MSJ872889_supplemental_table – Supplemental material for Treatment of multiple sclerosis with rituximab: A multicentric Italian–Swiss experience

Supplemental material, MSJ872889_supplemental_table for Treatment of multiple sclerosis with rituximab: A multicentric Italian–Swiss experience by Chiara Zecca, Francesca Bovis, Giovanni Novi, Marco Capobianco, Roberta Lanzillo, Jessica Frau, Anna Maria Repice, Bahia Hakiki, Sabrina Realmuto, Simona Bonavita, Erica Curti, Laura Brambilla, Giorgia Mataluni, Paola Cavalla, Alessia Di Sapio, Elisabetta Signoriello, Stefania Barone, Giorgia T Maniscalco, Ilaria Maietta, Isabella Maraffi, Giacomo Boffa, Simona Malucchi, Agostino Nozzolillo, Giancarlo Coghe, Claudia Mechi, Giuseppe Salemi, Antonio Gallo, Rosaria Sacco, Maria Cellerino, Maria Malentacchi, Marcello De Angelis, Lorena Lorefice, Elia…

Proceedings Of The 23Rd Paediatric Rheumatology European Society Congress: Part Two

Treatment of multiple sclerosis with rituximab: A multicentric Italian–Swiss experience

Background: Rituximab, an anti-CD20 monoclonal antibody leading to B lymphocyte depletion, is increasingly used as an off-label treatment option for multiple sclerosis (MS). Objective: To investigate the effectiveness and safety of rituximab in relapsing–remitting (RR) and progressive MS. Methods: This is a multicenter, retrospective study on consecutive MS patients treated off-label with rituximab in 22 Italian and 1 Swiss MS centers. Relapse rate, time to first relapse, Expanded Disability Status Scale (EDSS) progression, incidence of adverse events, and radiological outcomes from 2009 to 2019 were analyzed. Results: A total of 355/451 enrolled subjects had at least one follow-up visit an…

Additional file 2 of Opportunistic infections in immunosuppressed patients with juvenile idiopathic arthritis: analysis by the Pharmachild Safety Adjudication Committee

Additional file 2 figure. Hierarchy of MedDra clinically-validated international medical terminology.

The Italian version of the Juvenile Arthritis Multidimensional Assessment Report (JAMAR)

The Juvenile Arthritis Multidimensional Assessment Report (JAMAR) is a new parent/patient reported outcome measure that enables a thorough assessment of the disease status in children with juvenile idiopathic arthritis (JIA). We report the results of the cross-cultural adaptation and validation of the parent and patient versions of the JAMAR in the Italian language. The reading comprehension of the questionnaire was tested in 10 JIA parents and patients. Each participating centre was asked to collect demographic, clinical data and the JAMAR in 100 consecutive JIA patients or all consecutive patients seen in a 6-month period and to administer the JAMAR to 100 healthy children and their paren…

INSAID Variant Classification and Eurofever Criteria Guide Optimal Treatment Strategy in Patients with TRAPS: Data from the Eurofever Registry

Contains fulltext : 231528.pdf (Publisher’s version ) (Closed access) BACKGROUND: TNF receptor-associated periodic syndrome (TRAPS) is a rare autoinflammatory disease caused by dominant mutation of the TNF super family receptor 1A (TNFRSF1A) gene. Data regarding long-term treatment outcomes are lacking. OBJECTIVE: To assess correlations of genotype-phenotypes in patients with TRAPS, as defined by the International Study Group for Systemic Autoinflammatory Diseases (INSAID) classification and Eurofever criteria, with treatment responses. METHODS: Data from 226 patients with variants of the TNFRSF1A gene and enrolled in the Eurofever registry were classified according to the INSAID classifica…

Additional file 2 of Opportunistic infections in immunosuppressed patients with juvenile idiopathic arthritis: analysis by the Pharmachild Safety Adjudication Committee

Additional file 2 figure. Hierarchy of MedDra clinically-validated international medical terminology.

Proceedings of the 23rd Paediatric Rheumatology European Society Congress: part one

Additional file 1 of Opportunistic infections in immunosuppressed patients with juvenile idiopathic arthritis: analysis by the Pharmachild Safety Adjudication Committee

Additional file 1 figure. Flowchart of the process.

