Medication Summary
Medications used for the treatment of basal cell carcinoma (BCC) include the following:
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Topical antineoplastic agents (eg, 5-fluorouracil, imiquimod)
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The photosensitizing agent methyl aminolevulinate cream
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The acetylenic retinoid tazarotene
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Hedgehog pathway inhibitors (HHIs)
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A programmed death–1 (PD-1) inhibitor
Antineoplastic Agents
Class Summary
The most common chemotherapeutic agent used in superficial basal cell carcinoma is topical 5-fluorouracil.
Fluorouracil topical (Efudex)
5-Fluorouracil topical 5% cream or solution is used topically for the management of superficial BCC. It interferes with DNA synthesis by blocking methylation of deoxyuridylic acid and inhibiting thymidylate synthetase and, subsequently, cell proliferation.
Interferon alfa-2b (Intron-A)
Interferon alfa-2b is a protein product manufactured with recombinant DNA technology. The mechanism of antitumor activity is not clearly understood; however, direct antiproliferative effects against malignant cells and modulation of host immune response may be important.
As an investigational drug for nodular BCC, interferon alfa-2b was used in a randomized, placebo-controlled multicenter study with 172 patients. Intralesional injections of 1.5 million U administered 3 times/wk for 3 wk yielded an 86% complete-response rate, compared with 29% for placebo. A similar study did not show efficacy for morpheaform or aggressive BCCs.
Antineoplastics, Hedgehog Pathway Inhibitor
Class Summary
The Hedgehog signaling pathway is important in embryogenesis, but in adults, it is mostly inactive. Signaling is relayed by key proteins including Smoothened homologue (SMO). Hedgehog ligand-expressing cancerous epithelial cells that are activated by the hedgehog signaling pathway may cause growth-promotion.
Vismodegib (Erivedge)
Vismodegib is a Hedgehog (Hh) pathway inhibitor. Vismodegib binds to and inhibits SMO, a transmembrane protein involved in Hedgehog signal transduction. This agent is indicated for treatment of adults with metastatic basal cell carcinoma or with locally advanced basal cell carcinoma that has recurred following surgery, or those who are not candidates for surgery or radiation.
Sonidegib (Odomzo)
Sonidegib binds to and inhibits SMO, a transmembrane protein involved in Hedgehog signal transduction. Indicated for adults with locally advanced basal cell carcinoma that has recurred following surgery or radiation therapy, or those who are not candidates for surgery or radiation therapy.
PD-1/PD-L1 Inhibitors
Cemiplimab (Cemiplimab-rwlc, Libtayo)
Recombinant human immunoglobulin G4 monoclonal antibody binds to PD-1 and blocks its interaction with PD-L1 and PD-L2, releasing PD-1 pathway-mediated inhibition of the immune response, including the anti-tumor immune response. It is indicated for locally advanced BCC and granted accelerated approval for metastatic BCC previously treated with a HHI or for whom a HHI is not appropriate.
Photosensitizing Agent, Topical
Class Summary
Photodynamic therapy (PDT) for basal cell carcinomas is the process of using specific wavelengths of light to photoexcite porphyrins that have been applied to neoplastic and preneoplastic cells. This increased energy is rapidly absorbed by adjacent tissue oxygen, causing the formation of singlet oxygen radicals. These radicals rapidly react with adjacent tissue and destroy it.
Methyl aminolevulinate (Metvixia)
Methyl aminolevulinate cream is a porphyrin precursor used in combination with narrow-band, red-light illumination for nonhyperkeratotic, nonpigmented actinic keratoses. When used with photodynamic therapy, the accumulation of photoactive porphyrins produces a photodynamic reaction that results in a cytotoxic process dependent upon the simultaneous presence of oxygen.
Keratolytic Agents
Class Summary
These agents decrease cohesiveness of abnormal hyperproliferative keratinocytes and may reduce potential for malignant degeneration. It modulates keratinocyte differentiation.
Tazarotene (Avage, Fabior, Tazorac)
Tazarotene is a retinoid prodrug with an active metabolite that modulates differentiation and proliferation of epithelial tissue; it also may have anti-inflammatory and immunomodulatory properties. It is not approved by the US Food and Drug Administration for treating basal cell carcinoma.
