Publications by authors named "Ruperto N"
Objective: To evaluate the influence of pelvic magnetic resonance imaging (MRI) findings on axial disease assessment in juvenile spondyloarthritis (JSpA).
Methods: This was a cross-sectional study of patients with JSpA with suspected axial disease. Three experts reviewed each case and rated their confidence (-3 to +3) in the presence of axial disease, first with clinical data and second with clinical and MRI data.
Background: The Paediatric Rheumatology International Trials Organisation (PRINTO) recently undertook an effort to better harmonize the pediatric and adult arthritis criteria. These provisional criteria are being refined for optimal performance. We aimed to investigate differences between patients who did and did not fulfill these PRINTO criteria amongst youth diagnosed with juvenile spondyloarthritis (SpA) that met axial juvenile SpA (axJSpA) classification criteria.
View Article and Find Full Text PDFObjective: To assess changes in gene expression following tofacitinib treatment and investigate transcription patterns as potential predictors of treatment response in patients with active juvenile idiopathic arthritis (JIA).
Methods: Whole-blood samples were collected from patients with JIA at baseline and after 18 weeks of open-label tofacitinib treatment. Patients who achieved a JIA-American College of Rheumatology (ACR) response of 70% or above at week 18 were classified as treatment responders (TRs), whereas those with at most a JIA-ACR30 were classified as poor responders (PRs).
Article Synopsis
- The Physician Global Assessment (PhGA) is commonly used by pediatric rheumatologists to evaluate disease activity in juvenile idiopathic arthritis (JIA), but lacks a standardized scoring method.
- A survey conducted among members of pediatric rheumatology organizations identified which factors influence scoring, focusing on non-systemic (nsJIA) and systemic JIA (sJIA), as well as considering extra-articular manifestations and imaging.
- Results from 276 participants across 54 countries highlighted key factors for scoring, showing strong agreement on including certain extra-articular manifestations, while also indicating some variability and need for consensus in PhGA scoring practices.
Article Synopsis
- The article discusses a long-term study on the safety and effectiveness of etanercept, a treatment for juvenile idiopathic arthritis (JIA), in children and young adults over a span of ten years.
- JIA is an inflammatory condition that causes joint pain and stiffness, usually diagnosed before age 16, requiring ongoing treatment until the disease is in remission.
- The research highlights the need to evaluate the long-lasting safety of etanercept, which modulates the immune system to alleviate symptoms of arthritis.
Objective: We examine levels of candidate blood-based biomarkers (CBBs) in patients with juvenile idiopathic arthritis (JIA) treated with tofacitinib.
Methods: Patients with JIA who participated in clinical trial NCT02592434 received tofacitinib from baseline to week 18. Serial serum samples were assayed for CBBs (S100A8/9, S100A12, interleukin-18 [IL-18], serum amyloid A, resistin, vascular endothelial growth factor, angiopoietin-1, angiopoietin-2, matrix metalloproteinase 8 [MMP8], MMP2, tissue inhibitor of metalloproteinases 1, leptin, chemokine [C-X-C motif] ligand 9, soluble IL-2 receptor, intercellular adhesion molecule 1, soluble tumor necrosis factor receptor, IL-6, IL-23, monocyte chemotactic protein 1, chemokine [C-C motif] ligand 18 [CCL18], and CCL20).
Objective: To report pharmacokinetics (PK), immunogenicity, clinical effect, and safety of intravenous (IV) golimumab in children with active polyarticular-course juvenile idiopathic arthritis (pcJIA) who participated in A Study to Evaluate the Pharmacokinetics, Efficacy and Safety of Intravenous Golimumab in Pediatric Participants With Active Polyarticular Course Juvenile Idiopathic Arthritis Despite Methotrexate Therapy (GO-VIVA)'s open-label, long-term extension (LTE) through week 252.
Methods: GO-VIVA participants who continued IV golimumab (80 mg/m every 8 weeks) after week 52 were included. PK and safety were assessed through week 244 (last dose) and week 252, respectively, and clinical response through week 116.
Article Synopsis
- The study aims to evaluate the current treatment approaches for macrophage activation syndrome (MAS) globally and identify inconsistencies in clinical practices.
- A thorough literature review resulted in 57 relevant papers involving 1148 patients with MAS, primarily linked to systemic juvenile idiopathic arthritis (sJIA), systemic lupus erythematosus (SLE), and Kawasaki disease (KD).
- Findings indicate high-dose glucocorticoids and certain targeted therapies, such as IL-1 and IFNγ inhibitors, are effective, particularly for sJIA-associated MAS, but highlight a need for more standardized research to improve treatment consistency for different conditions.
Objective: The goal was to develop and validate classification criteria for axial juvenile spondyloarthritis (SpA; AxJSpA).
Methods: This international initiative consisted of four phases: (1) item generation, (2) item reduction, (3) criteria development, and (4) validation of the AxJSpA criteria by an independent team of experts in an internationally representative validation cohort.
Results: These criteria are intended to be used on youth with a physician diagnosis of juvenile SpA and for whom axial disease is suspected.
Objective: To develop parent- and child-centered versions of the Juvenile Arthritis Disease Activity Score (JADAS) and to provide preliminary evidence of their validity.
Methods: Validation analyses were conducted on two large multinational datasets of patients with juvenile idiopathic arthritis (JIA) and included assessment of construct validity, internal consistency and structure, discriminative validity, responsiveness to change, and predictive validity.
Results: The parJADAS and patJADAS include four parent/patient-reported outcomes, each measured on a 0-10 scale: assessment of overall disease activity; rating of pain intensity; assessment of activity of joint disease; duration of morning stiffness.
