Background/Purpose: Localized scleroderma (LS) is an autoimmune disease of the skin and underlying tissue that leads to progressive fibrosis and disability in growing children.  Pediatric LS is known to affect females more frequently than males, but differences by gender are not well characterized.  We examined the LS cohort enrolled in the Childhood Arthritis and Rheumatology Research Alliance (CARRA) registry for gender differences. 

Methods: Descriptive statistics for demographic, clinical, and patient-reported data were performed.  Statistical differences between genders were assessed using t-test or Wilcoxon rank sum test and Chi-square or Fisher exact test depending on the variable type and distribution.

Results: A total of 369 participants were included in the analysis.  The majority of participants were white (n = 333; 90%) and female (n = 279; 76%).  Median age at onset was 7.7 years old (IQR: 4.5-11.2), median age at first rheumatologist visit was 10.0 years old (IQR: 6.8-12.9), and median time to rheumatologist was 0.9 years (IQR: 0.4-2.4).  The majority of the population had linear subtype of either trunk/limb or head (n = 195; 52%).  A total of 53 (14%) patients were diagnosed with circumscribed subtype, 33 (9%) with generalized morphea, with other subtypes present in smaller proportions.  Patients with mixed subtype comprised 21% of the cohort.

Females were older at disease onset than males (median: 8.3 years old vs. 6.3 years old, p = 0.01).  Females also were older at time of first rheumatologist visit than males (median: 10.4 years vs. 8.8 years, p = 0.01), but time from onset to first rheumatologist visit was not significantly different by gender.  Males were more likely than females to have muscle atrophy (35% vs. 20%, p = 0.009) and abnormal CK levels (16% vs. 7%, p = 0.035).  Males were also more likely to experience hemifacial atrophy than females, with this trend approaching significance (15% vs. 7.5%, p = 0.056). Median global well-being score for the overall group was 1 (IQR: 0-3) on a 0-10 scale with lower scores indicating better well-being.  However, a higher proportion of females than males reported worse global well-being than the median (48.5% vs. 30.3%, p = 0.003).  When pain scale was dichotomized (0 = no pain vs. > 0 = pain reported), a trend was noted towards a higher proportion of females reporting the presence of pain (44% vs. 33%, p = 0.091).  No other gender differences for demographic, clinical, or patient-reported outcomes were demonstrated.

Conclusion: In this analysis of a large multicenter sample of LS patients, males more frequently showed evidence of muscle involvement and hemifacial atrophy despite comparable frequency of LS subtypes.  Although these clinical differences suggest males might have more severe disease features, female patients reported worse overall well-being and more frequently reported pain.  Further study is warranted to better characterize gender differences in LS clinical features and patient-reported outcomes and to examine underlying reasons for such differences.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/gender-differences-in-pediatric-localized-scleroderma-clinical-and-patient-reported-outcomes/