Quarter in review – Pediatric Rheumatology

Rakesh Kumar Pilania MD (Pediatrics), DM (Pediatric Clinical Immunology and Rheumatology), MAMS, Associate-FAMS
Assistant Professor, PediatricAllergy Immunology Unit, Department of Pediatrics, Advanced Pediatrics Centre, Post Graduate Institute of Medical Education and Research, Chandigarh (India)

Study in focus 1: Analysis of Coronary Arterial Aneurysm Regression in Patients with Kawasaki Disease by Aneurysm Severity: Factors Associated with Regression1

Kawasaki disease (KD) is a common childhood vasculitis. Coronary artery abnormalities (CAAs) constitute the most significant cardiovascular complication.

Kato et al have reported the timelines of regression of CAAs in 1006 patients from 44 institutions from all over Japan. CAAs were classified as small, medium-sized, and giant aneurysms during acute phases based upon the Z score-based classification. Right coronary artery (RCA) aneurysm regression after 5 and 10 years was observed at 86.8% and 95.5% for small, 75.5% and 83.2% for medium, and 29.6% and 36.3% for giant aneurysms. Corresponding figures for left anterior descending (LAD) aneurysms were 87.6% and 95.3% for small, 69.9% and 80.1% for medium, and 20.8% and 28.8% for giant aneurysms. Children diagnosed with KD in more recent decades experienced significantly greater regression rates, potentially attributed to enhanced early identification and increasing use of timely treatment intensification. Favourable factors for CAA regression were identified as small aneurysms, children below 1 year of age at the time of diagnosis, children with KD diagnosed in the recent period, and female gender.

This study is of seminal importance as it enumerates the factors contributing to regression of CAAs in children with KD.

Study in focus 2: Baricitinib in juvenile idiopathic arthritis: an international, phase 3, randomized, double-blind, placebo-controlled, withdrawal, efficacy, and safety trial2

Children with juvenile Idiopathic Arthritis (JIA) require long-term immunomodulatory therapy. A subset of patients with JIA may remain refractory to available therapeutic options. The role of janus kinase inhibition has been shown in managing children with JIA. A phase 3 trial has already demonstrated the efficacy of tofacitinib in JIA. Baricitinib, a selective JAK1/2 inhibitor known for its efficacy in rheumatoid arthritis, is being explored for JIA.

Ramanan et al. conducted a multinational study (JUVE-BASIS trial) to assess efficacy and safety of baricitinib in children with refractory JIA. The trial involved three phases– i) safety and pharmacokinetic assessment phase ii) open-label lead-in period (12 weeks baricitinib), and iii) double-blind withdrawal randomization phase (placebo vs. baricitinib until disease flare or 32 weeks duration). Eligible participants (2 to <18 years) with JIA subtypes (polyarthritis, extended oligoarthritis, enthesitis-related arthritis, psoriatic arthritis), refractory to 12 weeks of conventional or biologic DMARDs, received oral baricitinib (2mg once daily for 2-8 years, 4 mg for >8 years). A pharmacokinetic assessment verified the appropriateness of this regimen. Those demonstrating an initial response, defined as an American College of Rheumatology (ACR) 30 response after the lead-in phase, were randomized into double-blind withdrawal phase.

The study included 220 patients, 52.7% with prior biologic treatment, mainly anti-Tumour necrosis factor alpha agents. All had prior exposure to methotrexate, with 58% also concurrently receiving methotrexate during the study. After 12 weeks, 74% achieved ACR30 response, almost half reached ACR70. During withdrawal phase, disease flare occurred in 51% on placebo, compared to 17% in the baricitinib group. Median time to flare was 27.14 weeks in placebo arm, while baricitinib arm witnessed a flare rate in <50% patients only. Safety profile of baricitinib was consistent with available literature without any new safety signal. However, infections were marginally higher in baricitinib group and 1 patient had pulmonary embolism.

In conclusion, this study shows the potential role of oral molecule baricitinib in children with refractory JIA.


  1. Kato T, Miura M, Kobayashi T, Kaneko T, Fukushima N, Suda K, et al. Analysis of Coronary Arterial Aneurysm Regression in Patients With Kawasaki Disease by Aneurysm Severity: Factors Associated With Regression. J Am Heart Assoc. 2023;12(3):e022417.
  2. Ramanan AV, Quartier P, Okamoto N, Foeldvari I, Spindler A, Fingerhutová Š, et al. Baricitinib in juvenile idiopathic arthritis: an international, phase 3, randomised, double-blind, placebo-controlled, withdrawal, efficacy, and safety trial. Lancet. 2023;402(10401):555-570.