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

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

doi: 10.1097/QAD.0b013e32835a9b16
Clinical Science

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 cm2 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.

aDepartment of Obstetrics and Gynecology, Boston University Medical Center, Boston, Massachusetts

bMount Sinai School of Medicine, New York

cAlbert Einstein College of Medicine, Albert Einstein Cancer Center, Montefiore Medical Center, Bronx

dUniversity of California at San Francisco, San Francisco, California

eDivision of Infectious Diseases, Weill Cornell Medical College, New York, New York

fUniversity of Arkansas for Medical Sciences, Little Rock, Arkansas

gDepartment of Pathology, University of California at San Francisco, San Francisco, California

hBeth Israel Deaconess Medical Center, Boston, Massachusetts

iVirginia Mason Medical Center and University of Washington, Seattle, Washington, USA.

Correspondence to Elizabeth A. Stier, Department of Obstetrics and Gynecology, Boston University Medical Center, 85 E. Concord Street, 6th floor, Boston, MA 02118, USA. E-mail:

Received 3 August, 2012

Revised 14 September, 2012

Accepted 20 September, 2012

Copyright © 2013 Wolters Kluwer Health, Inc.