Objectives: We assessed the intermediate-term and long-term efficacy of intralesional injection of cidofovir used with surgical excision in juvenile-onset recurrent respiratory papillomatosis.
Methods: The protocol was revised during the study, from endoscopy at 4-week intervals with intralesional injection of cidofovir at 5 mg/mL, to a 2-week interval and a 7.5-mg/mL dosage. Partial surgical excision of hypertrophic papillomas was performed before the initiation of injection. Further injections at 4-week (or 2-week) intervals were performed until complete remission.
Results: Sixteen patients received a mean 8.9 injections. Complete remission was obtained in 12 patients (75%) after a mean 7.2 endoscopic treatments. Remission was stable at a mean 33.6 months' follow-up. Five of these 12 patients received 5.2 injections and remained disease-free at a mean 39.3 months' follow-up. Seven of the 12 had 1 relapse; they needed complementary treatment to become disease-free, and remained so thereafter over a mean 27.3 months' follow-up. The other 4 of the 16 patients (25%) continued to present active disease.
Conclusions: Active endoscopic treatment until complete remission led to a higher-than-expected complete remission rate on intermediate-term to long-term follow-up, with or without relapse. Transient relapse was associated with a long delay in initiating cidofovir treatment.