- Indian Rheumatology association
Anjani Gummadi MD Paediatrics, DM Paediatric Clinical Immunology and Rheumatology
Consultant Paediatric Rheumatologist and Immunologist, Ankura Hospitals for Women and Children, Hyderabad, India
Safety and efficacy of tofacitinib for the treatment of patients with juvenile idiopathic arthritis: preliminary results of an open-label, long-term extension study
Tofacitinib was approved by the US Food and Drug Administration in 2020 for the treatment of patients with polyarticular JIA and is currently being used for various other rheumatic diseases in children like systemic JIA, juvenile dermatomyositis, uveitis, etc. However, the safety and long-term efficacy in children has been debatable.
To evaluate the long-term safety, tolerability, and efficacy of tofacitinib, a long-term extension study was conducted at centers of the Paediatric Rheumatology INternational Trials Organisation (PRINTO) or the Pediatric Rheumatology Collaborative Study Group (PRCSG) in 22 countries. 225 JIA patients with ages ranging from 2 to 18 years were given tofacitinib 5 mg two times per day or equivalent weight-based doses. Among patients with polyarticular course JIA, JIA-ACR70/ 90 response rates were 60% and 33.6% respectively, at one month. Less than <5% flares were recorded and juvenile arthritis disease activity score, JADAS27 was 22 at baseline, 6.2 at month 1, and 2.8 at month 48. At month 48, 46.8% of patients had inactive disease. Serious infections occurred in <5% of patients. No new safety findings were observed during follow-up that were unique to the JIA population treated with tofacitinib.
This study supports that tofacitinib appears to be an effective oral option for long-term use in patients with JIA with an established safety profile and significantly reduced flares.
Defining Criteria for Disease Activity States in Systemic Juvenile Idiopathic Arthritis Based on the Systemic Juvenile Arthritis Disease Activity Score
Systemic JIA (sJIA)is the most severe form of childhood arthritis with an unpredictable course. Regular assessment of disease activity is important, however, JADAS cannot be used for sJIA as it stands apart from the other categories of JIA, owing to the prominent extra-articular manifestations. Systemic JADAS (sJADAS) has been developed with five components- physician global assessment of disease activity, parent and patient global assessments of child’s wellbeing, count of joints with active disease, acute phase reactants, and modified Systemic Manifestation Score (mSMS).
The current study was formulated to develop and validate cutoff values in the sJADAS10 that distinguish the states of disease activity in children with sJIA. The cutoff definition was performed using a large multinational data set comprising 400 patients enrolled at 30 pediatric rheumatology centers in 11 countries and 4 continents. The score yields a total score ranging from 0 (no disease activity) to 50 (maximum disease activity). The sJADAS10 cutoff that separated inactive from minimal disease activity was 2.9; minimal disease activity from moderate activity was 10, and moderate from high disease activity was 20.6(sensitivity of 94.2%) and a specificity of 85.5%).
These simple and easy-to-perform cut-off criteria will provide reference values for monitoring of the disease course in patients in day-to-day practice; optimizing treatment in the treat-to-target strategy and providing uniformity in research.
