Kawasaki Disease

Researchers are studying Kawasaki Disease (KD), a common blood vessel inflammation affecting young children under 5 years old, which can lead to heart problems and rarely early death. This trial focuses on children who did not improve after the usual treatment with intravenous immunoglobulins (IVIG). The study aims to evaluate whether Anakinra, an anti-inflammatory medicine, works better than a second IVIG infusion to reduce fever and inflammation in these patients. Participants are randomly assigned to receive either Anakinra or a second IVIG infusion. Anakinra is given by injection starting at 4 mg/kg, with possible dose increases based on fever response over 14 days. The IVIG group receives standard care with a 2g/kg infusion. Treatment effects on fever, inflammation, disease activity, and heart artery changes are compared between groups. Safety and side effects are closely monitored. During the study, children will have regular clinical exams, temperature checks, and laboratory tests including blood markers of inflammation. Parents will assess disease activity and record symptoms in a follow-up booklet. Heart ultrasounds are done to assess artery health. Monitoring continues up to 60 days to evaluate treatment response, side effects, and heart-related outcomes. The main measure of success is whether fever resolves within 48 hours after starting treatment.

Children with chronic health conditions have a higher chance of developing heart and blood vessel diseases earlier than their peers due to faster aging of these organs. Experts recommend careful monitoring and early care, but the exact causes of this increased risk are not well understood. This research seeks to better understand why these children face greater cardiovascular risks and to support future prevention efforts. The study observes children and young adults aged 6 to 25 who have chronic conditions linked to higher cardiovascular risk. It uses non-invasive methods to check the health of large blood vessels like the carotid artery and small vessels in the retina. These assessments include measuring arterial stiffness, endothelial function of the brachial artery, and retinal vessel features, all done at the start and during yearly follow-ups. Participants will undergo regular evaluations including scans of blood vessels and retinal imaging to monitor changes over time. The study measures key indicators such as arterial stiffness and retinal vessel diameter to track early signs of vascular aging. This ongoing observation helps researchers identify those at risk before obvious heart or vascular disease appears. The study is sponsored by Semmelweis University and runs until 2029, involving close monitoring without intervention.

Juvenile idiopathic arthritis (JIA) is a common chronic rheumatic disease in children that causes pain, joint swelling, and loss of function, which can reduce quality of life and lead to muscle weakness, limited motion, and walking difficulties. Researchers are evaluating the Dynamic Gait Index (DGI) as a new tool to assess walking ability in children with JIA, since current methods lack a functional walking test for this group. The study aims to determine whether the DGI is a reliable and valid measure for evaluating walking challenges in these children. The study includes two groups: children diagnosed with JIA and age-matched healthy volunteers. The DGI test measures walking on level ground, changing speed, head turns, pivot turns, jumping over obstacles, walking around obstacles, and climbing stairs. Each activity is scored to assess walking ability. Additional physical tests include the Timed Up and Go Test, walking speed measurement, Wong-Baker FACES Pain Scale for pain assessment, Pediatric Balance Scale, and Childhood Health Assessment Questionnaire. These tests will be conducted at the start and again two weeks later. Participants will undergo physical examinations involving several walking and balance tests. Researchers will measure outcomes such as the DGI score, walking speed, timed up and go performance, pain levels, and balance ability. These measurements will help evaluate the walking function and risks related to falls in children with JIA compared to healthy peers. The study will monitor participants over a two-week period after baseline testing to assess the consistency and reliability of the DGI for this purpose.

Kawasaki disease (KD) is a leading cause of acquired heart disease in children, particularly in developed countries. The study evaluates the performance of two scoring systems, Kobayashi and Kawanet, which aim to predict resistance to intravenous immunoglobulin (IVIG) treatment in KD patients. Resistance to IVIG occurs in 10-20% of patients and poses a significant risk for cardiac complications like coronary aneurysms. The study focuses on a multi-center European and North Indian cohort to assess the sensitivity and specificity of these scores in non-Asian populations. This observational study does not involve experimental treatments but monitors KD patients aged 1 to 17 years diagnosed according to American Heart Association (AHA) criteria. It compares the Kobayashi and Kawanet IVIG resistance scores to determine their accuracy in predicting which patients may not respond to standard IVIG therapy. The study spans an average duration of one year during which patients are observed for IVIG resistance. Participants will be followed up to measure the rate and percentage of IVIG resistance throughout the study period. Data collection includes clinical evaluations and monitoring of patient outcomes related to cardiac health. The study aims to validate the scoring systems for better prediction of treatment resistance, which may help guide future treatment decisions. Participants will be involved in regular assessments over the year-long study period to track their response and health status.

Researchers are exploring the genetics and biological processes behind diseases that cause intermittent fever, such as familial Mediterranean fever, TRAPS, hyper-IgD syndrome, and related disorders. This observational study aims to discover genetic variants linked to these inflammatory diseases and to understand immune system features and the clinical characteristics of these conditions through detailed genetic and medical data analysis. Participants include patients with known or suspected autoinflammatory diseases, their family members, and healthy volunteers. Patients may undergo a variety of tests and procedures, including physical exams, blood and urine tests, X-rays, specialist consultations, and genetic sampling via blood or saliva. Some may undergo leukapheresis to collect white blood cells, while relatives and healthy volunteers may also provide samples for genetic and functional studies. Follow-up visits occur approximately every six months to monitor symptoms and adjust care as needed. During the study, participants will provide medical and family histories, undergo physical exams, and complete various laboratory tests. Genetic counseling is offered to discuss risks and treatment options. Researchers will measure genetic linkages annually to identify variants related to inflammatory diseases. The study involves ongoing monitoring to collect clinical data and biological samples, supporting a comprehensive understanding of these rare inflammatory conditions.

