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Safety and effectiveness of abatacept in juvenile idiopathic arthritis: results from the PRINTO/PRCSG registry
Daniel J Lovell1, Nikolay Tzaribachev2, Michael Henrickson1
1Division of Rheumatology, Cincinnati Children's Hospital Medical Center, University of Cincinnati, Cincinnati, OH, USA.
Insights
This study shows abatacept is safe and effective for juvenile idiopathic arthritis (JIA) over five years. It effectively controlled disease activity, with many patients achieving inactive disease or remission.
Area of Science:
- Immunology
- Pediatric Rheumatology
- Clinical Trials
Background:
- Juvenile idiopathic arthritis (JIA) is a chronic autoimmune disease affecting children.
- Abatacept is an immunomodulatory drug used to treat JIA.
- Long-term safety and effectiveness data in a real-world setting are crucial for treatment decisions.
Purpose of the Study:
- To evaluate the interim 5-year safety and effectiveness of abatacept in pediatric patients with JIA.
- To report on adverse events and disease activity measures in a large JIA registry.
Main Methods:
- An observational study (Abatacept JIA Registry) enrolled children with JIA receiving abatacept.
- Safety was the primary endpoint, with effectiveness assessed by the clinical 10-joint Juvenile Arthritis Disease Activity Score (cJADAS10).
- Data were analyzed using as-observed methods following STROBE guidelines.
Main Results:
- 569 patients were analyzed with 1214.6 patient-years of safety data.
- The incidence rate of serious adverse events was 5.52 per 100 patient-years.
- Significant improvements in disease activity were observed, with 55.9% achieving low disease activity and 20.3% inactive disease by month 3, sustained over 5 years.
Conclusions:
- Abatacept demonstrated a favorable safety profile in JIA patients over 5 years.
- The treatment led to well-controlled disease activity, with a notable percentage achieving inactive disease or remission.
- No new safety signals were identified, supporting its use in JIA management.
Objective:
The aim of this study was to report the interim 5-year safety and effectiveness of abatacept in patients with JIA in the PRINTO/PRCSG registry.
Methods:
The Abatacept JIA Registry (NCT01357668) is an ongoing observational study of children with JIA receiving abatacept; enrolment started in January 2013. Clinical sites enrolled patients with JIA starting or currently receiving abatacept. Eligible patients were assessed for safety (primary end point) and effectiveness over 10 years. Effectiveness was measured by clinical 10-joint Juvenile Arthritis Disease Activity Score (cJADAS10) in patients with JIA over 5 years. As-observed analysis is presented according to the Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) guidelines.
Results:
As of 31 March 2020, 587 patients were enrolled; 569 are included in this analysis (including 134 new users) with 1214.6 patient-years of safety data available. Over 5 years, the incidence rate (IR) per 100 patient-years of follow-up of serious adverse events was 5.52 (95% CI: 4.27, 7.01) and of events of special interest was 3.62 (95% CI: 2.63, 4.86), with 18 serious infections [IR 1.48 (95% CI: 0.88, 2.34)]. As early as month 3, 55.9% of patients achieved cJADAS10 low disease activity and inactive disease (20.3%, 72/354 and 35.6%, 126/354, respectively), sustained over 5 years. Disease activity measures improvement over 5 years across JIA categories.
Conclusion:
Abatacept was well tolerated in patients with JIA, with no new safety signals identified and with well-controlled disease activity, including some patients achieving inactive disease or remission.
Trial Registration:
Clinicaltrials.gov, NCT01357668.
