ACR Pedi Research Articles

Abatacept treatment improves health-related quality of life, pain, and sleep quality in juvenile idiopathic arthritis patients

Methods 190 JIA patients were treated with abatacept for 4 months in an open-label lead-in period (Period A). ACR Pedi 30 responders (n = 123) were then randomized 1:1 to receive abatacept or placebo for up to 6 months in a double-blind withdrawal period (Period B). HRQOL was assessed by the Child Health Questionnaire (CHQ), sleep quality was measured by the Children's Sleep Habits Questionnaire (CSHQ), and pain by a 0–100 mm VAS. Mean change from baseline in each period was calculated and compared between the treatment groups (in Period B), and the change over time was examined.

7.4 Improvements in individual disease components are sustained with long-term adalimumab therapy for polyarticular Juvenile Idiopathic Arthritis

To control symptoms and prevent increasing disability in children with active polyarticular Juvenile Idiopathic Arthritis (JIA), long-term, effective treatment that controls all aspects of the disease is necessary. Individual ACR Pedi response criteria were analyzed for the 128 patients who entered the open-label extension (OLE) of the Phase III study of adalimumab in the treatment of polyarticular JIA. Measurements of disease activity were performed at each visit, including active joint count (AJC), number of joints with limitation of passive motion (LOM), parent's or patient's assessment of patient's pain (PaP), disability index of the Children's Health Assessment Questionnaire (CHAQ DI), and physician's global assessment of disease activity (PhDA). Observed data were examined for those patients who had been treated with adalimumab throughout the study and reached more than 1 year in the OLE (Week 56; 75% of entering patients had data available). Patients entering the study had active polyarticular JIA, with clinically significant joint involvement, limitation of motion, pain, and disability in performing daily living activities. Long-term treatment with adalimumab provided marked improvements in disease activity (Table ​(Table1).1). The established safety profile for adalimumab remained consistent. Table 1 Improvements in JIA with adalimumab therapy Long-term treatment with adalimumab substantially improves multiple aspects of polyarticular JIA.

Reduction in missed school days and improvement in parent activity participation in children with juvenile idiopathic arthritis treated with abatacept

Methods 190 JIA patients were treated with abatacept for 4 months in an open-label lead-in period (Period A). ACR Pedi 30 responders (n = 123) were then randomized 1:1 to receive abatacept or placebo for 6 months in a double-blind withdrawal period (Period B). Activity participation for the child and the parents were assessed, along with clinical and quality of life (QOL) parameters. Mean change from baseline in each period was analyzed, and the two treatment groups were compared.

Adalimumab with or without Methotrexate in Juvenile Rheumatoid Arthritis

Tumor necrosis factor (TNF) has a pathogenic role in juvenile rheumatoid arthritis. We evaluated the efficacy and safety of adalimumab, a fully human monoclonal anti-TNF antibody, in children with polyarticular-course juvenile rheumatoid arthritis. Patients 4 to 17 years of age with active juvenile rheumatoid arthritis who had previously received treatment with nonsteroidal antiinflammatory drugs underwent stratification according to methotrexate use and received 24 mg of adalimumab per square meter of body-surface area (maximum dose, 40 mg) subcutaneously every other week for 16 weeks. We randomly assigned patients with an American College of Rheumatology Pediatric 30% (ACR Pedi 30) response at week 16 to receive adalimumab or placebo in a double-blind fashion every other week for up to 32 weeks. Seventy-four percent of patients not receiving methotrexate (64 of 86) and 94% of those receiving methotrexate (80 of 85) had an ACR Pedi 30 response at week 16 and were eligible for double-blind treatment. Among patients not receiving methotrexate, disease flares (the primary outcome) occurred in 43% of those receiving adalimumab and 71% of those receiving placebo (P=0.03). Among patients receiving methotrexate, flares occurred in 37% of those receiving adalimumab and 65% of those receiving placebo (P=0.02). At 48 weeks, the percentages of patients treated with methotrexate who had ACR Pedi 30, 50, 70, or 90 responses were significantly greater for those receiving adalimumab than for those receiving placebo; the differences between patients not treated with methotrexate who received adalimumab and those who received placebo were not significant. Response rates were sustained after 104 weeks of treatment. Serious adverse events possibly related to adalimumab occurred in 14 patients. Adalimumab therapy seems to be an efficacious option for the treatment of children with juvenile rheumatoid arthritis. (ClinicalTrials.gov number, NCT00048542.)