Researchers are studying the role of Human Endogenous Retroviruses (HERVs), ancient DNA sequences, and gut microbiota in children with various inflammatory diseases such as Kawasaki Disease, IgA Vasculitis/Schonlein-Henoch's Purpura, SARS-CoV-2 infection, and febrile virosis. The study aims to understand how HERVs and gut bacteria might influence immune responses and disease symptoms in pediatric patients. This research could provide new insights for managing and treating these conditions. The study involves collecting blood and fecal samples from children diagnosed with the listed diseases. Blood samples are analyzed to study HERVs, while fecal samples are used to characterize the gut microbiota. Patients are enrolled consecutively as they receive a diagnosis, and the study monitors changes over approximately one year to assess associations between HERVs, gut bacteria, and clinical outcomes. Participants will have evaluations including sample collection for laboratory analyses. Researchers will measure HERV activity and gut microbiota profiles to explore their links with disease manifestations. The primary outcomes focus on these analyses throughout the study period, enabling a better understanding of immune regulation in these pediatric inflammatory diseases. The study is expected to last about one year for each participant.

Researchers are evaluating the impact of a machine-learning clinical decision support tool called Kawasaki MATCH on how healthcare providers diagnose and manage patients with Kawasaki Disease (KD) and other febrile illnesses. The study aims to assess the accuracy of Kawasaki MATCH when used at the point of care and its influence on clinical decisions such as additional testing and hospital admissions. Participants' healthcare providers will be randomly assigned to one of two groups: one group will use the Kawasaki MATCH decision support tool to help identify KD by entering patient information including age, laboratory results, and clinical features to generate a risk score; the other group will follow standard care without the decision support tool. This setup will allow researchers to compare usual care with care guided by the Kawasaki MATCH tool. During the study, providers will record assessments and decisions about patients presenting with fever and possible KD. Researchers will track outcomes including the time to treatment for KD patients, hospital admission rates, additional interventions, consultations, and return visits to the emergency department. The study will also gather feedback from providers on the usefulness of the decision support tool. Participation will involve follow-up over 90 days to monitor treatment timing and outcomes.

Researchers are evaluating a dosing regimen of rivaroxaban adjusted for age and bodyweight, equivalent to 15 mg, in Chinese children with giant coronary artery aneurysms following Kawasaki disease. This pilot study focuses on the feasibility of this dosing approach, aiming to provide preliminary data on its use in this population, which is at high risk for coronary artery thrombosis and major cardiovascular events due to their condition. The study builds on previous pharmacokinetic modeling and acknowledges challenges like increased bleeding risk and variability in young patients' development. The study involves administering rivaroxaban as an anticoagulant according to the proposed 15 mg-equivalent dosing regimen adjusted for age and weight. Patients will receive this treatment as part of dual antithrombotic therapy, including antiplatelet drugs, over a period of at least six months. This single-arm, single-center study does not include a comparison group and focuses on monitoring the effects of this dosing strategy in real-world clinical use. Participants will be followed for more than six months with regular assessments to record clinical outcomes such as new coronary artery thrombosis, major bleeding events, and adverse cardiovascular events. Rivaroxaban levels will be measured to evaluate the pharmacokinetic and pharmacodynamic aspects of the dosing regimen. Safety and any dose adjustments will also be evaluated during the study period. The total duration for each participant includes enrollment through six months of treatment and monitoring.

This research aims to evaluate the effectiveness of a machine-learning (ML) model in predicting serious cardiac events within three months among pediatric cardiac inpatients. The study focuses on determining whether the ML model increases palliative care team (PACT) consultations, reduces time to consultation, decreases ICU deaths, and improves documentation of care goals. The study is conducted at The Hospital for Sick Children, leveraging their strong collaboration between cardiology and palliative care. The ML model identifies high-risk cardiology patients daily. If a patient was seen by PACT in the past year, the update goes to the PACT team; otherwise, the cardiology physician in charge receives the alert to decide on consultation. The study compares patient outcomes before and after deploying the ML model, with a pre-deployment period covering 12 months starting 15 months before deployment, and a post-deployment period covering 12 months beginning 3 months after deployment. Participants are pediatric cardiology inpatients. Researchers will monitor PACT consultations within three months of admission, time to consultation, ICU death rates, and documentation of care goals. Outcomes among identified high-risk patients will be compared between the pre- and post-deployment periods to assess the ML model's impact on care processes and patient outcomes.

Researchers are investigating how certain drugs prescribed to children and young adults act within their bodies. The goal is to find the safest and most effective doses for these understudied drugs given as part of standard medical care. This observational study focuses on pharmacokinetics (PK), including how drugs are absorbed, distributed, metabolized, and eliminated, with special attention to participants with conditions such as COVID-19 and Down Syndrome. Participants include children and young adults under 21 years who are either prescribed drugs of interest by their healthcare providers or have tested positive for SARS-CoV-2 within 60 days before enrollment. The study collects bodily fluid samples, like whole blood and effluent samples, to analyze drug behavior. Data collection lasts up to 90 days from consent, extended to 210 days for participants with Down Syndrome at designated sites. During the study, participants undergo PK sampling to measure various drug-related factors such as clearance, volume of distribution, elimination rate, half-life, absorption rate, maximum concentration, and time to reach maximum concentration. Researchers gather data at intervals up to 90 or 210 days, depending on participant status. The study monitors safety and drug effects without altering prescribed treatments, aiming to better understand drug profiles in pediatric and young adult populations.