6080 Background: Recurrent respiratory papillomatosis (RRP) is caused by human papillomavirus (HPV) types 6 & 11. RRP proliferates in the respiratory tract impacting breathing, swallowing, and voice and carries a 1-4% risk of malignant transformation.There is no curative therapy for RRP. Given the tolerized host immune response against HPV, the safety and efficacy of pembrolizumab (pembro) as an alternative treatment for this patient population was evaluated in a phase II clinical trial. Patient reported outcomes (PROs) were assessed during the trial to capture the patient perspective of pembro as an alternative to surgery or in office procedures, both standard of care (SOC). Methods: RRP patients who had previously undergone >3 procedures in any year, or with known tracheal or pulmonary involvement prior to study entry were treated with pembro 200mg every 3 weeks. The primary endpoint of the trial was best ‘overall response rate’ (ORR) measured by an endoscopic-based disease burden score (lower score reflects better ‘response’) and/or RECIST 1.1, secondary endpoint included PROs. Twenty-one patients were required to assess the primary endpoint. Most of the QoL surveys used Likert scale to assess PROs (‘never, sometimes, often, most of the time, always’). The percentage reporting ’never’ having an issue with symptom or activity at baseline, 6 months, and at time of ORR (nadir disease burden score) is reported here. Results: Twenty-one patients were accrued. Median age (range) was 45 (19-68), 57% (12/21) were male and 67% (14/21) were white. Questionnaire completion rates were 100% at baseline, 90% at 6 months, and 85% at ORR. Improvement in: social interactions (less difficulty with: physical intimacy [38%,56%,65% reporting ‘never’ at baseline, 6 months, and at ORR respectively]), discussing disease diagnosis [19%,21%,39%]); personal feelings (less depression [14%,32%,33%], less anxiety [5%,16%,22%], less embarrassment [19%,37%,50%]), and work-related absences (less frequently fabricating reasons for work absence due to disease-related treatment [57%,78%,56%] and less utilization of family vacation or FMLA for disease treatment [29%,53%,56%]) were reported. At ORR, 72% (13/18) patients reported that IV infusion was not emotionally burdensome and 78% (14/18) reported it as the preferred treatment relative to their perceived experience with SOC surgery or in office procedures. Conclusions: PRO results show consistent benefit in key aspects of the patient experience with pembro over procedure based SOC further supporting its overall clinical benefit in patients with HPV-associated RRP. Clinical trial information: NCT02632344.
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