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
This public-private partnership seeks to develop new ways of identifying and validating promising biological targets for diagnostics and drug development.
What is Sjögren’s syndrome? Sjögren’s syndrome is a chronic (long-lasting) disorder that happens when the immune system attacks the glands that make moisture in the eyes, mouth, and other parts of the body. The main symptoms are dry eyes and mouth, but the disorder may affect other parts of the body. Many people with Sjogren’s syndrome say they feel tired often (fatigue). They also may have joint and muscle pain. In addition, the disease can damage the lungs, kidneys, and nervous system.
What is systemic lupus erythematosus (lupus)? Systemic lupus erythematosus (lupus) is a chronic (long-lasting) autoimmune disease that can affect many parts of the body, including the: Skin. Joints. Heart. Lungs. Kidneys. Brain. Lupus happens when the immune system, which normally helps protect the body from infection and disease, attacks its own tissues. This attack causes inflammation and, in some cases, permanent tissue damage. If you have lupus, you may have times of illness (flares) and times of wellness (remission). Lupus flares can be mild to serious, and they do not follow a pattern. However, with treatment, many people with lupus
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 scleroderma? Scleroderma is an autoimmune connective tissue and rheumatic disease that causes inflammation in the skin and other areas of the body. This inflammation leads to patches of tight, hard skin. Scleroderma involves many systems in your body. A connective tissue disease is one that affects tissues such as skin, tendons, and cartilage. There are two major types of scleroderma: Localized scleroderma only affects the skin and the structures directly under the skin. Systemic scleroderma, also called systemic sclerosis, affects many systems in the body. This is the more serious type of scleroderma and can damage your blood
What are autoimmune diseases? Autoimmune diseases refer to problems with the immune system, which usually fights off viruses, bacteria, and infection. The problem causes your immune cells to attack your body by mistake. These diseases can affect almost any part of the body.
What is rheumatoid arthritis? Rheumatoid arthritis (RA) is a chronic (long-lasting) disease that mostly affects joints, such as the wrist, hands, feet, spine, knees, and jaw. In joints, RA causes inflammation that leads to: Pain. Swelling Stiffness. Loss of function. Rheumatoid arthritis is an autoimmune disorder because the immune system attacks the healthy joint tissues. Normally, the immune system helps protect the body from infection and disease. RA may cause you to feel unusually tired, to have occasional fevers, and to have a loss of appetite. It also may cause other medical problems in the heart, lungs, blood, nerves, eyes
What are polymyalgia rheumatica and giant cell arteritis? Polymyalgia rheumatica and giant cell arteritis are closely linked inflammatory disorders. Some people have one of the disorders while others develop both of them. Polymyalgia rheumatica causes muscle pain and stiffness in the shoulders, upper arms, hip area, and sometimes the neck. Giant cell arteritis causes inflammation of arteries, especially those on each side of the head, scalp, and the aorta (the large artery that carries blood from the heart) and its main branches. The main symptoms of this disorder are: Headaches. Scalp tenderness. Jaw pain. Problems with your eyes, which may