title: Journeying Beyond Clinical Boundaries: Pragmatic patient-centric research into Childhood Interstitial Lung Disease (ChILD)
Creators: name: Gilbert, Carlee
Publisher: name: University of Liverpool Repository
Year: 2024

description: Abstract 
Childhood Interstitial Lung Disease (chILD) is a set of rare diseases (RDs) that encompasses an array of rare respiratory disorders that impact the lung interstitium in children and young adults (CYAs). While Interstitial Lung Disease (ILD) is more commonly recognised as affecting adults, there are rare instances where CYAs may develop ILD. Due to its rarity and chILD being a relatively newly explored area of ILD, more research is needed on the psychosocial impact of living with a child. 
This thesis had two main aims. The first was to explore the chILD research landscape to help provide a baseline of understanding of how chILD research is being conducted and to understand the needs and preferences of CYAs and their parent caregivers when participating in clinical research. The second aim was to explore the lived experiences of chILD in three different scenarios: a health event (COVID-19), an unexplored symptom of chILD (fatigue), and service provision (paediatric to adult transition services). Five studies using a mixed-methods approach were conducted: one systematic review, one quantitative study, and three qualitative studies. Due to chILD being rare in hospital clinic settings, this thesis used a non-clinical population recruitment strategy by recruiting CYAs and their parent caregivers from UK-based chILD patient organisations and chILD online communities.
Two Patient and Public Involvement (PPI) focused studies fulfilled the first aim. The first was a systematic review employing a unique approach using Clinical Trial identifiers as key search terms and applying the GRIPP2-SF framework to identify how PPI is acknowledged in clinical literature. Of 42 published articles, 9.5% (n = 4) included PPI mention, distributed across the Methods and Acknowledgment sections. While one mention provided a comprehensive PPI statement, others used a generic format. This study highlighted the continued challenges in reporting PPI representation, with potential limitations linked to authors' and publishers' non-mandatory nature of PPI reporting. The outcomes contribute to the academic discourse on paediatric respiratory health and provide practical implications for refining PPI strategies in similar clinical studies investigating PPI usage. The second study aimed to understand the perspectives of clinical research in 33 young people (aged 6 to 17) with a rare paediatric respiratory condition and their parents through a child-parent dyad survey. Results revealed that parents were more open to their children participating in clinical research compared to their children (p = .007). Preferences varied, with parents expressing less inclination for research involving untested medicines and surgery, whereas young people showed less preference for research involving untested medication, surgery, venepuncture and sharing of experiences. These novel findings suggest a potential disconnect between young people's needs and opinions and parents' preferences for clinical research. The study recommended enhanced communication of clinical research opportunities, and the role of an independent advocate for CYAs is to ensure representation of the CYA ‘voice’. 
The second aim of lived experience included three qualitative studies examining differing chILD perspectives: a global health event, a chILD symptom and a healthcare service. The first was a longitudinal study exploring COVID-19 and chILD (n = 8). Interviews in two timeframes captured experiences, coping mechanisms, and evolving perceptions, revealing themes of uncertainty and adaptation. The study contributes unique insights into the challenges individuals with rare diseases face during a global pandemic, emphasising the importance of caregiver traits and resilience in positive adaptation. The second qualitative study examined the complex phenomenon of fatigue in those with chILD. The research delved into the communication, symptoms, and impact of fatigue of 15 participants, uncovering three main themes: the experience of fatigue, its consequences on education and quality of life, and motivational strategies for managing fatigue. The findings highlight the multi-dimensional nature of fatigue in chILD, emphasising the need to discuss fatigue in clinical settings to assess its impact on quality of life. The final qualitative study investigated the experience of seven young people and parents in the UK transitioning from paediatric to adult healthcare services for chILD. Participants shared insights into the lack of transition preparation, challenges in adapting to adult services, and the role of parents as advocates. The study underscored the need for improved transition processes and offered recommendations for enhancing support and education for young people and parents entering this crucial phase.
To interconnect these studies, a Grounded Theory of chILD is presented. The theory accounts for the experience of the parent and the child and a process of adaptation to varying life events identified within the thesis. The theory aligns with Uncertainty of Illness theory (Mishel, 1988) whereby a process of appraisal (of life challenges/events), uncertainty, and the availability of supportive care needs (information, psychological and emotional, and social needs), and coping traits identified within the thesis support adaptation. Areas for further exploration and recommendations are provided to build on this chILD theory. 
This thesis highlights how CYAs and parent caregivers can contribute valuable insights into their healthcare experiences, ultimately enhancing the care provided for chILD and RD’s. Collecting their ‘voice’ of novel data and insights results in 1) the amplification of their unique voice in healthcare and policy discussions, 2) presenting patient-centred recommendations to help support the chILD research agenda, and 3) promoting a structured patient engagement framework, enabling meaningful involvement of chILD CYAs and parent caregivers in their healthcare decision-making processes.