Exploring the Role of Compounded Imiquimod in Ocular Surface Cancers

Compounded imiquimod 5% ointment was evaluated in a five-patient interventional case series for ocular surface malignancies, producing focal complete histologic clearance and partial clinical regression in more extensive lesions.

Investigators reported complete clinical and histologic resolution in some cases of conjunctival intraepithelial neoplasia (CIN) and primary acquired melanosis (PAM), partial clinical response in diffuse conjunctival melanoma in situ (MIS), and only transient ophthalmic adverse events that resolved within 1–2 weeks.

The case series enrolled five patients with histologically confirmed CIN, conjunctival squamous cell carcinoma, conjunctival MIS, or conjunctival melanoma with adjacent PAM and used clinical response plus histologic clearance as primary endpoints. Outcomes were assessed at roughly 12 weeks for most lesions and up to 15 weeks for PAM, documented by map biopsies and clinical photography, and reported as focal histologic clearance versus incomplete clinical regression in diffuse disease.

Treatment was a compounded topical imiquimod 5% ointment applied to the conjunctival surface five days per week, typically for at least 12 weeks and extended to ~15 weeks for broader lesions; ointment preparations were supplied by compounding pharmacies. The cohort included cases of diffuse conjunctival melanoma in situ where pigmentation regressed clinically in part but map-biopsy sites showed complete histologic response, and no systemic dosing or maintenance regimens were reported. Time to histologic response was most commonly observed by 12 weeks and sometimes by 15 weeks, supporting follow-up biopsy or imaging after a full induction course rather than within the first month.

All five patients experienced ocular-surface and periocular adverse events only, most commonly localized irritation and eyelid inflammation; there were no systemic toxicities and no events requiring long-term discontinuation. Ocular effects resolved within one to two weeks after a drug holiday or treatment completion and were managed conservatively with temporary cessation, topical lubrication, and short courses of anti-inflammatory drops when indicated. The safety profile in this small series appears manageable with routine monitoring, though clinicians should remain vigilant for ocular-surface complications.

Key limitations include the small sample size, lack of randomization, and short, variable follow-up—constraints that limit conclusions about long-term recurrence and comparative effectiveness. The authors recommend larger prospective studies with standardized compounding and dosing protocols to define durability, optimal schedules, and how topical imiquimod compares with established surgical and adjunctive approaches. For now, the series may inform cautious, short-term consideration of topical imiquimod for select focal ocular surface lesions while prospective validation is pursued.

Key Takeaways:

• Topical compounded imiquimod 5% showed complete histologic responses in focal CIN/PAM and partial clinical regression in diffuse MIS in a five-patient case series.
• Adverse events were limited to the ocular surface, resolved within 1–2 weeks after a drug holiday, and were manageable with topical measures and monitoring.
• Evidence is preliminary; larger prospective studies with standardized compounding and follow-up are needed before routine adoption.