While there are significant differences in how patients with juvenile localized scleroderma (JLS) are assessed and screened in the United Kingdom, treatment approaches are consistent, a study has found.
The study, "Multi-centre national audit of juvenile localised scleroderma: describing current UK practice in disease assessment and management," was published in the journal Pediatric Rheumatology.
JLS is a disease characterized by long-term inflammation of the skin and soft tissues, leading to their fibrosis (hardening) and eventually atrophy (breakdown). Approximately 22% of JLS patients also have extracutaneous (outside the skin) symptoms including neurological, muscle, and eye complications.
The best treatment for JLS is currently not known, because there is a lack of published research studies in this field. The recommended first-line treatment for patients with active JLS is methotrexate (an immune system suppressant agent). Mycophenolate mofetil, also an immune system suppressant, is often used as well.
The Paediatric Rheumatology European Society (PRES) working party for scleroderma has developed recommendations for the diagnosis, assessment, and management of JLS. The goal of these recommendations is to help provide guidelines for a disease area with limited research and clinical data.
Researchers in the U.K. set out to conduct a national audit to determine the current practice in the country regarding the assessment, management, and treatment of JLS, and how they align with the PRES scleroderma working party recommendations.
They collected retrospective data from 149 JLS patients (median age of 12.5 years at the time of audit) across 11 U.K. centers.
Results indicated that the assessment and screening measures recommended by the PRES scleroderma working party were not widely implemented in the country.
PRES recommends the use of two assessment tools: the physician global assessment of activity (PGA-A), which helps assess overall disease activity, and the localized scleroderma cutaneous assessment tool (LoSCAT), which helps assess local disease activity.
Results showed that only 54.4% of patients underwent the PGA-A test, and 37.6% the LoSCAT.
Researchers also investigated how patients are screened for extracutaneous manifestations. The PRES working party recommends that JLS patients with head involvement have regular screening for uveitis (inflammation of a layer of the eye), and a baseline brain magnetic resonance imaging (MRI) scan.
However, results showed that only 38.5% of the patients were ever screened for uveitis, and 71.2% had a brain MRI.
Finally, the team also investigated how patients with JLS are treated in the U.K.
The data showed that patients were treated with disease-modifying anti-rheumatic drugs (DMARDs) or biologics in 96% of cases.
In total, 95.5% of patients were treated with methotrexate as a first-line therapy, while 89.5% were treated with mycophenolate mofetil as a second-line treatment, in line with PRES scleroderma recommendations.
Researchers also found that 82.6% of patients received systemic corticosteroids (anti-inflammatory drugs), and 34.2% of patients required two or more DMARDs/biologics, which highlights the significant treatment burden that exists in these patients.
"There is wide variation in assessment and screening of patients with JLS, but a consistent approach to systemic treatment within U.K. pediatric rheumatology," the researchers concluded.
They emphasized that increased awareness of PRES recommendations is required to ensure standardized care. Additionally, because the recommendations are based on limited evidence, further studies are needed to establish outcome measures and adequate treatment regimens for JLS patients.
Tagged assessment, guidelines, juvenile localized scleroderma, Paediatric Rheumatology European Society (PRES), screening, treatment, United Kingdom.