Juvenile Systemic Sclerosis Treatment Practices in an International Cohort and Comparison to Recent SHARE Consensus Guidelines

Background Juvenile systemic scleroderma (jSSc) is an orphan disease with a prevalence of 3 in 1,000,000 children. Currently no medications are licensed for the treatment of jSSc. Due to its rarity, only recently have the first management and treatment guidelines been published, the jSSc SHARE (Single Hub and Access point for paediatric Rheumatology in Europe) recommendations, reflecting consensus opinion upon pediatric rheumatologists (1). Objectives To better understand treatment practices internationally for jSSc, both at baseline and over 24 months observation period and to compare if real world therapies are congruent with the recent SHARE recommendations. Methods The juvenile systemic sclerosis inceptions cohort (jSScC) is a multinational cohort that prospectively collects clinical data, including medications at baseline and subsequent visits. The jSScC enrollment criteria include age of onset of the first non-Raynaud symptom younger than 16 years and age younger than 18 years at cohort entrance. The frequency of medications (general category and specific medication) was calculated across the cohort at timepoint 0 (enrollment), 12 months and 24 months. Results We extracted data from the jSScC of patients who were followed for 12 or 24 months. 109 patients were followed at time point 0 (T0) and 12 months (T12), and data was available for 77 of them up at 24 months (T24). The mean age of the patients was 13.2 years at the timepoint 0. 77% were female and 75% had diffuse subtype. Disease duration at baseline visit was 3.1 years. The medications the patients were on recorded by the physician were captured at T0, T12 and T24 listed in Table 1. Table 1. MEDICATIONS Time point 0N=109 T12 monthsN=109 T24 months N=77 Any Medication 92% (100) 97% (106) 97% (75) Vascular medications Endothelial receptor antagonist 16% (17) 24% (26) 21% (16) PDE-5-Blocker 5% (5) 8% (9) 9% (7) Immunomodulators Corticosteroids 52% (57) 44% (48) 44% (21) All csDMARDs: 81% (88) 93% (101) 92% (71) csDMARDs monotherapy 61% (67) 66% (72) 60% (46) csDMARDs combination therapy 17% (18) 15% (16) 14% (11) Methotrexate 51% (56) 50% (55) 39% (30) Mycophenolate Mofetil 26% (28) 44% (48) 47% (36) Hydroxychloriquine 11% (12) 15% (16) 21% (16) Cyclophosphamide 12% (13) 2% (2) 1% (1) Azathioprine 2% (2) 2% (2) 3% (2) All bDMARDs: 5% (5) 14% (15) 18% (14) bDMARDs monotherapy 2%(2) 2%(2) 1% (1) bDMARDs combined with csDMARDs 3% (3) 12% (13) 17% (13) Tocilizumab 2% (2) 10% (11) 14% (11) Rituximab 2% (2) 4% (4) 4% (3) Adalimumab 1% (1) 0% (0) 0% (0) Autologous Stem cell transplantation 0% (0) 1% (1) 0% (0) csDMARDs: Conventional synthetic disease-modifying antirheumatic drugs b DMARDs: Biological disease-modifying antirheumatic drugs Conclusion At baseline half of the patients were on corticosteroids. This is more frequent than typical adult SSc practice but coincides with jSSc SHARE treatment recommendations (#1). After 12 months observation in the cohort over 90% of patients received a DMARD therapy. Methotrexate and mycophenolate mofetil were the most commonly prescribed DMARDs, which also reflects the SHARE treatment recommendations (#2, #3). At 12 months the use of glucocorticoid decreased and the use of bDMARDs increased. In general, biological DMARDs are typically considered in severe or refractory (SHARE recommendation #7), reflecting the lower percentage compared to csDMARDs. Autologous stem cell transplantation was observed in one patient at 12 months, reflecting an option in jSSc with progressive and refractory disease (SHARE recommendation #8). Endothelial receptor antagonists, such as bosentan, were used over time in approximately 20% of the patients, reflecting SHARE recommendation #6 for pulmonary hypertension and/or digital tip ulcers. This is the first evaluation looking at clinical medication practice pattern in jSSc, and its comparison to recently published consensus guidelines. References [1]Foeldvari I, Culpo R, Sperotto F et al. Consensus-based recommendations for the management of juvenile systemic sclerosis. Rheumatology (Oxford). 2021;60(4):1651-8. Disclosure of Interests None declared