ESPE Abstracts

Background: There is a clear need for improved patient-centric approaches in the treatment of chronic conditions, including paediatric growth hormone deficiency (GHD). Greater understanding of the patient’s treatment journey has the potential to inform clinical decisions and to improve clinical- and patient-reported outcomes. Connected Health (CH) combines state-of-the-art technologies, tools, methodologies and analytics to create new patient-centric health management models.¹ Emerging CH solutions and patient support programmes that address different challenges during treatment for GHD are increasingly transforming healthcare.²

Aim: To identify paediatric GHD patients’ challenges on the treatment journey that could be addressed with CH solutions.

Methods: Between 2016 and 2021, a series of qualitative studies (interviews and focus groups) targeting paediatric GHD patients and caregivers were used to collect patients’ perspectives. The collected data was analysed using grounded theory,³ and the insights were then condensed for user journey mapping. User journeys visualise the interactions between individuals and a product over time and across different channels.⁴

Results: A patient-centric paediatric GHD patient journey emerged, comprising six main phases: awareness, diagnosis, treatment planning, treatment initiation, treatment maintenance and transition. For each phase, relevant concerns and emotions faced by patients with paediatric growth disorders and their families were identified and mapped to the patient journey. These included: 1) pre-diagnosis concerns and uncertainty, 2) difficulties initiating contact with healthcare professionals due to insufficient communicative support, 3) increasing confusion and concern about the future, 4) concerns about whether self-management is being performed correctly, 5) the need to deal with inconvenient and hurtful routines, 6) uncertainty about the impact and role of adherence, 7) long-term therapy fatigue and 8) difficulties of change to self-administration during adolescence. Future opportunities using smart technologies may support patients in overcoming these challenges, which may improve treatment satisfaction and outcomes.

Conclusion: This qualitative analysis provides a detailed description of the patient journey from the perspective of patients receiving GH therapy for GHD that identifies the emotional sensitivities and perceived risks. This will help inform future development of effective CH interventions that improve both clinical- and patient-reported outcomes.

References:

1. Caulfield BM & Donnelly SC. QJM 2013;106(8):703-707;

2. Dimitri P, et al. J Med Internet Res 2021;23(5):e27446;

3. Corbin J & Strauss A. 2007. Basics of Qualitative Research: Techniques and Procedures for Developing Grounded Theory. 3rd ed. Sage Publications, Inc.;

4. Trebble TM, et al. BMJ 2010;341:c4078.