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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)

  • 1Email author,
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  • 4,
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Infectious Agents and Cancer20105 (Suppl 1) :A60

  • Published:


  • Intraepithelial Neoplasia
  • Cidofovir
  • Superficial Ulceration
  • Squamous Intraepithelial Neoplasia
  • Transient Proteinuria


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

Authors’ Affiliations

Department of Obstetrics and Gynecology, Boston University Medical Center, Boston, MA, USA
Department of Surgery, Mount Sinai School of Medicine, New York, NY, USA
Montefiore Medical Center and Einstein Cancer Center, Bronx, NY, >USA
Department of Medicine, University of California, San Francisco, San Francisco, CA, USA
Division of Infectious Diseases, Weill Cornell Medical College, New York, NY, USA
Department of Biostatistics, University of Arkansas for Medical Sciences, Little Rock, AR, USA
Division of Infectious Diseases, Beth Israel Deaconess Medical Center, Boston, MA, USA
Division of Hematology and Oncology, Virginia Mason Medical Center and Department of Hematology, University of Washington, Seattle, WA, USA


© Stier et al; licensee BioMed Central Ltd. 2010

This article is published under license to BioMed Central Ltd.