Adult Drug Improves Function in Patients with a Rare Pediatric Rheumatic Disease
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Adult Drug Improves Function in Patients with a Rare Pediatric Rheumatic Disease

ATLANTA, GA -- November 14, 2007 -- Anakinra (Kineret), a medication used in the treatment of rheumatoid arthritis in adults, improves physical function in children and young adults with neonatal-onset multisystem inflammatory disease, and may provide a clue about what causes these types of diseases, according to research presented this week at the American College of Rheumatology's Annual Scientific Meeting in Boston, Mass.

NOMID is a rare disorder that is caused by oversecretion of a protein called interleukin (IL)-1beta that leads to inflammation in numerous organs in the body. NOMID is often discovered between birth and 6 weeks of age when newborns develop an unusual rash. Other symptoms include fever, aseptic meningitis, swelling of the optic nerves, joint pain and deformity -- particularly in the knees, hearing and sight loss, mental disability, and stunted growth.

Researchers studied 18 patients between the ages of four and 28 to determine the functional outcomes of one year of treatment with anakinra. Participants received daily injections of anakinra while functional outcomes such as joint range-of-motion, pain, walking and running, motor and process skills, IQ, and disability were monitored.

After 12 months of treatment with anakinra, improvement in functional motor performance and reduction of pain and disability were seen using standardized measurements.

However, the absence of improvement in IQ and other more cognitively related scores suggests that, although motor disability may be partially reversible with the use of anakinra, the drug does lead to improvement in every aspect of the disease.

"We have shown that anakinra treatment has resulted in measurable improvement in daily life function (mobility and self care)," said investigators in the study, Scott M. Paul, MD; physiatrist; rehabilitation medicine department, National Institutes of Health, Clinical Center and Raphaela Goldbach-Mansky, MD, MHS, staff clinician and principal investigator; National Institute of Arthritis and Musculoskeletal and Skin Diseases; National Institutes of Health. "It has been most rewarding to actually see the impact on the subjects' daily lives, most strikingly demonstrated by some of the subjects who were unable to walk and now can -- some even without the assistance of a cane or walker."

SOURCE: American College of Rheumatology

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