Complete response of stage IV anal mucosal melanoma expressing KIT Val560Asp to the multikinase inhibitor sorafenib

Nat Clin Pract Oncol. 2008 Dec;5(12):737-40. doi: 10.1038/ncponc1251. Epub 2008 Oct 21.


Background: A 79-year-old man presented to his primary care physician with a 2-month history of pruritus ani and a pigmented nodular lesion was discovered in the posterior rectum. The patient had no other symptoms, or any family history of malignancy.

Investigations: Physical examination; excisional biopsy; CT scan of the chest, abdomen and pelvis; lung biopsy; blood tests; tumor immunohistochemistry for KIT, vascular endothelial growth factor platelet-derived growth factor receptor alpha and beta, and mismatch-repair proteins MLH1, MSH2, and MSH6; and KIT and BRAF tumor genotyping.

Diagnosis: Stage IV M1b metastatic anal mucosal melanoma.

Management: Wide local excision with mucosal advancement of the rectal wall, external-beam radiation, and sorafenib-temozolomide therapy.

Publication types

  • Case Reports

MeSH terms

  • Aged
  • Antineoplastic Agents / pharmacology
  • Antineoplastic Agents / therapeutic use*
  • Anus Neoplasms / drug therapy
  • Anus Neoplasms / therapy*
  • Benzenesulfonates / therapeutic use*
  • Dacarbazine / analogs & derivatives
  • Dacarbazine / therapeutic use
  • Humans
  • Male
  • Melanoma / drug therapy
  • Melanoma / therapy*
  • Neoplasm Staging
  • Niacinamide / analogs & derivatives
  • Phenylurea Compounds
  • Proto-Oncogene Proteins c-kit / metabolism
  • Pyridines / therapeutic use*
  • Sorafenib
  • Temozolomide
  • Treatment Outcome


  • Antineoplastic Agents
  • Benzenesulfonates
  • Phenylurea Compounds
  • Pyridines
  • Niacinamide
  • Dacarbazine
  • Sorafenib
  • Proto-Oncogene Proteins c-kit
  • Temozolomide