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Phase IIA trial of 1% topical cidofovir for treatment of high-grade perianal squamous intraepithelial neoplasia in HIV-infected men and women (AMC046)


Treatments for high-grade perianal intraepithelial neoplasia (PAIN 2-3), include surgical ablation/excision and have significant morbidity and recurrence rates. Cidofovir, a cytidine nucleotide analogue, has broad-spectrum antiviral activity. This multicenter study prospectively evaluated the efficacy, safety, and tolerability of topical cidofovir for treatment of PAIN 2-3 in HIV-positive individuals.


HIV-positive patients with biopsy-proven PAIN 2-3 ≥ 3 cm2 were eligible. Subjects applied 1% topical cidofovir for 6 two-week cycles consisting of 5 consecutive days of treatment and 9 days without treatment. Subjects were evaluated every 2 weeks. High-resolution anoscopy and biopsy were performed 6 weeks after the last cycle. Results were scored as stable disease (SD), partial response (PR) (> 50% reduction in size), complete response (CR), or progressive disease (PD) based on size and histology.


24 men and 9 women were enrolled. Mean age was 33 years, median HIV RNA level was <75 copies/ml, and mean CD4 count was 440/µl. HPV DNA was detected in intra-anal swabs of 31 of 32 (97%) subjects with analyzable specimens. The most common type was HPV16 (44%).

27 (82%) subjects completed treatment per protocol—CR: 4 (15%); PR: 12 (44%); SD: 9 (33%); PD: 2 (7%) (1 with a superficially invasive cancer and 1 with new PAIN 2-3). Six subjects did not complete treatment because of discomfort (1), poor compliance (4), and CR after 4 cycles (1).

26 of 33 subjects (79%) reported adverse events likely related to treatment. Most were mild or moderate, including self-limited, localized, superficial ulcerations in the disease area (2 mild, 19 moderate, 1 severe), discomfort (4 mild, 14 moderate), itching (1 mild, 3 moderate), and bleeding (6 mild). Seven (21%) had mild transient proteinuria.


Topical cidofovir is a well-tolerated and effective treatment for PAIN 2-3 in HIV-positive patients. A larger study is warranted.


This article has been published as part of Infectious Agents and Cancer Volume 5 Supplement 1, 2010: Proceedings of the 12th International Conference on Malignancies in AIDS and Other Acquired Immunodeficiencies (ICMAOI).The full contents of the supplement are available online at

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Correspondence to Elizabeth A Stier.

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Open Access This article is published under license to BioMed Central Ltd. This is an Open Access article is distributed under the terms of the Creative Commons Attribution 2.0 International License (, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.

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Stier, E.A., Goldstone, S.E., Einstein, M.H. et al. Phase IIA trial of 1% topical cidofovir for treatment of high-grade perianal squamous intraepithelial neoplasia in HIV-infected men and women (AMC046). Infect Agents Cancer 5 (Suppl 1), A60 (2010).

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