Title: Airway Intervention Registry (AIR) extension - Recurrent Respiratory Papillomatosis
Funder: Department of Health and Social Care

Abstract: Background: Recurrent Respiratory Papillomatosis (RRP) is a relatively rare condition which is more common and aggressive in children than in adults requiring multiple interventions to achieve symptomatic control. A recent cross-sectional survey of ENT consultants conducted by the applicants (supported by the National Institute for Health and Care Excellence, and the Chief Medical Officers in Scotland, Wales and Northern Ireland and ENT UK) identified 918 patients suffering RRP in the UK, with 16 different types of intervention being performed in the previous 12 months. Despite the large cost of care of RRP patients, there is a lack of national guidance on the management of RRP patients in the UK. Aims: To determine the short and long-term relative safety and efficacy of RRP treatments used in children in the UK. Plan of investigation: We will develop a secure, online database into which observational data on treatments and outcomes, and quality of life questionnaire responses, for all RRP patients aged 16 years old or less, attending NHS hospitals in the UK over a 3 year period, will be entered. Participation of data to this study requires patient/parent/guardian consent. Clinical support will be provided by ENT/respiratory consultants, and only registered consultants/research staff from NHS trusts provided with a username and password will enter data into the online database. Information captured in the online database will include: general demographic information, clinical history, patient and parent/guardian questionnaires, results from clinical examination, treatment details, post-procedure outcomes. In order to determine relative long-term safety and efficacy of RRP treatments we would ask for longitudinal data to be collected for each participating patient from all subsequent respiratory related outpatient/hospital visits (i.e. any complications, number of GP/A&E/outpatient attendances or hospital admissions as a result of RRP symptoms). Potential benefits to patients: Identifying the safest and most effective treatments for RRP will influence care of RRP patients in the future, reduce complications, hospital stay, hospital visits, unexpected A&E/GP visits, and maximising the time between interventions and improving the patient's quality of life. Potential benefits to NHS: Reduction in costs (associated with reduced hospital length of stay, fewer interventions over time and fewer unexpected healthcare visits) whilst providing the best quality of care for patients. Our study will also demonstrate that a database can be established quickly at minimal cost to collect observational evidence from the NHS for rare diseases. Elements of the database platform could be re-used for future clinical databases.

Lay abstract: Background: Recurrent Respiratory Papillomatosis (RRP) causes wart-like growths in the airway which make it difficult to breathe, speak and carry out normal everyday activities. It is a rare condition but is more common and aggressive in children than in adults, affecting 4 in every 100,000 children. There is no known cure for RRP, but symptoms are checked through regular hospital visits, with multiple therapies or procedures under general anaesthetic needed to remove or shrink the growths which can grow back quickly. The problem is that nobody knows which therapies or procedures work best. Aim: To identify which RRP treatments currently used in UK NHS hospitals are the most effective and safest in the short- and long-term. We will also identify which patients respond best to specific treatments, and those who are at higher risk of experiencing a complication after treatment. Method: This observational study does not require patients to undergo any additional intervention as part of the research. We will collect information from usual patient care and quality of life questionnaire responses in a secure online database. Participation in this study requires patient/parent/guardian consent. Patient & Public Involvement: Feedback from young people and parents/carers has already been sought and has influenced the study design and proposed outputs. Future and regular involvement with patients and public is included in our grant application including review of the study website, to gain young people's perspective on recruitment, interim results, and lay summaries. Dissemination: Results will be used to update national guidance on RRP care and published in a clinical journal to inform clinicians of the most effective/safe procedures; ultimately improving patients' quality of life. We will also disseminate the results in a format which is easily available to the public in order to better inform patients of expected outcomes of the different procedures.

Research activity codes: 8.1 ORGANISATION AND DELIVERY OF SERVICES
Health categories: RESPIRATORY