Background: Chronic graft-versus-host disease (cGvHD) is a major complication in pediatric hematopoietic stem cell transplantation (HSCT) in China, remaining a leading cause of morbidity and mortality among pediatric patients' post-transplantation, with limited treatment options specifically approved for pediatric and adolescents. The research aimed to characterize the patient journey of pediatric and adolescents with cGvHD, evaluate the current treatment landscape, and assess clinical trial experiences and awareness among pediatric cGvHD patients and their caregivers.

Methods: This mixed-methods study (China, July 2024-Feb 2025) investigated cGvHD pediatric patient (aged 3-17) and caregiver experiences. It used two focus group discussions (5 caregivers each) to generate initial qualitative insights, which subsequently informed the development of the quantitative survey instruments. Following this, quantitative surveys (30 respondents: 25 caregivers, 5 adolescent patients, aged 12-17) to quantify experiences, and in-depth interviews (5 survey participants: 4 caregivers, 1 adolescent patient, aged 12-17) for deeper qualitative understanding. Participants, mainland China residents diagnosed 3-24 months prior, were ZS recruited. Data was analyzed using descriptive statistics and thematic analysis.

Results: The quantitative survey revealed that disease awareness is relatively low, with 22/30 respondents indicating limited understanding of cGvHD. However, patients and caregivers showed a strong willingness to engage with healthcare professionals (HCPs) and follow recommended diagnostic and treatment plans. All 30 respondents accepted all prescribed diagnostic tests, reflecting high trust in HCPs and a commitment to managing the disease. Treatment experience, safety, and efficacy emerged as top priorities for patients and caregivers. Survey respondents reported generally high satisfaction with current treatments, particularly glucocorticoids (26/30) and calcineurin inhibitors (23/30).

Qualitative data revealed frustration with prolonged treatment durations (8/10), financial burdens (7/10), and concerns about long-term safety, including organ function and reproductive health (6/10). Additionally, half of the respondents highlighted the need for treatments with faster onset of action and more sustained relief. Caregivers also showed high vigilance in monitoring their children's health, with all indicating adherence to follow-up schedules. The frequency of follow-ups gradually decreased from weekly within the first month post-transplantation to every 3-4 weeks after six months.

The research also highlights challenges in cGvHD management and trial participation. From in-depth interviews, patients and caregivers have diverse perceptions of clinical trials, with some viewing them as experimental and potentially risky due to uncertainties about safety and efficacy, while others recognize their essential role in medical advancement and potential access to novel treatments. Key motivators for participation included expected efficacy improvements (18/30) and reduced side effects (18/30). Willingness to participate increased when respondents received detailed information on study background, prior results, and safety measures.

Conclusions: The findings suggest that enhancing disease education and ensuring transparent communication regarding the benefits and risks of clinical trials, alongside addressing practical barriers, can improve disease management and clinical trial participation rates. By developing targeted educational materials, improving information accessibility, and fostering collaborations with healthcare professionals and patient communities, the industry can cultivate greater trust in clinical trial processes and enhance enrollment in pediatric cGvHD trials in China. The comprehensive characterization of the pediatric cGvHD patient journey in China provides insights into disease awareness, treatment experiences, and unmet needs, offering a foundation for developing more effective, patient-centered approaches to both standard care and innovative therapeutic interventions. These actions are important for addressing identified unmet needs and demonstrating a commitment to patient-centered research practices.

This content is only available as a PDF.
2025
Sign in via your Institution