Mario Mandala, Geke A. P. Hospers, Ana Arance, Paola Queirolo, Axel Hauschild, Michael P. Brown, Lada Mitchell, Luisa Veronese, Christian U. Blank

*Corresponding author for this work

    Research output: Contribution to journalArticleAcademicpeer-review

    240 Citations (Scopus)

    Abstract

    Background The orally available BRAF kinase inhibitor vemurafenib, compared with dacarbazine, shows improved response rates, progression- free survival (PFS), and overall survival in patients with metastatic melanoma that has a BRAF(V600) mutation. We assessed vemurafenib in patients with advanced metastatic melanoma with BRAF(V600) mutations who had few treatment options.

    Methods In an open-label, multicentre study, patients with untreated or previously treated melanoma and a BRAF(V600) mutation received oral vemurafenib 960 mg twice a day. The primary endpoint was safety. All analyses were done on the safety population, which included all patients who received at least one dose of vemurafenib. This report is the third interim analysis of this study. This study is registered with ClinicalTrials.gov, number NCT01307397.

    Findings Between March 1, 2011, and Jan 31, 2013, 3226 patients were enrolled in 44 countries. 3222 patients received at least one dose of vemurafenib (safety population). At data cutoff, 868 (27%) patients were on study treatment and 2354 (73%) had withdrawn, mainly because of disease progression. Common adverse events of all grades included rash (1592 [49%]), arthralgia (1259 [39%]), fatigue (1093 [34%]), photosensitivity reaction (994 [31%]), alopecia (826 [26%]), and nausea (628 [19%]). 1480 (46%) patients reported grade 3 or 4 adverse events, including cutaneous squamous cell carcinoma (389 [12%]), rash (155 [5%]), liver function abnormalities (165 [5%]), arthralgia (106 [3%]), and fatigue (93 [3%]). Grade 3 and 4 adverse events were reported more frequently in patients aged 75 years and older (n= 257; 152 [59%, 95% CI 53- 65] and ten [4%, 2- 7], respectively) than in those younger than 75 years (n= 2965; 1286 [43%, 42- 45] and 82 [3%, 2- 3], respectively).

    Interpretation Vemurafenib safety in this diverse population of patients with BRAF(V600) mutated metastatic melanoma, who are more representative of routine clinical practice, was consistent with the safety profile shown in the pivotal trials of this drug.

    Original languageEnglish
    Pages (from-to)436-444
    Number of pages9
    JournalLancet Oncology
    Volume15
    Issue number4
    DOIs
    Publication statusPublished - Apr-2014

    Keywords

    • MALIGNANT-MELANOMA
    • BRAIN METASTASES
    • SURVIVAL
    • PROGRESSION

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