Background/Purpose: Despite continuous improvements in the therapeutic options for children with systemic juvenile idiopathic arthritis (sJIA), access to medications significantly differs among centres and countries. Furthermore, major concerns still exist in the management of refractory disease trajectories, seriously impacting patients’ quality of life. We aimed to capture major challenges and unmet needs in the treatment of sJIA from patient perspective.

Methods: In the context of METAPHOR project, a PReS/PRINTO initiative aimed to optimize therapeutic approaches to sJIA and macrophage activation syndrome (MAS), an international survey addressed to patients with sJIA was performed, exploring challenges and concerns regarding treatment. The survey was developed by a core team, constituted of 2 senior physicians and 2 patient representatives, and further refined in a focus group by 8 sJIA parents and 1 young adult patient. The survey was then forwarded via the sJIA Foundation to all their members.

Results: A total of 139 replies were collected, mainly from United States (64%) and Europe (19%). Almost all participants (94%) were parents or legal guardians. Patient ages were all similarly represented, with a median sJIA duration of 3-5 years. Almost half of patients experienced MAS (44%), and 23% had multiple episodes. At survey time, 38% of patients had an active disease, while 55% and 17% had an inactive disease with and without medications, respectively; 2 patients died. Nearly half patients received treatments for 1-5 years, and 23% for over 6 years. From patient perspective, poor therapy compliance at home is mainly affected by daily injections (90%) and prolonged treatment (51%). During hospitalization, missing daily activities (65%) and difficulties in venous access (47%), further negatively impact on patient compliance. Major concerns regarding steroids include mood changes (64%), weight gain (58%), and growth delay (61%). Starting a new treatment, the majority of parents were scared about the possibility of treatment failure (82%), side effects (77%) and the increased risk of infections (51%). Key treatment goals of sJIA treatment from patient perspective were the complete control of symptoms (79%), the improved quality of life (72%), and the reduction of steroid exposure (53%). Notably, 54% of patients faced difficulties in accessing necessary medications, primarily due to issues with private insurance or drug unavailability in their country

Conclusion: Treatment of sJIA still represent a challenge for patients. Addressing patient perspective, including improvement in the tolerability of home treatment and in access to medication, reduction of steroid exposure and less interference with daily life, is crucial for enhancing the quality of care for patients with sJIA.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/unveiling-major-challenges-and-unmet-needs-in-the-therapeutic-approach-to-systemic-juvenile-idiopathic-arthritis-the-patient-perspective/