Intralesional cidofovir for pediatric recurrent respiratory papillomatosis

OBJECTIVE: To compare disease response among children with recurrent respiratory papillomatosis (RRP) who underwent combined surgical debulking and intralesional cidofovir injections vs repeated surgical debulking only. DESIGN: Retrospective medical record review; follow-up range: 16 to 56 months. SETTING: Tertiary care children's hospital. PATIENTS: Seven children with RRP, including 4 subjects treated with cidofovir and 3 controls matched for age and initial papilloma staging score. INTERVENTIONS: Subjects treated with cidofovir underwent combined surgical debulking and intralesional cidofovir injection every 2 months until disease remission. Control subjects underwent repeated surgical debulking at individually determined intervals. MAIN OUTCOME MEASURES: Intraoperative endoscopic photographs were retrospectively assigned papilloma staging scores. Cidofovir and control group comparisons were made using nonparametric 2-sample Wilcoxon rank-sum (Mann-Whitney) testing, and comparisons of initial and final papilloma staging scores were made using nonparametric matched-pair Wilcoxon signed-rank testing. RESULTS: The final cidofovir group scores were significantly lower than the control group scores (P < .05). Within-group differences between initial and final scores were not significant (cidofovir group, P = .07; control group, P = .29). CONCLUSIONS: Four children with RRP were safely and successfully treated with intralesional cidofovir injection. Consideration should be given to using cidofovir more widely for treatment of pediatric RRP. Larger numbers in the cidofovir and control groups are needed in future studies to determine the true impact of cidofovir on management of this disease.