The Development of Paediatric Bronchiectasis Quality of Life Questionnaires for Four Demographic Groups: Children 8-12years, Young People 13 - 17 Years and Parent Proxy Versions for Ages 2-5years and 6-12years

To develop and refine a disease-specific quality of life (QoL) questionnaire for children with bronchiectasis, informed by qualitative interviews with affected children, young people, and their parents.

The paediatric bronchiectasis- specific quality of life (PQOL-B) questionnaire will be developed across four demographics:

• Children aged 8-12 years
• Young people aged 13 -17 years
• Parent-proxy for 2-5years
• Parent-proxy for 6-12years

The development of the questionnaires will be carried out by conducting two different stages of interviews over a 12- month time period:

• Concept Elicitation - Semi structured interviews - will be to talk about what life is like living with bronchiectasis or living with a child who has bronchiectasis.
• Cognitive testing interviews - Once we create a first draft of the PQOL-B questionnaire, we will ask children, young people, and parents to try it out. In these interviews, we will ask if the questions make sense, are easy to understand, and feel relevant. This helps us refine and improve the questionnaire.

Recruitment and Interview Participants will be recruited from existing bronchiectasis database at the Birmingham Children's Hospital. Clinicians involved in routine care will screen eligible patients using pre-defined criteria. Eligible children, alongside their parents/caregiver and eligible parents will be approached during scheduled outpatient clinical visits or inpatient settings or via telephone/email.

Inclusion criteria

for recruitment valid for both interviews:

• Patient, child and young persons, aged 8 -17 years with bronchiectasis diagnosed on CT scan
• Parent of child aged 2-12 years with bronchiectasis diagnosed on CT scan
• Written informed consent/assent available

Exclusion criteria

for recruitment valid for both interviews:

• Child under 8 years with bronchiectasis for patient direct participation
• No consent/assent received
• Confirmed Cystic Fibrosis or Primary Ciliary Dyskinesia diagnosis

Sample Size

Stage 1: Concept Elicitation - Semi structured interviews: We aim to recruit 24 Children and young people (CYP)- 12 from each age group, 8-12 years and 13-17years and 22 Parents - proxy version for children aged 2-5 - 10 interviews and parent proxy version for children aged 6-12years - 12 interviews.

Stage 2: Cognitive Testing Interviews: We aim to recruit 16 CYP- 8 from each demographic group, 8-12 years and 13-17years and 16 Parents - 8 for each parent proxy version - for children aged 2-5 and for children aged 6-12years.

Ideally new participants will be recruited for the second phase of the development of PQOL-B's where possible. But due to the rarity of the disease this may not always be possible to achieve. So, there may be some participants that will participate in both interview stages.

Consenting for interviews Potential participants will be provided with an age-appropriate participant information sheet and given adequate time to consider participation. Informed consent will be obtained from parents/guardians, and assent from children where appropriate - those 15years and younger.

Stage 1: Semi structured Interviews The research team have steered and completed an e-Delphi study with bronchiectasis experts from the paediatric national network in the UK (PBNET UK) which has led to the development of a conceptual framework, which will guide open-ended interviews with children, adolescents and their parents.

A schedule of open-ended questions has been developed to support data collection, with reference to existing literature and shaped by the research objectives.

Discussions will centre on participants' life experiences living with bronchiectasis or having a child diagnosed with the disease, the impact it has on daily living activities. (copy of example prompt questions are within the appendix of this REC form) Individual, semi-structured open-ended interviews will be conducted where possible in person, or by a virtual online meeting (via Microsoft teams' platform) in accordance with participant preference, by the research team who have extensive training and experience in conducting qualitative interviews as well as extensive experience of working with children and their families living with bronchiectasis.

Participation of the child/adolescent and their parent/caregiver is independent. A child or adolescent may take part in the interview even if their parent/caregiver chooses not to participate, and similarly, a parent/caregiver may participate without their child being a participant. If preferred, a child or adolescent may have their parent/caregiver present during the interview, even when the parent/caregiver is not enrolled as a study participant.

Informed consent and assent where required for interviews will be obtained from all participants prior to data collection.

Interviews will be conducted by the research team and last approximately 45- 60 minutes, depending on how much the participant has to say.

Whether the interview is conducted in person or via video-call using Microsoft Teams, in accordance with participant preference, both ways will be recorded using the Microsoft Team's platform.

The recordings will be transcribed verbatim by the research team. The video recording will be deleted once transcripts have been checked for accuracy and analysed. The transcripts which will be pseudonymised will be stored safely and securely within the Birmingham Children's Hospital.

The data will be accessible to the research team only, and we will only be sharing the anonymized transcripts for content analysis which includes an external collaborator as apart of the multi-disciplinary team, and this is for their expert academic input. Content analysis will be facilitated using NVivo software and will identify key themes, symptoms and impacts.

Ensuring no new information is emerging, and therefore saturation has been achieved.

Agreement on question selection and wording will be agreed during multi-disciplinary team discussions; these discussions will also include the results from a previously completed e-Delphi study, creating prototype of questionnaires ready for cognitive testing.

Stage 2: Cognitive testing Interview It is necessary to pre-test the prototype on the target population. Data will be collected using cognitive testing via an interview process. Interviews will be facilitated by the research team, trained in qualitative research methods and with experience of working with children and families living with paediatric bronchiectasis. These Interviews will last approximately 45 minutes, depending on how much the participant has to say.

Participation of the child/adolescent and their parent/caregiver is independent. A child or adolescent may take part in the interview even if their parent/caregiver chooses not to participate, and similarly, a parent/caregiver may participate without their child. If preferred, a child or adolescent may have their parent/caregiver present during the interview, even when the parent/caregiver is not enrolled as a study participant.

Informed consent and assent where applicable for interviews will be obtained from all participants prior to data collection, The method of the cognitive testing interview is as follows; Participants should complete the prototype PQOL-B using the instructions provided. Children must have the prototypes read to them.

After they have completed each section, they must complete a cognitive debriefing interview (example of the debriefing questions are included in the appendix of REC application). The questions will be asked after each section and will seek to examine what the participants thought of the questions and why they choose their rating of each item. The interviews usually last up to 45 minutes depending on how much the participants have to say.

Participants will be asked whether there were any words they did not understand or if any important content or impacts of bronchiectasis were missed.

The interviewer will record the interview via Microsoft Team's platform. The interview can be virtual or face to face interview, as per the patients/care givers preference. Any suggestion for change will be discussed with the research team. The recordings will be transcribed verbatim by the research team. The video recording will be deleted once transcripts have been checked for accuracy and analysed. The transcripts which will be pseudonymised will be stored safely and securely within the Birmingham Children's Hospital.

The data will be further anonymised and will be accessible to the research team only, and an external collaborator for their expert academic input.

The results of the interviews will be used to form a translated final version of PQOL-B, ready for formal validation.

Inclusion criteria

• Patient, child and young persons, aged 8 -17 years with bronchiectasis diagnosed on CT scan

• Parent of child aged 2-12 years with bronchiectasis diagnosed on CT scan
• Written informed consent/assent available

Exclusion criteria

• Child under 8 years with bronchiectasis for patient direct participation

• No consent/assent received
• Confirmed Cystic Fibrosis or Primary Ciliary Dyskinesia diagnosis