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

Elizabeth A. Stier, Stephen E. Goldstone, Mark H. Einstein, Naomi Jay, John M. Berry, Timothy Wilkin, Jeannette Y. Lee, Teresa M. Darragh, Maria Da Costa, Lori Panther, David Aboulafia, Joel M. Palefsky

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37 Scopus citations

Abstract

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 3cm 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 412cells/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.

Original languageEnglish (US)
Pages (from-to)545-551
Number of pages7
JournalAIDS
Volume27
Issue number4
DOIs
StatePublished - Feb 20 2013

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Keywords

  • Bowen's disease
  • HIV
  • cidofovir
  • human papillomavirus
  • perianal intraepithelial neoplasia
  • vulvar intraepithelial neoplasia

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology
  • Infectious Diseases

Cite this

Stier, E. A., Goldstone, S. E., Einstein, M. H., Jay, N., Berry, J. M., Wilkin, T., Lee, J. Y., Darragh, T. M., Da Costa, M., Panther, L., Aboulafia, D., & Palefsky, J. M. (2013). Safety and efficacy of topical cidofovir to treat high-grade perianal and vulvar intraepithelial neoplasia in HIV-positive men and women. AIDS, 27(4), 545-551. https://doi.org/10.1097/QAD.0b013e32835a9b16