Allogeneic Hematopoietic Cell Transplantation (HCT) in the National...
Background/Purpose: The deficiency of adenosine deaminase 2 (DADA2) is an autosomal recessive autoinflammatory disease caused by biallelic mutations in ADA2. The diagnosis of DADA2 is made by the...
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Experience with and Management of HLH-like Toxicities Following Chimeric...
Background/Purpose: Chimeric antigen receptor T-cell (CAR-T) therapy is a highly effective form of adoptive cell immunotherapy combining antigen specific targeting capabilities with T-cell based...
View ArticleCD3 Downregulation on T-cells Is Concomitant with Arginase Upregulation on...
Background/Purpose: Rationale: Juvenile idiopathic arthritis (JIA) is an inflammatory autoimmune disorder driven by dysfunction of the joint tissue and abnormal immune responses. As deep phenotyping of...
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Failure of Methotrexate Monotherapy and Subsequent Response to Tumor Necrosis...
Background/Purpose: Pediatric non-infectious uveitis is often treated with systemic immunomodulators in a step-wise fashion, with methotrexate (MTX) followed by tumor necrosis factor alpha inhibitors...
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Predictors of Moderate/High Disease Activity in Childhood-Onset Systemic...
Background/Purpose: Substantial risk of early morbidity and mortality exists for patients with childhood-onset systemic lupus erythematosus (cSLE), but there are limited studies identifying predictors...
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Medication Adherence in Adolescents and Young Adults with Childhood Onset SLE
Background/Purpose: Compared to adults with SLE, young patients with SLE face particular challenges, including more severe disease, more renal involvement and medications side effects like growth...
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Therapeutic Interferon Gamma Neutralization with Emapalumab in Patients with...
Background/Purpose: Interferon gamma (IFNγ) is a pivotal mediator of HLH. Emapalumab (a fully human anti-IFNγ monoclonal antibody) is efficacious in patients with primary HLH. In autoinflammatory...
View ArticleThe First 18 Months of a Pediatric Rheumatology Service at a Tertiary...
Background/Purpose: Pediatric rheumatic diseases impart a significant disease burden upon children and their families with the potential to cause diminished quality of life and significant direct and...
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Novel Algorithm to Increase Sensitivity of Detecting Active Arthritis in...
Background/Purpose: Juvenile idiopathic arthritis (JIA) is the most common rheumatic disease in children. The use of infrared thermal imaging for screening JIA has not utilized standardized image...
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The Impact of Psychiatric Diagnosis and Treatment on Medication Adherence in...
Background/Purpose: Youth with systemic lupus erythematosus (SLE) experience significant health care burden and high rates of psychiatric disorders. Optimizing medication adherence is critical for...
View ArticleGenetic Susceptibility Loci for Periodic Fever, Aphthous Stomatitis,...
Background/Purpose: Periodic fever, aphthous stomatitis, pharyngitis, cervical adenitis (PFAPA) syndrome is the most common periodic fever syndrome of childhood. Although familial clustering of the...
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The Killer Immunoglobulin-like Receptor KIR3DL1 in Combination with HLA-Bw4...
Background/Purpose: Pediatric Acute-onset Neuropsychiatric Syndrome (PANS) is characterized by abrupt onset of obsessive-compulsive disorder (OCD) and/or food restriction with other specified...
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Evaluation of Self-report Screening Measures in the Detection of Depressive...
Background/Purpose: Screening for co-morbid psychiatric disorder is of high clinical importance in childhood-onset systemic lupus erythematosus (cSLE). However, there are no validated screening...
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Long-Term Outcomes in Children Born to Anti-Ro and/or anti-La Positive Mothers
Background/Purpose: Neonatal Lupus Erythematosus (NLE) is an acquired autoimmune disorder associated with the transplacental passage of maternal anti-Ro and/or anti-La antibodies. NLE manifestations...
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Implications of Positive Tests for ANCA in a Pediatric Population
Background/Purpose: Testing for ANCA, particularly performed by ELISA (anti-MPO/PR3) is highly sensitive and specific for ANCA-associated vasculitis (AAV). However ANCA testing may be used in clinical...
View ArticleAdvanced Multidisciplinary Care: A New Approach to Childhood Arthritis
Background/Purpose: Many patients are overwhelmed with the diagnosis of chronic childhood arthritis and require long-term treatments. Our objective was to develop a new, child and family centered,...
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The Importance of the Patient Global Health Assessment of Disease Activity in...
Background/Purpose: Polyarticular juvenile idiopathic arthritis (pJIA) is a subtype of JIA wherein more than four joints are affected with arthritis; it is characterized by unpredictable flares and...
View ArticleClinical and Laboratory Characteristics in Juvenile-onset Systemic Lupus...
Background/Purpose: Systemic lupus erythematous (SLE) is a systemic autoimmune/inflammatory condition. Up to 20% of patients develop symptoms before their 18th birthday and are diagnosed with...
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Change in Treatments and Outcomes After Implementation of a National...
Background/Purpose: Juvenile idiopathic arthritis (JIA) is currently the most common childhood chronic rheumatic disease with high burden and socioeconomic costs for the patient’s family and the health...
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