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Tocilizumab in the treatment of systemic-onset juvenile idiopathic arthritis - single-centre experience
Justyna Roszkiewicz1, Krzysztof Orczyk1, Elżbieta Smolewska1
1Department of Paediatric Cardiology and Rheumatology, Medical University of Łódź, Poland.
Insights
Tocilizumab effectively treats systemic-onset juvenile idiopathic arthritis in children, rapidly reducing fever and inflammation. The treatment demonstrated a good safety profile with sustained efficacy over one year.
Area of Science:
- Pediatric Rheumatology
- Immunology
- Pharmacology
Background:
- Systemic-onset juvenile idiopathic arthritis (SoJIA) is a severe autoimmune disease in children.
- Effective and safe treatment options for SoJIA are crucial for long-term patient outcomes.
Purpose of the Study:
- To evaluate the efficacy and long-term safety of tocilizumab in pediatric patients with SoJIA.
- To assess the impact of tocilizumab on clinical symptoms and inflammatory markers.
Main Methods:
- Retrospective analysis of 10 pediatric patients with SoJIA treated with tocilizumab.
- Evaluation of treatment effectiveness using ACR Pedi criteria and monitoring of inflammatory markers (CRP, ESR).
- Assessment of side effects, including transaminase elevation and neutropenia.
Main Results:
- Tocilizumab rapidly reduced fever and inflammatory markers (CRP, ESR) by over 90% within 12 weeks.
- All patients achieved ACR Pedi 50, with significant improvements to ACR Pedi 70 and ACR Pedi 90 over one year.
- Observed side effects like elevated transaminases and neutropenia were generally mild and transient.
Conclusions:
- Tocilizumab is an effective treatment for severe systemic-onset juvenile idiopathic arthritis in children.
- The drug exhibits a favorable safety profile, supporting its consideration as a first-line therapy.
Objectives:
The aim of the study was to evaluate the efficacy and long-term safety of tocilizumab treatment in children with systemic-onset juvenile idiopathic arthritis in a single centre.
Material And Methods:
The study was based on a retrospective analysis of a cohort of 10 patients with systemic-onset juvenile idiopathic arthritis who were treated with tocilizumab in the period September 2011-July 2017. Their medical records were analysed taking into consideration the effectiveness of tocilizumab treatment and frequency of side effects.
Results:
Before the initiation of treatment, 9/10 patients from the study group complained of fever and had significantly increased values of inflammatory markers, with the median CRP concentration 41.1 mg/l (norm < 5 mg/l) and ESR 37 mm/h (norm < 12 mg/l). The period of the initial 12 weeks of treatment was a quantum leap in the course of the disease: all children were afebrile, and inflammatory markers values decreased by 99.4% in the case of CRP and 91.9% in ESR. All patients fulfilled ACR Pedi 50 criteria, and 3 of them achieved ACR Pedi 70. In the next stages of treatment the response to tocilizumab was sustained, reaching 10 children achieving ACR Pedi 70 and 5 ACR Pedi 90 after one year of therapy. Tocilizumab appeared to be relatively safe in the study group. Although elevation of transaminases and neutropenia were observed in 5/10 patients, they were usually mild and transitional in their course.
Conclusions:
Tocilizumab is both effective and has a relatively good safety profile in children with severe systemic-onset juvenile idiopathic arthritis. It should be considered in the recommendations as a first-line treatment of this disease.
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