Safety and efficacy of topical cidofovir to treat high-grade perianal and vulvar intraepithelial neoplasia in HIV-positive men and women. Academic Article uri icon



  • OBJECTIVE: To evaluate the safety and efficacy of topical cidofovir for treatment of high-grade squamous perianal intraepithelial neoplasia (PAIN) and vulvar intraepithelial neoplasia (VIN) lesions in HIV-positive individuals. DESIGN: Phase IIa prospective multicenter trial conducted at eight clinical sites through the AIDS Malignancy Consortium. METHODS: : HIV-positive patients with biopsy-proven high-grade PAIN that was at least 3 cm were enrolled. PAIN biopsy specimens were assessed for human papillomavirus (HPV) using PCR and type-specific HPV probing. Participants applied 1% topical cidofovir to PAIN and VIN (if present) for six 2-week cycles. Results were designated as complete response (CR), partial response (PR) (>50% reduction in size), stable disease, or progressive disease (PD). RESULTS: Twenty-four men and nine women (eight with high-grade VIN as well) were enrolled. Mean age was 44 years and mean CD4 cell count was 412 cells/μl. HPV DNA (most commonly HPV16) was detected in all pretreatment study specimens. Twenty six (79%) participants completed treatment per protocol: CR, five (15%); PR, 12 (36%), stable disease, seven (21%); PD, two (6%) (one with a superficially invasive cancer and one with new area of high-grade PAIN). Treatment was well tolerated with most common adverse events being mild to moderate affecting lesional skin: pain/burning/irritation (25 patients) and ulceration (13 patients). CONCLUSION: Topical cidofovir had 51% efficacy in the short-term treatment of high-grade PAIN and VIN with acceptable toxicity in HIV-positive individuals. Randomized control studies with more prolonged treatment courses and longer follow-up to assess the durability of the response are needed.

publication date

  • February 20, 2013



  • Acquired Immunodeficiency Syndrome
  • Antiviral Agents
  • Anus Neoplasms
  • Carcinoma in Situ
  • Cytosine
  • Organophosphonates
  • Perineum
  • Vulvar Neoplasms


PubMed Central ID

  • PMC3759510

Scopus Document Identifier

  • 84873362386

Digital Object Identifier (DOI)

  • 10.1097/QAD.0b013e32835a9b16

PubMed ID

  • 23032420

Additional Document Info


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