PEPR Overview The Validation of Pediatric Patient Reported Outcomes in Chronic Diseases (PEPR) Consortium (U19) will capitalize on recent advances in the science of Patient Reported Outcomes (PROs) to assess the health of children with a variety of chronic diseases and conditions in clinical research and care settings. Examples of
Robert Colbert, M.D., Ph.D., leads a team of scientists in the Pediatric Translational Research Branch studying the etiology and pathogenesis of early onset arthritis, including axial disease in juvenile spondyloarthritis.
What is juvenile idiopathic arthritis (JIA)? Juvenile idiopathic arthritis (JIA) is the most common type of chronic, or long-lasting, arthritis that affects children. It happens when the immune system attacks healthy joint tissues. JIA broadly refers to several different chronic (long-lasting) disorders involving inflammation of joints (arthritis), which can cause: Joint pain. Swelling. Warmth. Stiffness. Loss of motion. JIA may last a few months or years, or it may be a lifelong disease.
What is spinal stenosis? Spinal stenosis happens when the spaces in the spine narrow and create pressure on the spinal cord and nerve roots. The spinal cord is a bundle of nerves that comes out of the base of the brain and runs down the center of the spine. The nerve roots branch out from the cord. In spinal stenosis, the narrowing usually occurs over time.
What is epidermolysis bullosa? Epidermolysis bullosa is a group of rare diseases that cause fragile skin that leads to blisters and tearing. Tears, sores, and blisters in the skin happen when something rubs or bumps the skin. They can appear anywhere on the body. In severe cases, blisters may also develop inside the body. The symptoms of the disease usually begin at birth or during infancy and range from mild to severe.
What is fibrous dysplasia? Fibrous dysplasia happens when abnormal fibrous (scar-like) tissue replaces healthy bone. The fibrous tissue weakens the bone over time, which can lead to: Broken bones. Bones that are misshapen (bowed or crooked). The disease can affect any bone in the body. Some people have no symptoms or only a few symptoms. Other people may have more symptoms. Although there is no cure for fibrous dysplasia, treatments may help to lessen pain, and physical therapy may help strengthen muscle and improve movement.
What is Marfan syndrome? Marfan syndrome is a genetic disorder that affects the body’s ability to make healthy connective tissue, which supports the bones, muscles, organs, and tissues in your body. The condition can affect different areas of the body, including: Bones, ligaments, tendons, and cartilage. Organs, such as the heart and lungs. Skin.
A researcher from Stanford University asked IRP investigator Michael Ombrello, M.D., to help her team follow a new lead in the mystery of why some patients with a rare inflammatory condition called Still’s disease were coming down with a life-threatening lung ailment.
Dr. Sikora devotes his time in studying the pathogenesis of pediatric inflammatory disease, including juvenile arthritis and relapsing polychondritis.