Background: Juvenile idiopathic arthritis (JIA) comprises a heterogeneous group of conditions that can cause marked disability and diminished quality of life. Data on predictors of clinical response are insufficient to guide selection of the appropriate biologic agent for individual patients. This study aimed to investigate the propensity of S100A8/9 and S100A12 as predictive biomarkers of abatacept response in polyarticular-course juvenile idiopathic arthritis (pJIA).
View Article and Find Full Text PDFArticle Synopsis
- The study evaluates the safety, tolerability, and effectiveness of tofacitinib in children and adolescents with juvenile idiopathic arthritis (JIA) in a long-term extension (LTE) study.
- Out of 225 patients treated with tofacitinib, a high percentage experienced adverse events (AEs), although serious AEs were relatively low, with a focus on infection rates and disease activity.
- Results showed significant improvement in disease activity scores over time, with a notable increase in patients achieving inactive disease status after 48 months of treatment.
Objectives: The aim of the study was to establish an international multicenter registry to collect data on patients with Multisystem Inflammatory Syndrome in Children (MIS-C), in order to highlight a relationship between clinical presentation, age of onset and geographical distribution on the clinical outcome.
Study Design: Multicenter retrospective study involving different international societies for rare immunological disorders.1009 patients diagnosed with MIS-C between March and September 2022, from 48 centers and 22 countries were collected.
Article Synopsis
- - The study assessed safety events in patients with Autoinflammatory diseases using data from the Eurofever registry, focusing on adverse events (AEs) reported since 2009.
- - A total of 2464 patients were included in the analysis, highlighting that 479 AEs were reported, with serious AEs and drug-related AEs being particularly noted, especially in relation to biologic DMARDs and colchicine.
- - The findings underscore the need for consistent monitoring of AEs in rare conditions to better understand the safety of treatments.
Introduction: Vascular events account for a considerable burden of morbidity and mortality in Behçet syndrome (BS). Thrombosis occurs in 1.8-21 % pediatric BS patients, even if the real prevalence is still largely unknown.
View Article and Find Full Text PDFObjective: To investigate the safety and efficacy of subcutaneous tocilizumab (SC-TCZ) treatment in a long-term extension (LTE) of clinical trials in polyarticular or systemic juvenile idiopathic arthritis (pJIA or sJIA).
Methods: Patients with pJIA or sJIA from two open-label, 52-week phase 1b core trials of SC-TCZ who had adequate response per investigator assessment entered the LTE and continued SC-TCZ treatment according to body weight-based dosing regimens until commercial availability or up to 5 years. Pharmacokinetics, pharmacodynamics, and efficacy were assessed for up to 3 years, and safety for up to 5 years in the LTE.
Article Synopsis
- Scientists worked on a special test called JDMAI to help doctors understand how active a disease called juvenile dermatomyositis is in kids.
- They looked at information from 139 kids during a study and found numbers (cut-offs) that show different levels of the disease: inactive, low, moderate, and high activity.
- The test is good at showing the disease's status by comparing it to how the kids feel and how they do in daily life, and it's ready to be used in hospitals and research.
Objective: The aim of this study was to report the interim 5-year safety and effectiveness of abatacept in patients with JIA in the PRINTO/PRCSG registry.
Methods: The Abatacept JIA Registry (NCT01357668) is an ongoing observational study of children with JIA receiving abatacept; enrolment started in January 2013. Clinical sites enrolled patients with JIA starting or currently receiving abatacept.
Objectives: Limited information is available on the clinical features, treatment modalities and outcomes of the juvenile idiopathic arthritis (JIA) categories of enthesitis-related arthritis (ERA) and juvenile psoriatic arthritis (JPsA). This study was aimed to describe the characteristics of Italian children with ERA and JPsA and to compare them with those of patients with the other categories of JIA.
Methods: Patients were part of a multinational sample included in a study aimed to investigate the prevalence of disease categories, treatment approaches, and disease status in patients from across different geographical areas (EPOCA Study).
Article Synopsis
- The study aimed to validate the Pediatric Behçet's Disease criteria using an evidence-based method, involving 210 pediatric patients with various conditions.
- After evaluations by blinded experts, a consensus was reached for 66.2% of patients, identifying 24 as confirmed Behçet's Disease and 10 as probable.
- The PEDBD criteria showed high specificity but lower sensitivity compared to other criteria, highlighting the need for further research with larger international cohorts to assess their effectiveness.
Article Synopsis
- - The study aimed to compare the clinical features and treatment responses of patients with the TNFRSF1A-pR92Q variant to those with classical TRAPS, PFAPA, and SURF, using data from the Eurofever registry.
- - 361 patients were analyzed, revealing that pR92Q variant patients typically had an older onset of disease and shared more similarities with SURF patients, while classical TRAPS patients exhibited distinct traits like migratory rash and familial history.
- - Despite some differences in symptoms, all patient groups responded similarly to treatment, with steroids being the most commonly prescribed and effective; this suggests the need for a revised approach to diagnosing and managing pR92Q variant patients.
Breaking down the fences among registries on autoinflammatory diseases: the E-Merge project.
Orphanet J Rare Dis
July 2023
Background: Among the various numbers of different autoinflammatory diseases (AIDs), the absolute majority of them remains rare, with a single representative in large populations. This project, endorsed by PRES, supported by the EMERGE fellowship program, and performed in line with the Metadata registry for the ERN RITA (MeRITA), has the objective of performing a data synchronization attempt of the most relevant research questions regarding clinical features, diagnostic strategies, and optimal management of autoinflammatory diseases.
Results: An analysis of three large European registries: Eurofever, JIR-cohort and AID-Net, with a total coverage of 7825 patients from 278 participating centers from different countries, was performed in the context of epidemiological and clinical data merging.