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- Ruperto, N., et al.
(författare)
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A randomized, placebo-controlled trial of infliximab plus methotrexate for the treatment of polyarticular-course juvenile rheumatoid arthritis
- 2007
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Ingår i: Arthritis Rheum. - : Wiley. - 0004-3591. ; 56:9, s. 3096-3106
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Tidskriftsartikel (refereegranskat)abstract
- OBJECTIVE: To evaluate the safety and efficacy of infliximab in the treatment of juvenile rheumatoid arthritis (JRA). METHODS: This was an international, multicenter, randomized, placebo-controlled, double-blind study. One hundred twenty-two children with persistent polyarticular JRA despite prior methotrexate (MTX) therapy were randomized to receive infliximab or placebo for 14 weeks, after which all children received infliximab through week 44. Patients received MTX plus infliximab 3 mg/kg through week 44, or MTX plus placebo for 14 weeks followed by MTX plus infliximab 6 mg/kg through week 44. RESULTS: Although a higher proportion of patients in the 3 mg/kg infliximab group than in the placebo group had achieved responses according to the American College of Rheumatology (ACR) Pediatric 30 (Pedi 30) criteria for improvement at week 14 (63.8% and 49.2%, respectively), the between-group difference in this primary efficacy end point was not statistically significant (P = 0.12). By week 16, after the crossover from placebo to infliximab 6 mg/kg when all patients were receiving infliximab, an ACR Pedi 30 response was achieved in 73.2% of all patients. By week 52, ACR Pedi 50 and ACR Pedi 70 responses had been reached in 69.6% and 51.8%, respectively, of patients. Infliximab was generally well tolerated, but the safety profile of infliximab 3 mg/kg appeared less favorable than that of infliximab 6 mg/kg, with more frequent occurrences of serious adverse events, infusion reactions, antibodies to infliximab, and newly induced antinuclear antibodies and antibodies to double-stranded DNA observed with the 3 mg/kg dose. CONCLUSION: While infliximab at 3 mg/kg and 6 mg/kg showed durable efficacy at 1 year, achievement of the primary efficacy end point at 3 months did not differ significantly between infliximab-treated and placebo-treated patients. Safety data indicated that the 6-mg/kg dose may provide a more favorable risk/benefit profile. These results warrant further investigation in children with JRA.
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- Ruperto, N., et al.
(författare)
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The Pediatric Rheumatology International Trials Organization/American College of Rheumatology provisional criteria for the evaluation of response to therapy in juvenile systemic lupus erythematosus : prospective validation of the definition of improvement
- 2006
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Ingår i: Arthritis and Rheumatism. - : Wiley. - 0004-3591 .- 1529-0131. ; 55:3, s. 355-363
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Tidskriftsartikel (refereegranskat)abstract
- OBJECTIVE: To use the Pediatric Rheumatology International Trials Organization (PRINTO) core set of outcome measures to develop a validated definition of improvement for the evaluation of response to therapy in juvenile systemic lupus erythematosus (SLE).METHODS: Thirty-seven experienced pediatric rheumatologists from 27 countries, each of whom had specific experience in the assessment of juvenile SLE patients, achieved consensus on 128 patient profiles as being clinically improved or not improved. Using the physicians' consensus ratings as the gold standard measure, the chi-square, sensitivity, specificity, false-positive and false-negative rates, area under the receiver operating characteristic curve, and kappa level of agreement for 597 candidate definitions of improvement were calculated. Only definitions with a kappa value greater than 0.7 were retained. The top definitions were selected based on the product of the content validity score multiplied by its kappa statistic.RESULTS: The definition of improvement with the highest final score was at least 50% improvement from baseline in any 2 of the 5 core set measures, with no more than 1 of the remaining worsening by more than 30%.CONCLUSION: PRINTO proposes a valid and reproducible definition of improvement that reflects well the consensus rating of experienced clinicians and that incorporates clinically meaningful change in core set measures in a composite end point for the evaluation of global response to therapy in patients with juvenile SLE. The definition is now proposed for use in juvenile SLE clinical trials and may help physicians to decide whether a child with SLE responded adequately to therapy.
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- Apaz, Maria Teresa, et al.
(författare)
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Health-related quality of life of patients with juvenile dermatomyositis: results from the Pediatric Rheumatology International Trials Organisation multinational quality of life cohort study.
- 2009
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Ingår i: Arthritis and rheumatism. - : Wiley. - 0004-3591 .- 1529-0131. ; 61:4, s. 509-17
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Tidskriftsartikel (refereegranskat)abstract
- OBJECTIVE: To investigate the health-related quality of life (HRQOL) change over time, as measured by the Child Health Questionnaire (CHQ), and its determinants in patients with active juvenile dermatomyositis (DM). METHODS: We assessed patients with juvenile DM at both baseline and 6 months of followup, and healthy children age < or =18 years. Potential determinants of poor HRQOL included demographic data, physician's and parent's global assessments, muscle strength, functional ability as measured by the Childhood Health Assessment Questionnaire (C-HAQ), global disease activity assessments, and laboratory markers. RESULTS: A total of 272 children with juvenile DM and 2,288 healthy children were enrolled from 37 countries. The mean +/- SD CHQ physical and psychosocial summary scores were significantly lower in children with juvenile DM (33.7 +/- 11.7 versus 54.6 +/- 4.1) than in healthy children (45.1 +/- 9.0 versus 52 +/- 7.2), with physical well-being domains being the most impaired. HRQOL improved over time in responders to treatment and remained unchanged or worsened in nonresponders. Both physical and psychosocial summary scores decreased with increasing levels of disease activity, muscle strength, and parent's evaluation of the child's overall well-being. A C-HAQ score >1.6 (odds ratio [OR] 5.06, 95% confidence interval [95% CI] 2.03-12.59), child's overall well-being score >6.2 (OR 5.24, 95% CI 2.27-12.10), and to a lesser extent muscle strength and alanine aminotransferase level were the strongest determinants of poor physical well-being at baseline. Baseline disability and longer disease duration were the major determinants for poor physical well-being at followup. CONCLUSION: We found that patients with juvenile DM have a significant impairment in their HRQOL compared with healthy peers, particularly in the physical domain. Physical well-being was mostly affected by the level of functional impairment.
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