What is the treatment pattern of juvenile systemic sclerosis? Results from an international cohort.

Objectives: Clinical trial data are lacking for treatment of patients with juvenile systemic sclerosis (jSSc). Three published recommendations exist for jSSc but real-world data on treatment patterns are lacking. The aim of this study was to analyse treatments used in the jSSc inception cohort (jSSci) and compare to published recommendations on the treatment of jSSc. Methods: Data was extracted for patients with 24 months follow-up visits in the jSSci up until June 2023. Medications used and their association with clinical characteristics were analysed. Logistic regression analyses were performed to compare treatments between limited and diffuse cutaneous jSSc subtypes, organ involvement and time of initiation of treatment. Multilevel mixed effects logistic regression analyses were used to evaluate the change in medication use in follow-up. Treatment patterns were compared against published recommendations. Results: 93 patients had 24 months follow-up data. 77% of patients were receiving disease-modifying treatment (DMARD) at enrolment, which increased to 91% at 24 months (p<0.001). Patients with diffuse cutaneous jSSc subtype had significantly more frequently active ulcerations, skin involvement and received any kind of treatment more often compared with limited (97% vs 91%, p=0.047). Methotrexate was used in 52% of patients at enrolment which decreased to 37% at 24 months (p=0.001). Mycophenolate mofetil use increased from 24% to 46% (p<0.001). Biological DMARDs increased from 5% to 22% (p<0.001). The treatment pattern strongly overlapped with the published paediatric guidance. Conclusion: This is the first report regarding the pattern of medication use in real life in the currently largest patient cohort of patients with jSSc. The observed pattern overlaps with the published recommendations.