Exploring pediatric onset still's disease patient journey and parental perceptions in Türkiye through a survey

Abstract

Objective This survey-based study aimed to explore the journey to the diagnosis and management of children with pediatric onset Still's disease (PoSD) in Turkiye. This sought to understand parents' perceptions, feelings, and expectations, enabling the identification of unmet needs and fostering improvements in PoSD care. Methods A 42-question survey was administered to patients (< 21 years) with PoSD or their parents (the primary caregiver of each child younger than middle school age) by pediatric rheumatologists, during their clinic visits between January and May 2024. Data were descriptively analyzed. Results The study included 39 children (mean age: 9.92 +/- 4.93 years; 66.7% female). Ten children and 29 parents completed the survey. Persistent fever (84.6%), swollen joints and rash (64.1% each) were the most common initial manifestations. No patient received the diagnosis of PoSD at their initial visit: two-thirds were referred to other physicians and one-third were misdiagnosed, primarily with other inflammatory conditions (15.4%) and infections (12.8%). The median diagnostic delay was 1 (1-4) months. At the time of the survey, 21 patients (53.8%) had been receiving biologics (monthly (n = 10), bimonthly or weekly (n = 5, each) injections); 19 patients were compliant with this treatment. Seven patients (17.9%) had used biologics in past. PoSD substantially affected psychological well-being (33.3%) and daily tasks (13%). Fifteen of 26 school-age children missed school for a mean of 17.7 +/- 15.9 days/year due to PoSD. Most parents (90%) had not heard of PoSD at the time of diagnosis. Overall, 28% of parents hardly accepted the diagnosis. PoSD worsening in the future was their main concern (68.9%). The parents'/patients' primary expectation was to achieve a cure (82.1%). The main sources of information for parents were the physicians treating their children (96.6%), internet (72.4%) and other patient relatives (20.7%). Conclusion This survey of the PoSD patient journey in Turkiye identified several areas of improvement for better disease management. It underscores the need for enhanced continuous medical education for physicians and parental awareness of PoSD, higher access to credible information resources, and greater parental engagement in activities organized by pediatric rheumatology associations. Collecting quantitative and qualitative data on various aspects of PoSD could further refine approaches to optimize patient care.