The importance of children and young person involvement in scoping the need for a paediatric glucocorticoid-associated patient reported outcome measure.

BACKGROUND: For many children and young people (CYP) with paediatric rheumatic conditions, glucocorticoid medications and their associated side-effects have a substantial impact on disease experience. Whilst there are physician-rated measures of glucocorticoid toxicity, no parallel patient reported measure has been developed to date for CYP with rheumatic disease. This manuscript describes a series of public patient involvement (PPI) events to inform the development of a future paediatric glucocorticoid-associated patient reported outcome measure (PROM). METHODS: One large group PPI event was advertised to CYP with experience of glucocorticoid medication use and their parents through clinicians, charities and existing PPI groups. This featured education on the team's research into glucocorticoid medication and interactive polls/structured discussion to help participants share their experiences. Further engagement was sought for PPI group work to co-develop future glucocorticoid studies, including development of a glucocorticoid associated PROM. Quantitative and qualitative feedback was collected from online questionnaires. The initiative was held virtually due to the Covid-19 pandemic. RESULTS: Nine families (n = 15) including 6 CYP joined the large group PPI event. Online pre-attendance and post-attendance questionnaires showed improvement in mean self-reported confidence [1 = not at all confident, 5 = very confident] in the following: what steroid medications are (pre = 3.9, post = 4.8), steroid side effects (pre = 3.8, post = 4.6), patient-reported outcome measures (pre = 2.0, post = 4.5), available research on steroids (pre = 2.2, post = 3.5). Five families (n = 7) were involved in a monthly PPI group who worked alongside the research team to identify priorities in glucocorticoid research, produce age-appropriate study materials, identify barriers to study participation (e.g. accessibility & convenience) and recommend appropriate modalities for dissemination. The participants found discussing shared experiences and learning about research to be the most enjoyable aspects of the initiative. CONCLUSIONS: This PPI initiative provided a valuable forum for families, including young children, to share their perspectives. Here, the authors explore the effective use of PPI in a virtual setting and provide a unique case study for the involvement of CYP in PROM development. The monthly PPI group also identified a need for the development of a new PROM related to glucocorticoid medication use and provided unique insights into how such a study could be structured.

What can families and researchers learn from each other about glucocorticoid medication - Lessons from a virtual patient public involvement event?

Introduction: Patients and public involvement (PPI) events allow families to use their own experience to contribute meaningfully to health research, as well as learn more about research processes and findings. Glucocorticoid medications, or steroids, and their side effects remain a central part of the patient experience across a range of paediatric rheumatic diseases. The research team hosted a virtual PPI event around the use of glucocorticoid medication in children and young people (CYP). Objectives: The aims of the virtual event were to share experiences of glucocorticoid treatment, provide education about the research team's work, and to identify future key CYP/parents that would continue to be involved in a smaller that PPI group to co-develop glucocorticoid associated clinical studies. Methods: The event was advertised to CYP with experience of glucocorticoid medication use and their parents through clinicians (n=4), charities (n=9) and patient groups (n=2), including their associated social media channels. The event was held virtually on Zoom due to Covid-19 restrictions. During the session, the research team shared findings from four on-going studies involving glucocorticoid medications. Interactive polls and structured discussion were used to help participants share their experiences of steroids. Pre- and post-attendance online questionnaires were used to collect quantitative and qualitative feedback about the event. Results: 11 families with a range of conditions necessitating glucocorticoid medication (JIA, Behcet's, Lupus, Nephrotic Syndrome, Allergic Bronchopulmonary Aspergillosis) participated from across the UK. The ages of CYP ranged from 2-26. Participants had used glucocorticoid medication through a variety of administration routes (oral, intravenous, intra-articular, topical, inhaled). The duration of use varied from <1 year to >10 years. Online pre-attendance and post-attendance questionnaires showed an improvement in mean self-reported confidence [1 = not at all confident, 5 = very confident] in the following: what steroid medications are (pre = 3.9, post = 4.8), steroid side effects (pre = 3.8, post = 4.6), patient-reported outcome measures (pre = 2.0, post = 4.5), available research on steroids (pre = 2.2, post = 3.5). Most participants (86%) reported that they were likely or very likely to attend future PPI events. Diversity was seen in the experiences and views of CYP in relation to glucocorticoid medication. Many reported several benefits including rapid improvement in their disease, reduced pain and improved mobility. However, there were a greater number of negative effects reported, impacting upon health-related quality of life, including effects on body image, school, mood and relationships with others. A number of participants reported the most enjoyable part of the event was hearing other participants' experiences. This suggests that PPI events offer avenues for insight and therapeutic benefit through sharing common experiences. Conclusion: PPI initiatives provide a valuable forum for both researchers and families to share their perspectives. They can be a useful educational tool to disseminate research findings that are relevant to patients and improve health research knowledge. Families found the experience to be beneficial and enjoyed the opportunity to share experiences on the effect of glucocorticoid medication on health-related quality of life. Five families opted into further PPI group work to engage in the co-development of future glucocorticoid studies.