Residual disease distribution and risk stratification for staged surgery in pediatric congenital cholesteatoma

To investigate residual disease in congenital cholesteatoma (CC), focusing on its anatomical distribution, and to explore a risk-stratified surgical hypothesis.

In this retrospective review, 72 children (72 ears; age ≤15 years) who underwent surgery for CC between January 2013 and July 2023 were included. The main outcomes were the cumulative incidence of residual disease after the initial surgery, estimated using the Kaplan-Meier method and stratified by Potsic stage, the anatomical distribution of residual cholesteatoma, and risk factors for residual disease.

Among 58 ears with at least 3 years of follow-up, the cumulative incidence of residual disease differed significantly by Potsic stage (8.3%, 20.0%, 26.7%, and 47.6% for stages 1-4, respectively; log-rank p = 0.022; p = 0.004 for stage 4 vs. stages 1-3). Residual cholesteatoma was identified at the planned second-stage surgery in 31.7% (13/41) of ears. Of the 19 residual sites, 14 (74%) clustered around the stapes and tympanic sinus. Potsic stage 4 (mastoid extension) was a significant risk factor for residual disease (OR, 5.00; 95% CI, 1.54-16.26; p = 0.005).

Potsic stage 4 and involvement of the stapes or tympanic sinus were significant risk factors for residual disease. We propose, as a hypothesis for prospective evaluation, a risk-stratified approach in which planned second-stage surgery is prioritized for high-risk ears with mastoid extension or stapes or tympanic sinus involvement, where intraoperative clearance cannot be confirmed, whereas routine re-exploration may be avoidable in selected low-risk cases.