Tocilizumab therapy in juvenile systemic sclerosis: a retrospective single centre pilot study

Area of Science:

• Rheumatology
• Immunology
• Pediatrics

Background:

• Juvenile systemic sclerosis (JSS) is a rare autoimmune disease causing skin fibrosis and internal organ damage.
• Current treatments for JSS have limited efficacy, necessitating exploration of novel therapeutic options.

Purpose of the Study:

• To evaluate the efficacy and safety of tocilizumab (TCZ), an anti-interleukin-6 receptor antibody, as an add-on therapy for juvenile systemic sclerosis (JSS).

Main Methods:

• Nine JSS patients received TCZ in addition to conventional treatments.
• Efficacy was assessed using modified Rodnan skin score (mRSS), carbon-monoxide diffusion capacity (DLCO), thorax high-resolution tomography (HRCT), patient global assessment (PGA), and Juvenile Systemic Sclerosis Severity (J4S) score.

Main Results:

• Significant improvements were observed in mRSS (26.1 to 19.7), DLCO (69.14% to 79.50%), PGA (3.7 to 2.3), and J4S (8.2 to 4.7) (p<0.05 for all).
• Radiologically confirmed improvement on thorax HRCT was noted in 77.8% of patients.
• All patients tolerated TCZ treatment well, with no reported adverse events.

Conclusions:

• Tocilizumab (TCZ) demonstrates potential as an effective and safe treatment for JSS patients, particularly those unresponsive to conventional therapies.
• Further long-term prospective studies with larger patient cohorts are warranted to validate these findings.