Background/Purpose: Localized scleroderma (LS) is a chronic inflammatory and fibrosing skin disease. We present baseline data on the juvenile LS (jLS) cohort from the Childhood Arthritis and Rheumatology Research Alliance (CARRA) registry, a multicenter observational pediatric rheumatic disease registry.

Methods:  Descriptive statistics were used for demographic, clinical and laboratory features. Data analysis included the two-sample t-test, chi-square test, Fisher’s exact test, logistic regression, and analysis of variance as appropriate.

Results: Of 386 children in the database, 76% were female and 80% were Caucasian. Mean age at onset was 8.2 yr (± 4.2). Mean age at first pediatric rheumatology (PRH) evaluation was 9.6 yr (± 4.1), yet 18% had ≥ 2 yr delay from onset to first PRH visit. Linear scleroderma (LiS) was the most common subtype (54%), followed by circumscribed morphea (CM) (16%), generalized morphea (GM) (9%), eosinophilic fasciitis (1%), and pansclerotic morphea (1%). 19% of children had mixed subtype, and LiS-CM was the most frequent combination (61%). Among LiS patients with face-scalp localization (34%), neurologic and ocular diseases were reported in 11% and 4%, respectively.

ANA positivity was found in 48% tested and was not associated with disease subtype or age at onset. ANA positivity, however, was associated with features of non-cutaneous disease damage, specifically joint contracture (p=0.04), muscle atrophy (p=0.01), and extremity shortening (p<0.01). Other laboratory findings included elevated aldolase was associated with joint contracture (p=0.01), and elevated CK was associated with muscle atrophy (p=0.04) and extremity shortening (p=0.02).

Children with any functional limitation (baseline worst ever ACR functional class II, III, and IV) (30%) had earlier first PRH visit (mean 0.98 yr ± 1.52) compared to those without limitation (class I) (mean 1.6yr ± 2.2, p=0.01). Poorer function also correlated with presence of arthritis, muscle atrophy, joint contracture, and extremity shortening (all p<0.001). Medications were similar to the 259 subjects prior analyzed (CARRA 2014 abstract) with subcutaneous or oral methotrexate (97%) and pulse or long term daily corticosteroids (68%) being the most commonly used, followed by mycophenolate mofetil (16%). The only significant difference in treatment according to subtype was the use of topical therapy instead of systemic in superficial morphea.

Conclusion: In the CARRA registry, jLS occurred more frequently in females and Caucasians. LiS was the most common subtype. Almost 1/5 of children had a ≥ 2 yr delay from symptom onset to PRH referral. Children without limitations are referred later, highlighting the insidious onset and need for educating referring providers. There is significant morbidity, with 30% of children reporting functional limitations. Poorer function correlated with presence of arthritis, muscle atrophy, joint contracture, and limb shortening. An elevated CK or aldolase was associated with presence of muscle atrophy, joint contracture, and/or limb shortening, suggesting they may be possible predictors of muscle involvement.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/description-of-the-juvenile-localized-scleroderma-subgroup-of-the-childhood-arthritis-and-rheumatology-research-alliance-carra-registry/