For children and young adults who suffer from a rare and debilitating disorder called neonatal-onset multisystem inflammatory disease (NOMID), a drug called anakinra brings marked improvement both in symptoms and the inflammation underlying the disease, a new study shows. The study, published in the August 10 issue of the New England Journal of Medicine, was conducted in the Intramural Research Program of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), a component of the National Institutes of Health.
NOMID, also known as chronic infantile neurologic cutaneous articular (CINCA) syndrome, is an inflammatory disorder that affects numerous organs and body systems, including the skin, joints, eyes and central nervous system. For most children, the first sign of the disease is a rash that develops within the first six weeks of life. Other problems, including fever, meningitis, joint damage, vision and hearing loss, and mental retardation, can follow. NOMID is one of a group of illnesses that NIAMS Clinical Director Daniel Kastner M.D., Ph.D. has designated autoinflammatory diseases, because of their seemingly unprovoked episodes of inflammation. Despite treatment to control the inflammation -- including high-dose corticosteroids, disease-modifying antirheumatic drugs such as methotrexate, and nonsteroidal anti-inflammatory drugs such as ibuprofen or naproxen -- the disease is progressive and often fatal. As many as 20 percent of children with NOMID don't survive to adulthood.
"This research shows the importance of studying rare but enormously instructive diseases, a unique strength of the NIH intramural science program," says NIH Director Elias A. Zerhouni, M.D. "This study provides new insights on fundamental mechanisms of inflammation. More importantly this new therapy will reduce the pain and suffering of these young patients allowing them to live a fuller life than previously possible."
Contact: Dr. Janet Austin
NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases