1540MO - The patient voice: An analysis of real-world data using the recurrent respiratory papillomatosis foundation/CoRDS patient registry (RRPF/CoRDS)

Background

Recurrent Respiratory Papillomatosis (RRP) is an orphan disease caused by infection with human papillomavirus (HPV) types 6 and 11. RRP is a disabling condition with no cure and adversely affects breathing in children and adults of both sexes. Up to 8.9% of patients with RRP have pulmonary involvement, which carries a 32-fold increased lifetime risk of malignant transformation. We present the economic and psychosocial disease burden of RRP from the patient and caregiver perspective.

Methods

Disease-related questions surrounding the economic, clinical, and humanistic burden of RRP were incorporated into the RRPF/CoRDS Patient Registry. Responses entered within the patient registry from May 28, 2020, to July 31, 2023, were analyzed. The descriptive analyses of all outcomes of interest were performed across juvenile-onset (Jo-) and adult-onset (Ao)-RRP participants. Tests of comparison with binary or categorical variables were assessed via the Pearson Chi-square or Fisher’s Exact test. Tests of comparison with continuous variables were conducted via pooled t-tests.

Results

A total of 123 participant responses from the United States (US) were included in the analyses, which represented 55% (67/122) Jo-RRP, 45% (55/122) Ao-RRP, and 1 uncategorized. The greatest concern with an RRP diagnosis was permanent communication issues (35.7%), distal spread (22.4%), financial burden (15.3%), and death from RRP (15.3%). Participants also reported feeling isolated due to the inability to participate in social events due to their RRP (70%), social anxiety (72.2%), and depression (45.1%). 81% of participants traveled >20 kilometers and 38% reported spending >10% of the family income to obtain specialty care. The comparative analyses between the Jo-RRP and Ao-RRP participants are ongoing.

Conclusions

For the first time, the critical perspective of disease impact and burden of RRP from the patient and/or caregiver is reported. The study highlights the significant financial and psychosocial burden of RRP, which is preventable through HPV vaccination. Recent observational data in the US and Australia have shown that the introduction of quadrivalent HPV vaccine has led to declines in the incidence of Jo-RRP.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

The authors.

Funding

Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

Disclosure

S. Pai: Financial Interests, Personal, Advisory Board: Cue Biopharma, Scopus, Sensei, Lyell Biopharma, Merck, MSD, Recurrent Respiratory Papillomatosis; Financial Interests, Institutional, Research Funding: Merck, AstraZeneca; Financial Interests, Institutional, Research Grant: Cue Biopharma; Financial Interests, Institutional, Sponsor/Funding: National Cancer Institute, National Institute of Dental and Craniofacial Research; Non-Financial Interests, Personal, Other, Senior Editor: Cancer Research. N. Hedlund: Financial Interests, Personal, Stocks/Shares: Baxter, Takeda; Financial Interests, Institutional, Advisory Role: WL Gore, CardioDiagnostic Holdings. A. Martino: Financial Interests, Personal, Full or part-time Employment: Merck & Co., Inc; Financial Interests, Personal, Stocks/Shares: Merck & Co., Inc. N. Chohan: Financial Interests, Personal, Full or part-time Employment: Merck & Co., Inc. E. Morais: Financial Interests, Personal, Full or part-time Employment: MSD France; Financial Interests, Personal, Stocks or ownership: Merck & Co., Inc. K. McClellan: Financial Interests, Personal, Advisory Role: Inovio, Precigen. All other authors have declared no conflicts of interest.