This paper reviews the recently published scientific information regarding ANCA-associated vasculitis (AAV), aiming to highlight the most important data from the clinical nephrologists' perspective. The classification, pathomechanism, recent achievements of the treatment, short-term and long-term outcomes of the disease, and the difficulties nephrologists face when taking care for patients with AAV are summarized. There has been significant progress in the understanding of the genetic and pathologic background of the disease in the last years, and results of histological studies guide us to predict long-term renal function. Findings of several multicentered trials with reasonable number of participants provide comparison of the efficacy and safety of different remission induction and maintenance therapies, and evaluate recently introduced immunosuppressive agents. Although the clinical outcome of patients with AAV has improved significantly since modern immunosuppressive drugs are available, the treatment-related complications still contribute to the morbidity and mortality. To improve the survival and quality of life of patients with AAV further, knowledge of the predictors of relapse, end-stage kidney disease, and mortality, also prevention of infections and other treatment-related adverse events are important. The eligibility for renal transplantation and the option for successful pregnancies for young women are also important factors which influence the patients' quality of life. In order to provide favorable outcome, the clinicians need to establish personalized treatment strategies to optimize the intensity and minimize the toxicity of the immunosuppressive therapy.
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http://dx.doi.org/10.1007/s11255-016-1419-4 | DOI Listing |
J Clin Invest
January 2025
Department of Pharmacology, University of Michigan Medical School, Ann Arbor, United States of America.
Dravet syndrome (DS) is a developmental and epileptic encephalopathy (DEE) that begins in the first year of life. While most cases of DS are caused by variants in SCN1A, variants in SCN1B, encoding voltage-gated sodium channel β1 subunits, are also linked to DS or to the more severe early infantile DEE. Both disorders fall under the OMIM term DEE52.
View Article and Find Full Text PDFBMC Rheumatol
January 2025
Department of Nephrology and Rheumatology, Aichi Medical University, 1-1 Karimata, Yazako, Nagakute, Aichi, 480-1195, Japan.
Background: Avacopan, an oral C5a receptor antagonist, demonstrated efficacy as an alternative to glucocorticoid therapy in patients with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) in the phase 3 ADVOCATE trial. However, limited real-world data exist on the outcomes and experiences associated with avacopan use for AAV in Japan.
Methods: We performed a single-centre retrospective analysis and evaluated 21 patients with newly diagnosed or relapsed AAV who received avacopan.
Internet Interv
March 2025
Lyra Health, 270 East Lane Burlingame, CA 94010, United States of America.
Background: Scalable evidence-based treatments for anxiety and depression, such as blended care therapy (BCT) that integrate digital tools are effective, but reporting on long-term outcomes is limited.
Method: This pragmatic observational study examined the symptom stability and trajectories of individuals in the year following engagement in a BCT program. Participants included adults with clinical anxiety and/or depression measured by the Generalized Anxiety Disorder-7 (GAD-7) or Patient Health Questionnaire-9 (PHQ-9).
BMJ Case Rep
January 2025
Department of Nephrology, Barts Health NHS Trust, London, UK.
Anti-neutrophil cytoplasmic antibody-associated vasculitides (AAV) represent a heterogeneous multisystem group of disorders typified by necrotising inflammation of smaller blood vessels, classically yielding a pauci-immune, crescentic glomerulonephritis. Without prompt treatment, there is a significant risk of irreversible damage and ensuing renal impairment.Diagnosis is often challenging, exacerbated by the disorder's often vague and insidious presentation.
View Article and Find Full Text PDFGene Ther
January 2025
Shanghai Bao Pharmaceuticals Co., Ltd., No. 28 Luoxin Road, Baoshan, Shanghai, China.
The approved intravenous adeno-associated virus (AAV) therapies are limited by the widespread prevalence of pre-existing anti-AAV antibodies in the general population, which are known to restrict patients' ability to receive gene therapy and limit transfection efficacy in vivo. To address this challenge, we have developed a novel recombinant human immunoglobulin G degrading enzyme KJ103, characterized by low immunogenicity and clinical value for the elimination of anti-AAV antibodies in gene transfer. Herein, we conducted two randomized, blinded, placebo-controlled, single ascending dose Phase I studies in China and New Zealand, to evaluate the pharmacokinetics, pharmacodynamics, safety and immunogenicity of KJ103 in healthy volunteers.
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