Grant: Project Grant

Vasculitis is a rare disease in children and adults that develops when blood vessels carrying oxygen to critical organs in the body become inflamed. In children with vasculitis, the kidney is one of the organs where the blood vessels are most commonly affected, but it can also affect the lungs, eyes, brain or skin. If organ damage is severe, the child may die or suffer permanent organ damage. Since vasculitis is less common in children than adults, most of what we understand for children with the disease, and how to treat it, comes from experience with adults. Current treatment involves drugs used for cancer patients. These treatments can be life-saving, but they do not prevent the disease from returning and they have serious side effects such as infections, cancer, difficulty having children, and heart disease. Finding the right drug at the right dose for each patient can be a tedious process of stopping and starting treatment, and repeatedly taking a small sample of affected organs with a small surgical procedure (biopsy) - this is not always feasible, especially for children. Doctors treating adults with vasculitis have found that 'markers' in blood or urine may tell them which patients have a higher risk of recurring disease and/or more severe disease in the kidneys. It is not clear if these markers are present in children, and if so, if they work in the same way. While their bodies (and immune system) are still growing, children may respond differently to the disease and treatments compared to adults. Our team created the largest international network of doctors and scientists in the world studying vasculitis in children. In this project, we will use collected information and samples from over 300 pediatric patients to check if the adult markers are present and if they predict risks in children. If so, they will provide doctors with tools to "personalise" treatment for each child.