Phase 2 study of sunitinib in patients with metastatic mucosal or acral melanoma

Elizabeth I. Buchbinder, Jeffrey A. Sosman, Donald P. Lawrence, David F. McDermott, Nikhil H. Ramaiya, Annick D. Van Den Abbeele, Gerald P. Linette, Anita Giobbie-Hurder, F. Stephen Hodi*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

42 Scopus citations


BACKGROUND Patients with mucosal and acral melanomas have limited treatment options and a poor prognosis. Mutations of the KIT oncogene in these melanoma subtypes provide a potential therapeutic target. METHODS A multicenter phase 2 trial of sunitinib was conducted in patients with unresectable stage III or IV melanoma of a mucosal or acral primary origin. Patients were treated in 2 cohorts: cohort A received sunitinib at a dose of 50 mg daily for 4 weeks of a 6-week cycle, and cohort B received sunitinib at a dose of 37.5 mg daily on a continuous basis. Dose reductions were permitted for treatment-related toxicities, and tumor assessments were performed every 2 months. RESULTS Fifty-two patients were enrolled: 21 in cohort A and 31 in cohort B. Four patients had confirmed partial responses, which lasted 5 to 10 months (1 with a KIT mutation). In both cohorts, the proportion of patients alive and progression-free at 2 months was 52% (95% confidence interval, 38%-66%); this was significantly larger than the hypothesized null of 5%. There was no significant difference in response or overall survival between the 25% of patients with a KIT mutation and those without one (response rate, 7.7% vs 9.7%; overall survival, 6.4 vs 8.6 months). The overall disease control rate was 44%, and a high rate of toxicity was associated with the treatment. CONCLUSIONS Sunitinib showed activity in the treatment of mucosal and acral melanoma that was not dependent on the presence of a KIT mutation. However, the medication was poorly tolerated, and there were no prolonged responses. Cancer 2015;121:4007-4015.

Original languageEnglish (US)
Pages (from-to)4007-4015
Number of pages9
Issue number22
StatePublished - Nov 15 2015


  • KIT
  • acral melanoma
  • mucosal melanoma
  • sunitinib
  • vascular endothelial growth factor (VEGF)

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


Dive into the research topics of 'Phase 2 study of sunitinib in patients with metastatic mucosal or acral melanoma'. Together they form a unique fingerprint.

Cite this