A phase I study of intra-anal artesunate (suppositories) to treat anal high-grade squamous intraepithelial lesions

by Sandy Hwang Fang, Mihaela Plesa, Evie H. Carchman, Nicole A. Cowell, Emily Staudt, Kyleigh Ann Twaroski, Ulrike K. Buchwald, Cornelia L. Trimble

Background

Ablation or surgical excision is the typical treatment of anal high-grade squamous intraepithelial lesions (HSIL). Recurrences are common due to the persistence of underlying human papillomavirus (HPV) infection. Additional well-tolerated and effective non-surgical options for HPV-associated anal disease are needed.

Methods

This 3+3 dose escalation Phase I clinical trial evaluated the safety and tolerability of artesunate suppositories in the treatment of patients with biopsy-proven HSIL.

Results

The maximal tolerated dose was 400 mg, administered in 3 cycles. All adverse events associated with the use 200- and 400-mg artesunate suppositories were Grade 1. At the 600-mg dose, patients experienced clinically significant nausea.

Conclusion

Artesunate suppositories are a safe treatment option for anal HSIL.