OP0271 JUVENILE LOCALIZED SCLERODERMA: A LARGE RETROSPECTIVE COHORT STUDY FROM A TERTIARY CARE CENTER
Authors: Egeli B. , Dallas J. , Anderson E. , Min M. , Mazori D. , Gellis S. , Son M. B. , Sundel R. , Vleugels R. , Dedeoglu F. 
Published: May 2021

Background:

Juvenile Localized Scleroderma (jLS) is a rare pediatric inflammatory disease of skin and underlying tissues that may cause significant functional impairment and disfigurement. Management approaches vary and an optimum treatment regimen is lacking. In 2012, a group of jLS researchers of Childhood Arthritis and Rheumatology Research Alliance (CARRA) proposed consensus treatment plans (CTPs), aimed to streamline the approach to care for jLS patients.

Objectives:

This study aimed to evaluate a large jLS patient cohort seen over a 21-year period in a single tertiary care pediatric hospital in the USA, in order to examine treatments utilized and determine parameters for systemic therapy initiation.

Methods:

This retrospective cohort study included jLS patients with disease onset in childhood (≤18-years of age) who were seen in rheumatology, dermatology, or combined rheumatology-dermatology clinics from 1999-2020, with ≤ 3 years of follow-up. Data on demographics, disease characteristics, therapies prescribed, and treatment trends were analyzed.

Results:

Of the 270 jLS patients identified, 101 fulfilled the inclusion criteria. The primary reason for exclusion was <3 years of follow-up. Selected demographic data and disease characteristics of patients are shown in Table 1. There were no statistically significant differences in most patient and disease characteristics between patients who received systemic treatment and those who did not. There were no significant differences in baseline laboratory values. The group treated with systemic therapy did have higher rates of extracutaneous involvement and had a higher proportion of patients with a generalized morphea phenotype.

Table 1.

Demographic and Disease Characteristics

All Patients
(n=101)On Systemic Therapy
(n=63)No Systemic Therapy
(n=38)p valueAge-onset (Y), median (IQR)7.5 (6.4)9 (6)7 (4)NSAge-diagnosis(Y), median (IQR)9 (7.9)10 (6.7)9 (3.7)NSDiagnostic delay(M), median (IQR)10 (2.9)12 (13)7.5 (17)NSFollow-up (M), median (IQR)74 (69.3)65 (44.5)78 (47.7)NSSubtype Linear563917NS Face29209NS Circumscribed23419<0.0001 Mixed220NS Generalized201820.004Extracutaneous Involvement191810.001Clinic type Dermatology29227<0.0001 Rheumatology211920.003 Combined51429<0.0001

The majority of patients who were on systemic immunomodulatory therapy were treated with methotrexate (59/63, 93.6 %) and/or systemic corticosteroids (21/63, 33 %). 5 patients were treated with hydroxychloroquine, 2 of which were also on methotrexate. 6 patients on methotrexate were either switched to or had mycophenolate mofetil added as concomitant therapy. The most common adverse effects observed in methotrexate-treated patients were gastrointestinal complaints (12/61, 19.7%) and fatigue (7/61, 11.5%). The median treatment duration was 50 months (IQR: 33.5). Patients were more likely to receive systemic therapy if they were followed in rheumatology or combined rheumatology-dermatology clinics as compared to dermatology clinics. Finally, 78% of patients with jLS received systemic treatment after 2013 (a year after publication of the CARRA jLS CTP) as compared to 55% of patients prior to 2013 (p < 0.05).

Conclusion:

This jLS cohort is one of the largest reported from a single center and reflects an increase in the use of systemic therapy since publication of CARRA CTPs in 2012. Further studies on long-term treatment outcomes and therapeutic approaches utilized when first-line treatment failures occur are warranted.

References:

[1]Li SC, Torok KS, Pope E, et al. Development of consensus treatment plans for juvenile localized scleroderma: a roadmap toward comparative effectiveness studies in juvenile localized scleroderma. Arthritis Care Res (Hoboken). 2012;64(8):1175-1185.

Disclosure of Interests:

Bugra Egeli: None declared, Johnathan Dallas: None declared, Edwin Anderson: None declared, Michelle Min: None declared, Daniel Mazori: None declared, Stephen Gellis: None declared, Mary Beth Son: None declared, Robert Sundel: None declared, Ruth Vleugels: None declared, Fatma Dedeoglu Consultant of: Novartis