Topical Skin Products
Class Summary
Products that induce cytokines, including interferon alpha, have been shown to be effective.
Imiquimod (Aldara)
The precise mechanism of imiquimod for superficial BCC is unknown. It may increase tumor infiltration by lymphocytes, dendritic cells, and macrophages. It is indicated for biopsy-confirmed primary nonfacial superficial BCC in adults with normal immune systems. Additionally, tumors must not exceed 2 cm in diameter on certain areas of the body. Imiquimod is indicated only when surgical methods are not appropriate.
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A pink, scaly lesion on the skin. Superficial basal cell carcinoma (BCC) is often misdiagnosed as eczematous dermatitis or guttate psoriasis and is often difficult to distinguish clinically from Bowen disease (squamous cell carcinoma in situ). Features that suggest the diagnosis of superficial BCC are the absence of significant white, adherent scale and a history of the lesion remaining unchanged for several months or years. Treatment options for this tumor include electrodesiccation and curettage, surgical excision, cryosurgery, 5-fluorouracil, 5% imiquimod cream, and superficial radiotherapy. Electrodesiccation and curettage is the modality most commonly used, with a cure rate of approximately 95%.
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Basal cell carcinoma.
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A 68-year-old patient presenting with an advanced basal cell carcinoma (BCC) of the right periorbital region, frontal view. Courtesy of M Abraham Kuriakose, DDS, MD.
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Lateral view of face showing extent of tumor. Courtesy of M Abraham Kuriakose, DDS, MD.
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Basal cell carcinoma of the right lower lid.
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Biopsy-proven basal cell carcinoma of the upper lid margin. Note the loss of cilia (madarosis) in the area of the tumor.
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Medial canthal/lower lid basal cell. Note the pearly nodular surface with characteristic telangiectatic vessels. Proximity to the lacrimal system will impact its treatment and reconstruction.
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Nodular basal cell carcinoma.
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Nodular basal cell carcinoma appearing as a waxy, translucent papule with central depression and a few small erosions.
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Scale, erythema, and a threadlike raised border are present in this superficial basal cell carcinoma on the trunk.
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Large, superficial basal cell carcinoma.
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Basal cell carcinoma. Courtesy of Hon Pak, MD.
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Pigmented basal cell carcinoma.
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Pigmented basal cell carcinoma.
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Pigmented basal cell carcinoma has features of nodular basal cell carcinoma with the addition of dark pigmentation from melanin deposition. The pigmentation often has the appearance of dark droplets in the lesion, as shown here.
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This infiltrating basal cell cancer has ill-defined borders and telangiectases.
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This translucent pink papule has telangiectases and a crusted erosion, characteristic of nodular basal cell carcinoma.
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Large, scarlike morpheaform basal cell cancer.
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Nodular basal cell carcinoma. Nodular aggregates of basalioma cells are present in the dermis and exhibit peripheral palisading and retraction artifact. Melanin is also present within the tumor and in the surrounding stroma, as seen in pigmented basal cell carcinoma.
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Histologic pattern of a well-differentiated basal cell carcinoma (original magnification X140). Courtesy of Prof Pantaleo Bufo, University of Foggia, Italy.
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Histologic pattern of a well-differentiated basal cell carcinoma (original magnification X250). Courtesy of Prof Pantaleo Bufo, University of Foggia, Italy.
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Micronodular basal cell carcinoma often has an absence of retraction artifact. The characteristic histology is small size and uniformity of the tumor nodules. Courtesy of Shang I Brian Jiang, MD.
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Infiltrative basal cell carcinoma. Tumor cells are arranged in narrow strands, and mucin-rich stroma is often present. Courtesy of Shang I Brian Jiang, MD.
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Keratotic basal cell carcinoma. Rare type characterized by keratocysts. Courtesy of Shang I Brian Jiang, MD.
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Basosquamous basal cell carcinoma. Foci of neoplastic cells with squamous differentiation are present. Courtesy of Shang I Brian Jiang, MD.
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Histology of superficial basal cell carcinoma. Nests of basaloid cells are seen budding from the undersurface of the epidermis. Courtesy of Michael L Ramsey, MD.