Disease status, reasons for discontinuation and adverse events in 1038 Italian children with juvenile idiopathic arthritis treated with etanercept

Background: Data from routine clinical practice are needed to further define the efficacy and safety of biologic medications in children with juvenile idiopathic arthritis (JIA). The aim of this analysis was to investigate the disease status, reasons for discontinuation and adverse events in Italian JIA patients treated with etanercept (ETN). Methods: In 2013, all centers of the Italian Pediatric Rheumatology Study Group were asked to make a census of patients given ETN after January 2000. Patients were classified in three groups: group 1 = patients still taking ETN; group 2 = patients discontinued from ETN for any reasons; group 3 = patients lost to follow-up while receiving ETN. All three…

Additional file 4 of Opportunistic infections in immunosuppressed patients with juvenile idiopathic arthritis: analysis by the Pharmachild Safety Adjudication Committee

Additional file 4 Table 2. Concomitant medications administered at the time of “confirmed OI”. Bio: biologic, mtx: methotrexate; ste: systemic steroids; sDMARDs: synthetic disease modifying antirheumatic drugs; *sDMARDs are intendend other than MTX.

Exposure to natalizumab throughout pregnancy: effectiveness and safety in an Italian cohort of women with multiple sclerosis.

ObjectiveAssessing the risk of clinical and radiological reactivation during pregnancy and post partum in women with multiple sclerosis (MS) treated with natalizumab (NTZ) throughout pregnancy (LONG_EXP) compared with women interrupting treatment before (NO_EXP) and within >−30 days and ≤90 days from conception (SHORT_EXP), and describing newborns’ outcomes.MethodsMaternal clinical and radiological outcomes and obstetric and fetal outcomes were retrospectively collected and compared among groups (NO_EXP, SHORT_EXP, LONG_EXP). Predictors of clinical and radiological reactivation were investigated through univariable and multivariable analysis.Results170 eligible pregnancies from 163 women…

The Latvian version of the Juvenile Arthritis Multidimensional Assessment Report (JAMAR).

The Juvenile Arthritis Multidimensional Assessment Report (JAMAR) is a new parent/patient reported outcome measure that enables a thorough assessment of the disease status in children with juvenile idiopathic arthritis (JIA). We report the results of the cross-cultural adaptation and validation of the parent and patient versions of the JAMAR in the Latvian language. The reading comprehension of the questionnaire was tested in 10 JIA parents and patients. Each participating centre was asked to collect demographic, clinical data and the JAMAR in 100 consecutive JIA patients or all consecutive patients seen in a 6-month period and to administer the JAMAR to 100 healthy children and their paren…

Phenotypic Variability and Disparities in Treatment and Outcomes of Childhood Arthritis Throughout the World: Results from the EPOCA Study

Background: The characteristics and burden of childhood arthritis have never been studied on a worldwide basis. We aimed to investigate prevalence of disease categories, treatment modalities and disease status across different geographic areas. Methods: International paediatric rheumatologists were asked to enrol a consecutive sample of children with juvenile idiopathic arthritis. Each patient underwent retrospective and cross-sectional assessments, including parent-reported outcomes. Level of disease activity and damage were correlated with wealth of the country, expressed as gross domestic product per capita. Findings: Between 2011 and 2016, 9,081 patients were enrolled at 130 centres in …

Phenotypic variability and disparities in treatment and outcomes of childhood arthritis throughout the world: an observational cohort study

Made available in DSpace on 2019-10-05T16:54:20Z (GMT). No. of bitstreams: 0 Previous issue date: 2019-04-01 IRCCS Istituto Giannina Gaslini Background To our knowledge, the characteristics and burden of childhood arthritis have never been studied on a worldwide basis. We aimed to investigate, with a cross-sectional study, the prevalence of disease categories, treatment methods, and disease status in patients from across different geographical areas and from countries with diverse wealth status. Methods In this multinational, cross-sectional, observational cohort study, we asked international paediatric rheumatologists from specialised centres to enrol children with a diagnosis of juvenile …

Additional file 1 of Opportunistic infections in immunosuppressed patients with juvenile idiopathic arthritis: analysis by the Pharmachild Safety Adjudication Committee

Additional file 1 figure. Flowchart of the process.

