Juvenile RA/Pediatric Rheumatology
Sangeeta Sule, M.D.
Abstract 719: Assessment of Open Label Co-Stimulation Blockade with Abatacept in Children and Adolescents with Active Juvenile Idiopathic Arthritis (JIA)
Authors: Daniel J. Lovell1, Nicolino Ruperto2, Anne-Marie Prieur2, Eliana Paz2, Nadina Eugenia Rubio Pérez2, Clovis A. Silva2, Carlos Abud Mendoza2, Ruben Burgos-Vargas2, Claudia Magalhães2, Jose A. Melo-Gomes2, Valeria Gerloni2, Flavio R. Sztajnbok2, Morton Scheinberg2, Leonard Sigal3, Alan Block3, Ye Zhou3, Patricia L. Cornet3, Lori Pagliaro3, Edward Giannini1, Alberto Martini2. 1For PRCSG, Cincinnati Children's Hospital Medical Center, Cincinnati, OH; 2For PRINTO, IRCCS Istituto G Gaslini, Genoa, Italy; 3Bristol-Myers Squibb Co, Princeton, NJ
Purpose: Abatacept (ABA) is a “costimulation modulator” blocking signaling required for antigen-specific T cell activation. ABA has been effective in treating adult RA. These data are from an open-label lead-in period to evaluate ABA in systemic onset juvenile idiopathic arthritis (JIA).
Methods: 190 patients aged 6-17 years with active JIA (extended oligoarthritis, polyarthritis (RF + or -) or systemic with articular manifestations only) who had continued active disease not responsive to ≥ 1 DMARD. Patients were treated with open-label ABA (10mg/kg IV) on Day 1, 15, 29, and every 28 days thereafter until Day 113. At that time, patients were randomized to double blind therapy for 6 months. All DMARDs (except methotrexate) were discontinued before entry. The data presented are from the open-label phase and the double-blind study is ongoing.
Results: Patients were predominately Caucasians (77.4%) and female (72.1%) with a mean age of 12.4 ± 3.0 years. 74.2% were receiving methotrexate. At Day 113 the mean percent reduction from baseline was 55.7%. Of the 190 patients, 123 (64.7%) had an ACR Pedi 30 response, 49.5% and ACR Pedi 50, 28.4% an ACR Pedi 70. Headache (13.2%), nausea (10%), cough (8.9%), and diarrhea (8.9%) were the most common non-serious adverse reactions. One case of acute lymphocytic leukemia occurred but was not thought to be related to treatment. Other serious adverse reactions included flares of JIA, 1 case of varicella, and 1 ruptured ovarian cyst.
Conclusions: Treatment with open-label ABA in JIA was well tolerated and some patients had a positive response to treatment.
Editorial Comments: This is an important study of ABA in the treatment of JIA. ABA has already been shown to be effective in adult RA and it is important that studies be conducted in the pediatric population. The treatment responses in this study were not as striking as in other biological- therapy studies in JIA but it is difficult to make this comparison. The ABA study included patients who had failed one or more DMARD and this may be a more resistant population. The long-term safety of ABA, including risk of infections or malignancy, needs to be carefully studied in JIA.