Nevoid Basal Cell Carcinoma Syndrome (Basal Cell Nevus Syndrome) Medication

Updated: Jun 14, 2021
  • Author: Daniel Berg, MD; Chief Editor: Dirk M Elston, MD  more...
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Medication Summary

Topical agents such as imiquimod and 5-fluorouracil are available and have been approved for treatment of basal cell carcinoma (BCC) in the United States. These medications have a lower cure rate than surgical therapy but may be useful adjuncts in patients with multiple lesions.

Investigations and clinical trials are evaluating the use of these and other novel agents such as ALA and methyl-ALA in topical photodynamic therapy, tazarotene (Tazorac), and newly developed HH pathway inhibitors, both topical and systemic. [6] Tazarotene and photodynamic therapy are available (off-label), and vismodegib (hedgehog inhibitor) and more recently sonidegib are now FDA approved for the treatment of metastatic or locally advanaced BCC—as such, their use solely in NBCSS, while clearly effective, [32] also remains off-label at present.


Topical Skin Product

Class Summary

Agents such as imiquimod and 5-fluorouracil are approved in the United States for use topically for BCC. Despite the lower cure rate (than with surgery), imiquimod and 5-fluorouracil may play a useful role in allowing patients to treat their own selected smaller lesions, especially superficial BCCs on the trunk and extremities. In general, such medications should be considered best for use in smaller or more superficial lesions, away from critical anatomic sites.

Imiquimod (Aldara)

The precise mechanism of imiquimod on superficial BCC is unknown. It may increase tumor infiltration of lymphocytes, dendritic cells, and macrophages. It is indicated for biopsy-confirmed, primary superficial BCC in adults with normal immune systems. Additionally, tumors generally should not exceed 2 cm in diameter on certain areas of the body. It is indicated primarily when surgical methods are not appropriate, including in situations in which, because of multiple lesions, scarring from surgical methods may be a consideration.

5-Fluorouracil (Efudex, Carac, Fluoroplex)

5-Fluorouracil is a cycle-specific agent that has activity as single agent and, for many years, has been combined with the biochemical modulator leucovorin. It is shown to be effective in an adjuvant setting. 5-Fluorouracil inhibits tumor cell growth through at least 3 different mechanisms that ultimately disrupt DNA synthesis or cellular viability. It is used topically for the management of superficial BCC.