Pilot Study of Atorvastatin and Anakinra in Children With Coronary Artery Abnormalities Secondary...
Kawasaki DiseaseKawasaki disease (KD) is the leading cause of acquired heart disease in children in the developed world. Despite available treatment, 25% of children in San Diego County appropriately treated for KD develop coronary artery abnormalities that could lead to complications later in life, including heart attack. Although we can identify children with KD that have these coronary artery abnormalities, there is no approved additional treatment to decrease coronary artery inflammation and arrest or prevent damage to the coronary arteries. Statins, a class of drugs that is known for lowering cholesterol, have also been shown to decrease inflammation in general as well as at the level of the vessel wall. Anakinra, a therapy that blocks the high levels of interleukin 1 (IL1) that leads to inflammation during acute KD, has been shown in the KD mouse model to prevent the development of coronary artery damage. Both of these therapies have been demonstrated to be safe and well-tolerated in KD patients. Therefore, we propose to study the effects of combination therapy with atorvastatin and anakinra in children with acute KD and early coronary artery abnormalities.
Defibrotide in Children With High Risk Kawasaki Disease
Kawasaki DiseaseThis study evaluates the safety of defibrotide with IVIG in children with high risk Kawasaki disease.
Efficacy of Immunoglobulin Plus Prednisolone in Reducing Coronary Artery Lesion in Patients With...
Kawasaki DiseaseThis study evaluates the efficacy of the addition of prednisolone to conventional initial treatment (intravenous immunoglobulin [IVIG] plus aspirin) in reducing coronary artery lesion in children with Kawasaki disease (KD) .
A Trial Comparing the Efficacy and Safety of Anakinra Versus Intravenous Immunoglobulin (IVIG) Retreatment,...
Kawasaki DiseaseKawasaki disease (KD) is the most frequent vasculitis in younger children <5years, and the first cause of acquired ischemic myocardiopathy in childhood. Exceptionally, KD may cause early death during the acute phase by myocardial infarction, but may compromise the long-term cardiovascular outcome by accelerating atherosclerotic disease. The incidence of KD is high in far-Eastern countries and Hawaii but KD is relatively rare in other regions (10/100000 children <5years in northern Europe) which makes it difficult to develop research on these rare population. Early recognition and treatment by intravenous immunoglobulins (IVIG) influences the prognosis positively. IVIG are the standard of care and decrease significantly the risk of coronary aneurysms. However, despite a first infusion of IVIG, 20% of KD patients remain febrile and have high risk of coronary vasculitis. Recent Japanese research group assessed additional cyclosporine treatment in first line KD treatment but failed preventing relapse. To date there is no agreement for a more effective second line treatment. Based on the auto-inflammatory pattern of KD, the investigators hypothesize that anti IL-1 blocking agents could bring a rapid and sustained effect on systemic and coronary inflammation in patients with KD. Our hypotheses are: Anakinra treatment may reduce the early and long-term mortality of patients with Kawasaki Disease (KD), by a rapid and sustained effect on vascular inflammation. The safety of anakinra is good, as the drug has a very short half-life, which allows its rapid withdrawal in case of serious adverse event. The use of anakinra is not associated with the risk of contamination by infectious agents, which remain even minimal, a possibility with the use of IVIG.
Cardiovascular Status of Children 5 Years After Kawasaki Disease
VasculitisSystemic1 moreThe aim of present study is to determine cardiovascular status of children who had KD in past and to identify possible biochemical markers of cardiovascular damage in those patients. In this cross-sectional study children with history of KD will be examined 5 years after receiving intravenous immunoglobulin treatment (IVIG) and compared to healthy controls in terms of: serum levels of endothelial injury markers (circulating endothelial cells, endocan, soluble thrombomodulin, vascular endothelial growth factor (VEGF) and soluble E-selectin), peripheral blood pressure, central blood pressure, arterial stiffness parameters (measured by applanation tonometry), carotid intima media thickness (cIMT), capillaroscopy and echocardiography.
Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children...
Coronavirus Infection (COVID-19)Pulmonary Arterial Hypertension23 moreThe study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose for children. The primary objective of this study is to evaluate the PK of understudied drugs currently being administered to children per SOC as prescribed by their treating provider.
Clinical Precise Diagnosis and Treatment in the Kawasaki Disease
Kawasaki DiseaseThe purpose of this project is to explore the differential gene expression profile of Kawasaki disease, and will explore the diagnosis and treatment targets related to coronary artery injury or kawasaki disease susceptibility, vascular damage, IVIG (intravenous immunoglobulin) treatment resistance, incomplete Kawasaki disease, etc.
Rivaroxaban Versus Warfarin for Thromboprophylaxis in Children With Giant Coronary Aneurysms After...
Kawasaki DiseaseCoronary Aneurysm3 moreTo evaluate the efficacy and safety of rivaroxaban compared to warfarin for thromboprophylaxis in children with giant coronary aneurysms after Kawasaki disease
Characterization of Multisystem Inflammatory Syndrome in Children (MIS-C) and Its Relationship to...
Kawasaki DiseaseInflammationBeginning in mid-March 2020, pediatricians in communities in Western Europe, the UK, and the Eastern U.S. that had been severely affected by the Covid-19 pandemic noted an increased number of children presenting with fever and evidence of severe inflammation who required admission to intensive care. The syndrome was branded by the CDC in the U.S. as Multisystem Inflammatory Syndrome in Children (MIS-C). The most severely affected children presented with heart failure leading to shock and the absence of significant pulmonary disease. The clinical presentation in these patients shared many features with Kawasaki disease (KD), a self-limited pediatric vasculitis that can result in coronary artery aneurysms.The inflammatory markers, however, were much higher even than KD shock syndrome, a variant of KD presenting with distributive shock and requiring inotropic and vasoactive support in the ICU. Some patients were polymerase chain reaction (PCR)+ for SARS-CoV-2 while most were virus-negative but had detectable antibody suggesting that MIS-C was an immune-mediated reaction to antecedent exposure to the virus. While patients were being diagnosed with shock and MIS-C, children with a milder version of MIS-C that shared many features of KD were being diagnosed in these same regions.
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford...
Rare DisordersUndiagnosed Disorders316 moreCoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures for rare diseases. The CoRDS team works with patient advocacy groups, individuals and researchers to help in the advancement of research in over 7,000 rare diseases. The registry is free for patients to enroll and researchers to access. Visit sanfordresearch.org/CoRDS to enroll.