A5087750749- Autoimmune and Inflammatory Disorders Research
- Inflammasome and immune disorders
- Adolescent and Pediatric Healthcare
- Kawasaki Disease and Coronary Complications
- Inflammatory Myopathies and Dermatomyositis
- Rheumatoid Arthritis Research and Therapies
- Immunodeficiency and Autoimmune Disorders
- Salivary Gland Disorders and Functions
- Skin Diseases and Diabetes
- Systemic Sclerosis and Related Diseases
- Immune Cell Function and Interaction
- IL-33, ST2, and ILC Pathways
- Eosinophilic Disorders and Syndromes
- T-cell and B-cell Immunology
- Systemic Lupus Erythematosus Research
- Interstitial Lung Diseases and Idiopathic Pulmonary Fibrosis
- Acute Lymphoblastic Leukemia research
- Dermatological and Skeletal Disorders
- Tuberculosis Research and Epidemiology
- Autoimmune and Inflammatory Disorders
- Salivary Gland Tumors Diagnosis and Treatment
- Sarcoidosis and Beryllium Toxicity Research
- Family and Disability Support Research
- Childhood Cancer Survivors' Quality of Life
- Neurogenetic and Muscular Disorders Research
Istituti di Ricovero e Cura a Carattere Scientifico
2014-2025
Bambino Gesù Children's Hospital
2015-2025
Center for Rheumatology
2014-2024
Izaak Walton Killam Health Centre
2022
Dalhousie University
2022
Penn State Milton S. Hershey Medical Center
2022
University of California, San Francisco
2021
Hackensack University Medical Center
2021
Boston Children's Hospital
2013
Objective The aim of this study is to report the safety and efficacy CD19‐targeting chimeric antigen receptor (CAR) T cells in a child with refractory juvenile dermatomyositis (JDM). Methods We describe 12‐year‐old White boy severe, chronically active JDM multiple immunosuppressive treatment lines, including B cell depletion rituximab. patient received single infusion fresh, autologous, second‐generation anti‐CD19 CAR product (lentiviral vector) manufactured on Prodigy device (1 × 10 6...
Objective To evaluate the expression of type I interferon ( IFN α/β)– and II γ)–inducible genes in muscle biopsy specimens from patients with juvenile dermatomyositis DM ) to correlate their levels histologic clinical features. Methods Expression ‐inducible proinflammatory cytokines were assessed by quantitative polymerase chain reaction (n = 39), Duchenne's muscular dystrophy DMD ), healthy controls. Muscle sections stained scored for severity histopathologic The charts reviewed features at...
Abstract Objective Sjögren syndrome in children is a poorly understood autoimmune disease. We aimed to describe the clinical and diagnostic features of diagnosed with explore how 2016 ACR/EULAR classification criteria apply this population. Methods An international workgroup retrospectively collected cases under 18 years age from 23 centres across eight nations. analysed patterns symptoms, workup, applied criteria. Results identified 300 syndrome. The majority patients n = 232 (77%) did not...
To assess anakinra as a therapy for systemic juvenile idiopathic arthritis (sJIA) in single-center series.We reviewed 25 patients with sJIA treated at least 6 months. The primary outcome was the number of who achieved clinically inactive disease months, according to preliminary criteria and clinical remission JIA.Among evaluated, 14 (56%) met months were classified responders. For each individual patient, we compared dose administered ideal found that there no relation response. We also...
<h3>Objective</h3> To report the first case of a patient with refractory childhood-onset SLE successfully treated anti-CD19 CAR-T cell therapy. <h3>Methods</h3> A single infusion fresh, autologous second-generation product (lentivirus) manufactured on Prodigy device was administered (1x10<sup>6</sup> cells/kg). <h3>Results</h3> female one-year history presented at our attention age 15 years persistent clinical (hemolytic anemia, thrombocytopenia, malar rash, class II/V lupus nephritis,...
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 this analysis was investigate disease status, reasons for discontinuation adverse events Italian JIA patients treated etanercept (ETN). In 2013, all centers Pediatric Rheumatology Study Group were asked make a census given ETN after January 2000. Patients classified three groups: group 1 = still taking ETN; 2...
Janus kinase inhibition (JAKi) has been proposed as a treatment for idiopathic inflammatory myopathies to target increased interferon signalling. Predominantly retrospective reports have demonstrated effectiveness of JAKi in refractory juvenile dermatomyositis (JDM). However, remains an off-label JDM and there may be variation use worldwide. An international survey was conducted investigate approaches JDM. The Childhood Arthritis Rheumatology Research Alliance (CARRA) Therapeutics workgroup...
To evaluate safety, tolerability, and efficacy of etanercept in a cohort patients with juvenile idiopathic arthritis (JIA) under 4 years age.Data were collected at every visit during treatment 25 children who began mean age 3 (range 18-48 months). Safety endpoints included the incidence any adverse events. Efficacy American College Rheumatology (ACR) Pediatric 30, 50, 70 criteria for improvement.Data from JIA treated period 23 months analyzed. All received concomitant medications: 24...
