ESPE Abstracts

Background: The diagnosis, treatment, and management of growth disorders can have a substantial burden on patients and caregivers. Research was conducted with patient experts to understand challenges experienced by patients with growth disorders and their caregivers, and identify opportunities to improve care.

Methods: A mixed-methods approach was used to obtain insights from patient experts between October 2022-January 2023, via multiple methods of engagement: a pre-meeting survey to obtain qualitative and quantitative feedback across key themes; a 3.5-hour virtual global roundtable, chaired by ICOSEP, including patient advocates from eight countries (France, Italy, Brazil, UK, Netherlands, Canada, US, and Argentina), alongside a pediatric endocrinologist from Turkey; and a patient advocate interview from Japan.

Results: Patient experts identified three challenge areas negatively impacting growth disorder care: delays to diagnosis, low adherence to treatment, and a lack of shared decision-making (SDM) with healthcare professionals (HCPs). Most [80% (n=8)] of patient experts were only moderately satisfied with diagnostic processes in their country. Patient experts highlighted that delays to diagnosis are largely attributed to a lack of awareness and limited understanding of the medical consequences associated with growth disorders beyond height, among the public and HCPs. Long wait times for specialist consultations, costly diagnosis processes, and inequities in care access, can also contribute to delays. Patient experts stated that education of parents, schools, and HCPs is needed to improve growth disorder diagnosis. Most [90% (n=9)] of patient experts reported low or moderate satisfaction with treatment support in their country. The burden of daily injections, emotional and practical challenges associated with treatment, and lack of robust treatment support, particularly for adolescent patients, may contribute to adherence challenges. Patient experts highlighted a need for standardized information on treatment administration, alongside support programs and materials that encourage adherence throughout the patient journey. SDM in growth disorders may improve treatment adherence. However, 60% (n=6) of patient experts were unsure of its use in practice. Systemic barriers and varying familiarity with SDM contribute to the lack of widespread adoption. Patient experts stated that HCP and patient education, combined with patient empowerment, are required to facilitate uptake of SDM.

Conclusion: Patients with growth disorders and their caregivers face many challenges on the path to diagnosis and throughout treatment, impacted by low treatment adherence and a lack of widespread SDM. There is an urgent need for solutions to overcome these challenges and improve the care experience for those affected by growth disorders.