Epstein-Barr Virus Infection Triggers Hyperinflammation and Cytokine Storm in Healthy Children
Abstract format and assignment number: Oral presentation 0829
Presenting author: P. Lee (USA)
Date: Saturday, 16th November 2024
This abstract discusses immune profiling of children who presented to the emergency room with >= 3 days of fever and confirmed viral infections with Olink proximity extension assay and flow cytometry. Acute EBV infection uniquely triggered high levels of multiple cytokines, and the pattern was similar to the cytokine storm associated with HLH/MAS. This informs the unique presentation of EBV and its connection to HLH.
Approach to Janus Kinase Inhibition for Juvenile Dermatomyositis Among Childhood Arthritis and Rheumatology Research Alliance (CARRA) and Paediatric Rheumatology European Society (PReS) Providers
Abstract format and assignment number: Poster 1265
Presenting author: E. Datyner (USA)
Date: Sunday, 17th November 2024
This abstract discusses the results of an international survey to investigate the use of JAKi by pediatric rheumatology providers in JDM. 66% of responders had used JAKi, and 77% noted clinical improvement in most or all patients. This highlights the potential application of JAKi in refractory JDM and the need for clinical trials.
Efficacy and Safety of Tofacitinib in Patients with Active Systemic Juvenile Idiopathic Arthritis
Abstract format and assignment number: Poster L09
Presenting author: H. Brunner (USA)
Date: Monday, 18th November 2024
This abstract reports the results of a Phase 3, global, 2-phase, randomized withdrawal study investigating the efficacy, safety, and tolerability of tofacitinib in patients with active sJIA. While fewer patients experienced flares with tofacitinib, the study did not meet the primary endpoint of demonstrating statistically significant prolongation in time to disease flare in the double-blind phase. Improvements in sJIA disease activity were observed with open label tofacitinib with >80% of patients achieiving an aJIA-ACR30 response by 8 weeks.
Current Treatment of Macrophage Activation Syndrome Worldwide: The METAPHOR Project, a PReS/PRINTO Real-life International Survey
Abstract format and assignment number: Poster 2174
Presenting author: F. Minoia (Italy)
Date: Monday, 18th November 2024
This abstract discusses the results from an international survey on MAS treatment. Methylprednisolone was the most common glucocorticoid used for all types of MAS. There were geographical differences noted when looking at other medications, notably anakinra. Anakinra is used as 1st/2nd line by more than half of patientsfrom North America and Western Europe but is unavailable to most patients from Asia and South America. This survey identifies gaps in access to medications and heterogeneity in treatment.
Efficacy and Safety of Ixekizumab in Children with Active Juvenile Psoriatic Arthritis and Enthesitis Related Arthritis (COSPIRIT-JIA): 16-week Results of a Multicentre, Randomised, Open-label Study
Abstract format and assignment number: Oral presentation 2573
Presenting author: A. Ramanan (UK)
Date: Monday, 18th November 2024
This abstract discusses the results through week 16 of the COSPIRIT-JIA trial, an ongoing multicenter, randomized, open-label Phase 3 study of ixekizumab (IXE) in patients aged 2- <18 with active JPsA and ERA. The 1st 16 weeks is open label treatment with adalimumab as the reference arm. In the IXE arm 88.9% achieved JIA ACR30, demonstrating efficacy in both JPsA and ERA. Safety findings were consistent for known safety profile. These results suggest that another treatment for these JIA subtypes is on the horizon.
Development and Validation of a Novel Score System to Guide Diagnostic Procedures in Children with Concerns of Chronic Nonbacterial Osteomyelitis
Abstract format and assignment number: Oral abstract 2627
Presenting author: R. Pooni (USA)
Date: Monday, 18th November 2024
This abstract discusses the development of a score system to determine the likelihood of CNO prior to the decision of whether to do a bone biopsy. Both the development and validation cohorts consisted of CNO and mimicker cases. Imaging findings and routinely measured features were used to develop the model. The sensitivity was 75% and specificity was 88% when using a threshold of >=1. This model could have clinical applications and decrease the need for bone biopsy in those with high probability of CNO.
Efficacy and Safety of Emapalumab in Children and Adults with Macrophage Activation Syndrome (MAS) in Still’s Disease: Results from a Phase 3 Study and a Pooled Analysis of Two Prospective Trials
Abstract format and assignment number: Oral abstract L19
Presenting author: A. Grom (USA)
Date: Tuesday, 19th November 2024
This abstract provides additional data from an expanded population of patients with MAS in Still’s disease who received emapalumab after inadequate response to high-dose glucocorticoids. At week 8, 53.8% met the primary endpoint of complete response, and 85% achieved complete response at any time point. After initiation of emapalumab, biomarkers of hyperinflammation rapidly reduced, and 72% had a clinically meaningful GC dose reduction.
Erin Treemarcki
Erin is an Associate Professor of Pediatric Rheumatology at the University of Utah. Her research interests include juvenile spondyloarthritis, childhood Sjogren Disease, and mental health in pediatric rheumatology.
Erin is a member of the Childhood Arthritis and Rheumatology Research Alliance (CARRA) and is currently the Early Investigator Committee Vice-Chair and the co-leader of the Juvenile Spondyloarthritis Workgroup.