Sorafenib in advanced melanoma: a Phase II randomised discontinuation trial analysis - PubMed (original) (raw)
Clinical Trial
. 2006 Sep 4;95(5):581-6.
doi: 10.1038/sj.bjc.6603291. Epub 2006 Aug 1.
T Ahmad, K T Flaherty, M Gore, S Kaye, R Marais, I Gibbens, S Hackett, M James, L M Schuchter, K L Nathanson, C Xia, R Simantov, B Schwartz, M Poulin-Costello, P J O'Dwyer, M J Ratain
Affiliations
- PMID: 16880785
- PMCID: PMC2360687
- DOI: 10.1038/sj.bjc.6603291
Clinical Trial
Sorafenib in advanced melanoma: a Phase II randomised discontinuation trial analysis
T Eisen et al. Br J Cancer. 2006.
Abstract
The effects of sorafenib--an oral multikinase inhibitor targeting the tumour and tumour vasculature--were evaluated in patients with advanced melanoma enrolled in a large multidisease Phase II randomised discontinuation trial (RDT). Enrolled patients received a 12-week run-in of sorafenib 400 mg twice daily (b.i.d.). Patients with changes in bi-dimensional tumour measurements <25% from baseline were then randomised to sorafenib or placebo for a further 12 weeks (ie to week 24). Patients with > or =25% tumour shrinkage after the run-in continued on open-label sorafenib, whereas those with > or =25% tumour growth discontinued treatment. This analysis focussed on secondary RDT end points: changes in bi-dimensional tumour measurements from baseline after 12 weeks and overall tumour responses (WHO criteria) at week 24, progression-free survival (PFS), safety and biomarkers (BRAF, KRAS and NRAS mutational status). Of 37 melanoma patients treated during the run-in phase, 34 were evaluable for response: one had > or =25% tumour shrinkage and remained on open-label sorafenib; six (16%) had <25% tumour growth and were randomised (placebo, n=3; sorafenib, n=3); and 27 had > or =25% tumour growth and discontinued. All three randomised sorafenib patients progressed by week 24; one remained on sorafenib for symptomatic relief. All three placebo patients progressed by week-24 and were re-started on sorafenib; one experienced disease re-stabilisation. Overall, the confirmed best responses for each of the 37 melanoma patients who received sorafenib were 19% stable disease (SD) (ie n=1 open-label; n=6 randomised), 62% (n=23) progressive disease (PD) and 19% (n=7) unevaluable. The overall median PFS was 11 weeks. The six randomised patients with SD had overall PFS values ranging from 16 to 34 weeks. The most common drug-related adverse events were dermatological (eg rash/desquamation, 51%; hand-foot skin reaction, 35%). There was no relationship between V600E BRAF status and disease stability. DNA was extracted from the biopsies of 17/22 patients. Six had V600E-positive tumours (n=4 had PD; n=1 had SD; n=1 unevaluable for response), and 11 had tumours containing wild-type BRAF (n=9 PD; n=1 SD; n=1 unevaluable for response). In conclusion, sorafenib is well tolerated but has little or no antitumour activity in advanced melanoma patients as a single agent at the dose evaluated (400 mg b.i.d.). Ongoing trials in advanced melanoma are evaluating sorafenib combination therapies.
Figures
Figure 1
BRAF mutational status of advanced melanoma patients is not associated with disease status (status of 15 patients evaluable for response). These patients were also evaluated for oncogenic BRAF mutations within exon 11 and oncogenic NRAS and KRAS mutations. One oncogenic NRAS (61K) mutation was found. No other oncogenic NRAS or KRAS mutations were detected. Two patients were unevaluable for response (_n_=1 wild-type BRAF; _n_=1 BRAF V600E) and are not included in the above histogram. DNA could not be extracted from a further five biopsies. The mutational status of the biopsies from five patients was, therefore, not determined (_n_=3 PD; _n_=2 SD).
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