During the long-term period, two patients discontinued due to insufficient efficacy or patient decision

During the long-term period, two patients discontinued due to insufficient efficacy or patient decision. Results All 20 patients who received study medication completed the short-term period. During the long-term period, two patients discontinued due to insufficient efficacy or patient decision. Median age and disease duration at baseline were 10.5 and 0.75?years, respectively. Week 16 JIA-ACR30 response rate (primary endpoint) was 90.0% (18/20). JIA-ACR50/70/90 response and inactive disease rates at Week 16 were 75.0% (15/20), 70.0% (14/20), 35.0% (7/20), and 25.0% (5/20), respectively. At Week 52, JIA-ACR30/50/70/90 response and inactive disease rates were observed by 88.9% (16/18), 88.9% (16/18), 83.3% (15/18), 66.7% (12/18) and 44.4% (8/18), respectively. CHAQ-DI improved after Week 12. JADAS27-CRP remission and MDA were achieved by 15.0% (3/20) and 45.0% (9/20) of patients at Week 16, CACNG1 and by 50.0% (9/18) and 78.0% (14/18) of patients at Week 52, respectively. The mean abatacept pre-dose serum concentration was above the target therapeutic exposure (10?g/ml) from Week 8 through Week 16. All adverse events were of moderate/moderate intensity, except for one case of severe gastroenteritis. No deaths, malignancies, or autoimmune disorders were observed. No antidrug antibodies were detected RAF265 (CHIR-265) through Week 16; one patient had a positive immunogenic response during the cumulative period. Conclusion Intravenous abatacept was efficacious and well tolerated in Japanese patients with active pJIA. Trial registration ClinicalTrials.gov: “type”:”clinical-trial”,”attrs”:”text”:”NCT01835470″,”term_id”:”NCT01835470″NCT01835470. Date of registration: April 19, 2013. Childhood Health Assessment Questionnaire-Disability Index, juvenile arthritis disease activity score 27 active joint count-C-reactive protein, juvenile idiopathic arthritis, limitation of motion, methotrexate, rheumatoid factor, standard deviation, tumor necrosis factor, visual analog scale Efficacy The proportion of patients who achieved JIA-ACR30, 50, 70 and 90 response, and inactive disease over time from baseline to Week 52 of the cumulative period are shown in Fig.?2. At Week 16, 18/20 (90%) patients achieved a JIA-ACR30 response (primary endpoint). JIA-ACR50, 70, and 90 response rates, and inactive disease RAF265 (CHIR-265) rate were 75.0, 70.0, 35.0, and 25.0%, respectively. During the cumulative period to Week 52, JIA-ACR30 and 50 response rates increased progressively from Week 2 (first assessment) to Week 16 (end of the short-term period) and remained high to Week 52 (Fig. ?(Fig.2).2). JIA-ACR70 and 90 response rates and inactive disease rate also gradually increased to Week 16 followed by a sustained improvement to Week 52 (Fig. ?(Fig.2).2). At Week 52 (Juvenile idiopathic arthritis-American College of Rheumatology criteria 30/50/70/90% improvement All six JIA-ACR core set variables improved from baseline to Week 16 and throughout the cumulative period to Week 52 (Fig.?3). Rapid improvement, as early as Week 2, was observed for number of active joints, number of joints with LOM, PGA score, and CRP level. The improvement observed for active joints and joints with LOM plateaued at Week 28 but was sustained thereafter out to Week 52, whereas improvements in PGA and CRP continued to increase up to Week 52. Little, if any, improvement in CHAQ-DI or PaGA was observed during the early phase of the study (within 12?weeks of starting treatment with abatacept); however, both parameters started to show continuous improvement after 12?weeks of treatment. Open in a separate windows Fig. 3 Time course of JIA-ACR core set variables improvement from baseline to Week 52 of the cumulative period. The six JIA-ACR core RAF265 (CHIR-265) set variables were evaluated as the median (%) improvement from baseline at indicated time points (all treated patients; Childhood Health Assessment Questionnaire-Disability Index, C-reactive protein, Juvenile idiopathic arthritis-American College of Rheumatology criteria, limitation of motion, Parental Global Assessment of patient overall well-being, Physician Global Assessment At baseline, mean JADAS27-CRP score was 13.9, with no patients in remission (JADAS27-CRP score? ?1) or with minimal disease activity (JADAS27-CRP score? ?3.8). Mean JADAS27-CRP score gradually decreased over time from baseline to Week 52 RAF265 (CHIR-265) (Fig.?4a), with a mean change from baseline in JADAS27-CRP score of ??8.7 at Week 16, and C10.8 at Week 52. Remission was achieved in.