Disease Control and Voice Outcomes Following Treatment With PRGN-2012 in Adults With Recurrent Respiratory Papillomatosis.

OBJECTIVE

More than 50% of adult patients with recurrent respiratory papillomatosis that receive PRGN-2012 develop a protocol-defined complete response. The relationship between papilloma disease control and change in voice handicap following PRGN-2012 treatment requires clarity as patients and providers consider treatment.

STUDY DESIGN

A post hoc study of pretreatment and post treatment Derkay and Voice Handicap Index-10 (VHI-10) data collected prospectively as predetermined exploratory outcome measures on a completed single-arm, single-cohort, phase 1/2 registration clinical trial of PRGN-2012 monotherapy. All patients (n = 38) that were enrolled and treated were included in this study. Patients underwent prospective collection of recorded clinical nasopharyngolaryngoscopy and were administered a VHI-10 questionnaire before, during, and after treatment with PRGN-2012. Fisher's exact test, Wilcoxon rank sum test, Spearman's correlation, and multiple linear regression analyses were used to associate demographic and clinical variables to clinical response (CR) to PRGN-2012 treatment. Wilcoxon rank sum and Spearman's correlation tests were performed to compare change in anatomic Derkay to reduction in clinically-indicated interventions and VHI-10 scores after PRGN-2012 treatment compared to before treatment.

RESULTS

Greater reduction in anatomic Derkay score associated with greater percent reduction in clinically-indicated interventions after PRGN-2012 treatment (r = 0.68, P = 0.0001). Greater reduction in anatomic Derkay score significantly associated with greater reduction in VHI-10 score (r = 0.81, P < 0.0001). No association (r = 0.10, P = 0.5586) was observed between the number of clinically-indicated interventions required in the 12 months prior to treatment and CR. A reduced number of total lifetime clinically-indicated interventions associated with greater CR (r = 0.46, P = 0.0037) in univariate analysis, but the significance of this relationship was lost on multi-variate analysis (P = 0.184) adjusting for multiple co-variates.

CONCLUSIONS

These data link greater papilloma disease control with reduced need for clinically indicated procedures and reduced voice handicap after PRGN-2012 treatment independent of short- or long-term measures of disease severity.