Long-term follow-up on effectiveness and safety of etanercept in juvenile idiopathic arthritis: the Dutch national register

Objective:We undertook an observational study to obtain a complete overview of the long-term effectiveness and safety of etanercept in patients with different juvenile idiopathic arthritis (JIA) subtypes.Methods:At baseline we collected...

Efficacy and safety of tocilizumab in patients with systemic-onset juvenile idiopathic arthritis: a randomised, double-blind, placebo-controlled, withdrawal phase III trial

Systemic-onset juvenile idiopathic arthritis does not always respond to available treatments, including antitumour necrosis factor agents. We investigated the efficacy and safety of tocilizumab, an anti-interleukin-6-receptor monoclonal antibody, in children with this disorder. 56 children (aged 2-19 years) with disease refractory to conventional treatment were given three doses of tocilizumab 8 mg/kg every 2 weeks during a 6-week open-label lead-in phase. Patients achieving an American College of Rheumatology Pediatric (ACR Pedi) 30 response and a C-reactive protein concentration (CRP) of less than 5 mg/L were randomly assigned to receive placebo or to continue tocilizumab treatment for 12 weeks or until withdrawal for rescue medication in a double-blind phase. The primary endpoint of the double-blind phase was an ACR Pedi 30 response and CRP concentration of less than 15 mg/L. Patients responding to tocilizumab and needing further treatment were enrolled in an open-label extension phase for at least 48 weeks. The analysis was done by intention to treat. This study is registered with ClinicalTrials.gov, numbers NCT00144599 (for the open-label lead-in and double-blind phases) and NCT00144612 (for the open-label extension phase). At the end of the open-label lead-in phase, ACR Pedi 30, 50, and 70 responses were achieved by 51 (91%), 48 (86%), and 38 (68%) patients, respectively. 43 patients continued to the double-blind phase and were included in the efficacy analysis. Four (17%) of 23 patients in the placebo group maintained an ACR Pedi 30 response and a CRP concentration of less than 15 mg/L compared with 16 (80%) of 20 in the tocilizumab group (p<0.0001). By week 48 of the open-label extension phase, ACR Pedi 30, 50, and 70 responses were achieved by 47 (98%), 45 (94%), and 43 (90%) of 48 patients, respectively. Serious adverse events were anaphylactoid reaction, gastrointestinal haemorrhage, bronchitis, and gastroenteritis. Tocilizumab is effective in children with systemic-onset juvenile idiopathic arthritis. It might therefore be a suitable treatment in the control of this disorder, which has so far been difficult to manage.

Long-term outcome of juvenile idiopathic arthritis following a placebo-controlled trial: sustained benefits of early sulfasalazine treatment

Objectives:A previous 24-week randomised trial demonstrated that sulfasalazine (SSZ) treatment was superior to placebo (PLAC) in suppressing disease activity in patients with oligo- and polyarticular onset juvenile idiopathic arthritis (JIA)....

Leflunomide in juvenile idiopathic arthritis

The gold standard of therapy for patients with juvenile idiopathic arthritis (JIA), previously referred to as juvenile rheumatoid arthritis (JRA), has been methotrexate and, if methotrexate fails, a biological agent. Leflunomide, an oral pyrimidine synthesis inhibitor, has been shown to be well tolerated and effective in both short-term and long-term studies of adult rheumatoid arthritis (RA). This article reviews the current state-of-the-art use of leflunomide in JIA. A Phase Ib study demonstrated that leflunomide is effective in patients who have either failed methotrexate or are intolerant of methotrexate (50% American College Rheumatology [ACR] Pedi 30). Following the encouraging results of this initial study, a large, multicentered comparator study of leflunomide versus methotrexate was performed. This study demonstrated that both drugs had excellent response rates (ACR Pedi 30 rates of 68 and 89%, respectively) although there was a statistically significant higher response rate for methotrexate.