Objective. To evaluate the rate of flare after etanercept (ETN) withdrawal in patients with juvenile idiopathic arthritis (JIA) who attained clinical remission while taking medication, and to identify predictors flare. Methods. Patients were included oligo- (oJIA) rheumatoid factor–negative polyarticular JIA (pJIA) received a first course ETN for at least 18 months, maintained clinically inactive disease (CID) 6 months during treatment, followed 12 withdrawal. Demographic features collected...
Pansclerotic morphea (PSM) is a rare skin disease characterized by progressive stiffening of the with or without typical superficial changes usually seen in (localized scleroderma). Standard therapy, consisting combination systemic glucocorticoids and methotrexate mycophenolate mofetil, does rarely stop progression, which may lead to severe cutaneous sclerosis secondary contractures. Little known about efficacy newer biologicals such as abatacept, fusion protein antibody against CTLA-4,...
Objectives Paediatric Sjögren’s syndrome (pSS) is a rare chronic autoimmune disorder, characterised by inflammation of exocrine glands. B cell hyperactivation plays central role in adult-onset Sjogren. This study was designed to analyse and T phenotype, levels BAFF, selection autoreactive cells patients with pSS. Methods A total 17 diagnosed pSS 13 healthy donors (controls) comparable for age were enrolled the study. subsets frequency peripheral blood analysed flow cytometry. Levels BAFF...
Incidence rates for varicella and herpes zoster were similar in patients with juvenile idiopathic arthritis receiving etanercept/methotrexate (n = 85, 184.9 patient-years [PY]) or methotrexate alone 71, 199.4 PY); no complicated cases reported; labialis incidence was higher versus (0.38 vs 0.24 PY).
Abstract Background Pleural effusion in systemic lupus erythematous (SLE) is a common symptom, and recent studies demonstrated that IL-6 has pivotal role its pathogenesis. Case presentation We report case of 15 years old Caucasian boy with history persistent pleural without lung involvement or fever. Microbiological neoplastic aetiologies were previously excluded. Based on the presence pleuritis, malar rash, reduction C3 C4 levels positivity antinuclear antibody (ANA) anti-double stranded...
<h3>Background</h3> Systemic juvenile idiopathic arthritis (sJIA) accounts for 10-20% of all patients with JIA. The prominent systemic clinical features, the marked elevation inflammatory markers and absence autoantibodies make this disease different from other JIA forms. sJIA should be considered as a polygenic autoinflammatory disease. Interleukin 1 (IL-1) has been shown to major mediator cascade that underlies sJIA. Treatment anakinra reported effective in sizable portion...
<h3>Background</h3> Little is known about the risk of flare after etanercept (ETN) withdrawal in children with Juvenile Idiopathic Arthritis (JIA). Recently we conducted a retrospective chart review 110 patients JIA who discontinued ETN due to persistent clinically inactive disease (CID) on medication and were followed up 12 months withdrawal. We showed that 60% flared arthritis. Male gender, presence ANA elevated CRP at baseline associated higher flare. <h3>Objectives</h3> To evaluate...
Chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature (CANDLE) syndrome is a newly described autoinflammatory disease.
Cryopyrin-associated periodic syndrome (CAPS) is an autoinflammatory caused by heterozygous mutations of CIAS1/NLRP3 gene. Affected patients may present three different phenotypes: familial cold (FCAS), Muckle-Wells and CINCA syndrome. Common symptoms include sporadic or cold-induced non pruritic urticarial rash fever. Severe cases suffers from deafness, meningitis, articular contracture secondary amyloidosis.
<h3>Background</h3> Pediatric Sjögren's syndrome (pSS) is a rare disorder that often diagnosed late due to the lack of validated diagnostic criteria and biomarkers. The pathogenesis largely unknown, but there evidence involvement both innate adaptive branch immune system. Immunological overactivity central in pSS. Several studies showed presence B cells abnormalities patients with SS an expansion naïve decrease frequency memory cells. <h3>Objectives</h3> We set out investigate distribution...
<h3>Background:</h3> Methotrexate (MTX) is the most used drug for treatment of children and adults suffering from arthritis although its intake burdened by well-known side effects. The Intolerance Severity Score (MISS) was originally developed in English [1] to identify patients intolerant MTX. <h3>Objectives:</h3> treat with use adverse MTX intolerance severity score who are aim this study translate validate MISS Italian. <h3>Methods:</h3> Italian version following "guidelines process...
<h3>Background:</h3> Juvenile Idiopathic Arthritis (JIA) is the most common form of chronic arthritis in childhood. The adaptive branch immune system plays a crucial role development JIA. We have showed that switched memory B cells are expanded patients with oligoarticular- and polyarticular-JIA. <h3>Objectives:</h3> To characterize antigen-experienced immunoglobulin repertoire, we performed deep sequencing repertoire peripheral blood (PB) oligo-/ploy-JIA patients(n=14) age matched...
<h3>Background:</h3> Juvenile localised scleroderma (jLS) is a rare rheumatic disease in children characterized by inflammation and fibrosis the skin [1, 2]. The cause pathogenesis of jLS remain unclear, both lesions possible extracutaneous involvement may result functional impairment growth disturbances [2]. treatment options to cure are limited [3]. In recent years, omics technologies have been used identify novel biomarkers different diseases [4, 5]. Among technologies, metabolomics...