Clinical Alert: Enbrel® Shown Safe, Effective in Children and Teenagers with Juvenile Rheumatoid Arthritis
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
17 March 2000
Enbrel® (etanercept) has been shown to be a safe and effective drug in the treatment of children and teenagers with polyarticular juvenile rheumatoid arthritis (JRA), according to clinical trial results reported in the New England Journal of Medicine.
In this clinical trial, 69 children, ages 4 to 17, were injected with Enbrel® twice a week; 74 percent responded with measurable improvement when treated for three months. At the end of three months of treatment, on average, there was a 56 percent decrease in the number of joints with active arthritis, a 75 percent decrease in the amount of joint stiffness and a 63 percent decrease in the amount of joint pain. All measures of arthritis impact — symptoms, joint abnormalities, ability to perform daily functions and laboratory tests — were dramatically improved. The drug was well tolerated.
The trial was coordinated at the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) Multipurpose Arthritis and Musculoskeletal Diseases Center at the Children's Hospital Medical Center of Cincinnati, Ohio. It was performed by investigators in the Pediatric Rheumatology Collaborative Study Group. Its success is the culmination of many years of basic research supported by the NIAMS and other NIH components.
"These findings show a significant — often profound — improvement for most children with JRA when treated with Enbrel® compared to placebo," said Daniel J. Lovell, M.D., M.P.H., principal investigator and lead author. "Before Enbrel®, many children with severe JRA had a poor response to existing treatment options. Often, they would have to stop attending school. Now, there is hope for these children."
NIAMS Director Stephen I. Katz, M.D., Ph.D., stated, "Juvenile rheumatoid arthritis can be a devastating disease, not only to the children who have it, but to their families as well. We are pleased to have supported the basic research behind this drug that relieves so much pain and reduces the amount of joint destruction."
Enbrel® belongs to a new class of drug treatments called "biologic agents" that are designed to interfere with the specific biological process of a disease. Enbrel® is a tumor necrosis factor (TNF) antagonist, a substance that blocks the action of TNF, a naturally occurring protein in the body that helps cause inflammation. It acts as a "sponge" to absorb TNF.
The clinical trial adhered to the Food and Drug Administration's "Pediatric Rule," which requires manufacturers of new drugs and biologic agents that will be commonly used for children to provide specific information about safe pediatric use.
JRA is a type of arthritis that causes joint inflammation and stiffness for more than six weeks, beginning when the child is 16 years of age or less. Inflammation causes redness, swelling, warmth and soreness in the joints, although many children with JRA do not complain of joint pain. Any joint can be affected and inflammation may limit the mobility of affected joints. There are three types of JRA: polyarticular (affecting five or more joints), pauciarticular (affecting four or fewer joints) and systemic, also called Still's disease (joint swelling, fever, rash, and organ involvement).
For more information on juvenile rheumatoid arthritis, and for a fact sheet on arthritis drugs, call 1-877-22-NIAMS, or visit the NIAMS web site at http://www.nih.gov/niams/healthinfo. The American Juvenile Arthritis Organization, at http://www.arthritis.org/, can also provide information.
The mission of the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) is to support research into the causes, treatment and prevention of arthritis and musculoskeletal and skin diseases, the training of basic and clinical scientists to carry out this research, and the dissemination of information on research progress in these diseases. For more information about NIAMS, call our information clearinghouse at 1-877-22-NIAMS or visit the NIAMS web site at http://www.nih.gov/niams.
Enbrel® (etanercept) is a trademark of Immunex Corporation, which markets Enbrel® in North America with Wyeth-Ayerst, a division of American Home Products Corporation. Manufactured by Immunex, Seattle, Wash.
To interview Dr. Daniel Lovell, contact Jim Feuer, media relations manager, Children's Hospital Medical Center of Cincinnati at 513-636-4656.
Lovell D, Giannini, E, Reiff A, Cawkwell G, Silverman E, Nocton J, Stein L, Gedalia A, Ilowite N, Wallace C, Whitmore J, Finck B. Etanercept in children with polyarticular juvenile rheumatoid arthritis. NEJM 2000;342:763-9.