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Chapter 220. Topical and Intralesional Cytotoxic Agents

Aieska De Souza; Megan M. Moore; Bruce E. Strober

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Topical Cytotoxic Agents at a Glance
  • Topical and intralesional cytotoxic agents are effective in the treatment of skin cancers and inflammatory and infectious dermatologic conditions.
  • They aim for maximum efficacy against cutaneous targets while sparing normal tissue and minimizing systemic toxicity.
  • They comprise 5-fluorouracil, nitrogen mustard, carmustine, vinblastine, bleomycin, methotrexate, podophyllin, and miltefosine.

Mechanism of Action and Formulations

Topical 5-fluorouracil (5-FU) has been used in clinical practice since the 1960s. It is a structural analogue of thymine that blocks DNA synthesis by inhibiting thymidylate synthetase (Table 220-1). Topical 5-FU is commercially available as a 0.5% cream carried in a microsphere vehicle, as a 1% solution or 1% cream, as a 2% or 5% solution, and as a 5% cream. The solution can also be used for intralesional injection.

Table 220-1 Topical and Intralesional Cytotoxic Agents Used in Dermatology: Mechanism of Action, Formulation, Main Indications, and Common Side Effects
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Table 220-1 Topical and Intralesional Cytotoxic Agents Used in Dermatology: Mechanism of Action, Formulation, Main Indications, and Common Side Effects

Drug

Mechanism of Action

Formulations

Indications

Common Side Effects

5-fluorouracil

Blockage of DNA synthesis by inhibition of thymidylate synthetase

0.5% cream (microsphere vehicle)

1%, 5% cream 1%, 2%, 5% solution

Intralesional injection

Actinic keratoses

Superficial basal cell carcinomas

Local irritation, erythema, pain, swelling, pruritus, dyspigmentation, allergic contact dermatitis, photosensitivity

Mechlorethamine (nitrogen mustard)

Alkylating activity, immune stimulation

Compounded: 0.01%–0.04% aqueous solution, ointment

Mycosis Fungoides

Stages IA, IB

Irritant or allergic contact dermatitis, immediate-type hypersensitivity, secondary cutaneous malignancies, dyspigmentation

Carmustine

Alkylation agent: inhibits DNA, RNA, and protein synthesis

Compounded: solution or ointment 10 mg or 20 mg/dL

Mycosis fungoides

Stages IA, IB

Myelosupression, leukopenia, erythema, skin tenderness, and telangiectasia

Vinblastine

Antimitotic agent: acts by disrupting microtubules, blocking cell division in metaphase

Intralesional injection: 0.1–0.6 mg/mL in saline solution

Kaposi sarcoma

Local pain, hyperpigmentation

Rarely: mononeuropathy, transient flu-like syndrome

Bleomycin

Disrupts DNA synthesis and causes scission of DNA strands

Intralesional injection: 1 U/mL solution

Viral warts, hemangiomas, keloids and hypertrophic scars

Local pain and swelling

Methotrexate

Antimetabolite: interferes with DNA synthesis (inhibition of dihydrofolate reductase)

Intralesional injection: 5–75 mg

Keratoacanthoma

Hematologic toxicity, hepatotoxicity, nausea, abdominal pain, oral aphthae, reversible alopecia, pancytopenia

Podophyllin

Binds tubulin, disrupting the cellular cytoskeleton

0.5% ethanol

0.15% cream

Anogenital warts

Facial angiofibromas

Erythema, erosions, tenderness

Miltefosine

Cytotoxic: promotes phospholipid turnover and modulates membrane signal transduction by inhibition of protein kinase C

6% solution

Cutaneous metastases of breast cancer Cutaneous lymphomas

Cutaneous mastocytosis

Local: burning, erythema, and fine scaling

Indications

5-FU 5% cream has been approved by the US Food and Drug Administration (FDA) since 1970 for the treatment of actinic keratoses in any location and superficial basal cell carcinomas; 5-FU 0.5% cream is FDA approved for treatment of actinic keratoses of the face and anterior scalp. 5-FU is used twice daily until an ...

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