Background/Purpose:

Juvenile systemic scleroderma (jSSc) is an orphan disease with a prevalence 3 in a 1 000 000 children(1). Published data is limited regarding the course of jSSc with standardized assessment of patients. We report our data from the juvenile scleroderma inception cohort over the first 24 months after enrollment.

Methods:

The juvenile scleroderma inception cohort is a prospective multicenter registry of patients with jSSc, who fullfill the adult classification criteria, are less than 16 years old at onset, and are less then 18 years at the time of inclusion in the cohort. We evaluated the clinical characteristics of those followed at least 24 months in the registry.

Results:

Forty jSSc patients met this criteria. The majority were female (80%) with diffuse cutaneous subtype (77.5%) and 20% had overlap features. Mean disease duration at time of inclusion was 3.2 years. Mean age of onset of Raynauds was 8.2 years and the first non-Raynauds 8.7 years. Those on DMARDs increased from 73% at the time of inclusion and to 95% after 24 months. 85% of the patients were ANA positive, approximately 25% anti-Scl70 positive and 3% anticentrome positive. The mean modified skin score decreased from 14.4 to 12.9. The frequency of Raynaud´s stayed around 87.5%. The frequency of the nailfold capillary changes increased from 55% to 62%, but the frequency of active ulcerations decreased from 23% to 20%. The number of patients with FVC <80 % decreased from 42% to 39% (p=0.8). The number of patients with DLCO< 80% decreased from 54 to 44% (p=0.58). The number of patients with pulmonary hypertension assessed by ultrasound increased from 5% to 10% (p=0.396). No patient developed hypertension or renal crisis. Gastrointestinal involvement decreased from 32.5% to 22.5% (p=0.317). Number of swollen joints decreased from 23% to 17.5% (p=0.53). Total muscle weakness decreased from 5 % to 0% (p=0.224) and elevated CK deacreased from 17% to 11% (p=0.54). Several patient related outcomes improved significantly. Patient global disease activity (VAS 0-100) from 49.2 to 29.4(p=0.001), patient global disease damage (VAS 0-100) from 43.9 to 29.8 (p=0.013) and patient Raynaud activity VAS 0-100) from 26.7 to 14.2 (p=0.045) as physician global disease activity (VAS 0-100) from 48.3 to 33.2 (p=0.021).

Conclusion:

Over the 24 months observation period patient related outcomes improved significantly and fortunately most other clinical parameteres did not revealed any significant deterioration. It seems, that the current treatment can reach improvement judged by the patients and stablisation of the disease outcomes.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/after-24-months-observation-period-the-patients-related-outcomes-improve-significantly-in-the-juvenile-scleroderma-inceptions-cohorte/