Additional file 3 of Opportunistic infections in immunosuppressed patients with juvenile idiopathic arthritis: analysis by the Pharmachild Safety Adjudication Committee

Additional file 3 Table 1. Complete table with the frequency of the 682 infections adjudicated by the SAC. Infections were reported after evaluation of the cases available in Pharmachild compared to the pathogens/presentations in the provisional list approved by the Safety Adjudication Committee (SAC). Data are presented as per the MedDRA High Level Term (HLT) and Preferred Term (PT) sorted by frequencies in descending order (HLT and then PT). *For definition see Step 5.

Efficacy of different rituximab therapeutic strategies in patients with neuromyelitis optica spectrum disorders

Abstract Objective To evaluate disease activity according to rituximab (RTX) induction and maintenance regimens in a multicenter real-life dataset of NMOSD patients. Methods This is an observational-retrospective multicentre study including patients with NMOSD treated with RTX in 21 Italian and 1 Swiss centers. Demographics, relapse rate and adverse events over the follow-up were summarized taking into account induction strategy (two-1 g infusions at a 15-day interval (IND-A) vs. 375 mg/m2/week infusions for one month (IND-B)) and maintenance therapy (regimen A (M-A) with fixed time-points infusions vs. regimen B (M-B) based on cytofluorimetric driven reinfusion regimens, the least further …

Development and initial validation of the macrophage activation syndrome/primary hemophagocytic lymphohistiocytosis score, a diagnostic tool that differentiates primary hemophagocytic lymphohistiocytosis from macrophage activation syndrome

OBJECTIVE: To develop and validate a diagnostic score that assists in discriminating primary hemophagocytic lymphohistiocytosis (pHLH) from macrophage activation syndrome (MAS) related to systemic juvenile idiopathic arthritis. STUDY DESIGN: The clinical, laboratory, and histopathologic features of 362 patients with MAS and 258 patients with pHLH were collected in a multinational collaborative study. Eighty percent of the population was assessed to develop the score and the remaining 20% constituted the validation sample. Variables that entered the best fitted model of logistic regression were assigned a score, based on their statistical weight. The MAS/HLH (MH) score was made up with the i…

Dissecting the Heterogeneity of Macrophage Activation Syndrome Complicating Systemic Juvenile Idiopathic Arthritis

Objective.To seek insights into the heterogeneity of macrophage activation syndrome (MAS) complicating systemic juvenile idiopathic arthritis (sJIA) through the analysis of a large patient sample collected in a multinational survey.Methods.International pediatric rheumatologists and hemato-oncologists entered their patient data, collected retrospectively, in a Web-based database. The demographic, clinical, laboratory, histopathologic, therapeutic, and outcome data were analyzed in relation to (1) geographic location of caring hospital, (2) subspecialty of attending physician, (3) demonstration of hemophagocytosis, and (4) severity of clinical course.Results.A total of 362 patients were incl…

Additional file 4 of Opportunistic infections in immunosuppressed patients with juvenile idiopathic arthritis: analysis by the Pharmachild Safety Adjudication Committee

Additional file 4 Table 2. Concomitant medications administered at the time of “confirmed OI”. Bio: biologic, mtx: methotrexate; ste: systemic steroids; sDMARDs: synthetic disease modifying antirheumatic drugs; *sDMARDs are intendend other than MTX.

Pediatric Recurrent Pericarditis: Appropriateness of the Standard of Care and Response to IL-1 Blockade

Objective: To analyze, in a cohort of pediatric patients with recurrent pericarditis undergoing anti-interleukin (IL)-1 treatment: the agent and dosing used as first-line treatment, the long-term efficacy of IL-1 blockers, the percentage of patients achieving a drug-free remission, and the presence of variables associated with drug-free remission. Study design: Data were collected from patients' charts. The annualized relapse rate (ARR) was used for evaluation of treatment efficacy, and bivariate logistic regression analysis was used for variables associated with drug-free remission. Results: Fifty-eight patients, treated between 2008 and 2018, were included in the study (mean follow-up. 2.…

Additional file 3 of Opportunistic infections in immunosuppressed patients with juvenile idiopathic arthritis: analysis by the Pharmachild Safety Adjudication Committee

Additional file 3 Table 1. Complete table with the frequency of the 682 infections adjudicated by the SAC. Infections were reported after evaluation of the cases available in Pharmachild compared to the pathogens/presentations in the provisional list approved by the Safety Adjudication Committee (SAC). Data are presented as per the MedDRA High Level Term (HLT) and Preferred Term (PT) sorted by frequencies in descending order (HLT and then PT). *For definition see Step 5.