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Executive Summary

I.V. IMMUNOGLOBULIN/ASPIRIN FOR KAWASAKI SYNDROME SHOULD BE "STANDARD" treatment, participants in a National Institutes of Health consensus development conference recommended May 23. A childhood condition of unknown cause, Kawasaki syndrome is characterized by acute high fever accompanied by rash, conjunctivitis, changes in the mucus membrane of the upper respiratory tract, and edema and skin scaling at the extremities. The long-term concern is coronary artery damage. Currently, six intravenous immunoglobulin products are available in the U.S., but none carry labeling for Kawasaki's syndrome. Cutter Labs (Gammimune), Sandoz (Sandoglobulin), Travenol (Gammagard), Immuno AG (Iveegam), Alpha Therapeutics (Venoglobulin-I), Armour (Perimmun) and the American Red Cross are the U.S. suppliers of I.V. immunoglobulin, which is made by fractionating human plasma. All of the products are approved for primary immunodeficiencies, five for idiopathic thrombocytopenia (a bleeding disorder characterized by increased platelet destruction due mostly to autoantibodies against platelet glycoproteins), and one for chronic lymphocytic leukemia, according to the panel. The NIH consensus conference group agreed that studies of I.V. immunoglobulin 400 mg/kg [of body weight] given daily over four days for Kawasaki's "indicate a prompt anti-inflammatory response in the acute phase and a significant decrease in the formation of coronary aneurysms compared with low/moderate aspirin administration regimens." In addition, the panel noted that a more recently tested dosage schedule "of 2 g/kg as a single administration has been shown to be at least as effective as a the four-dose schedule." The panel estimated that the current incidence of Kawasaki's syndrome in the U.S. is roughly 3,000 to 5,000 cases per year. However, the group acknowledged that Kawasaki's is probably underreported and that the size of the patient population will likely increase as more physicians become familiar with diagnosing the disease. Panel Chairman Peter Lipsky, MD, University of Texas Southwestern Medical Center, estimated the cost of the currently available immunoglobulin products at $20 to $40 per gram, putting total treatment costs for patients with chronic conditions at $10,000-$20,000 per year. The panel also suggested that it is "reasonable" to use I.V. immunoglobulin in pediatric HIV-infected patients "given the success of I.V. IG in primary immunodeficiencies" and several small uncontrolled studies suggesting that immunoglobulin is effective in "decreasing common bacterial pathogens, as well as measles." However, the panel declined to take a more definitive position on use in pediatric AIDS, citing the absence of controlled trials. NIH is sponsoring two large-scale prospectively randomized trials. One, sponsored by the National Institute of Child Health and Human Development and consponsored by Cutter Labs, involves more than 350 children in 25 medical centers. The study's hypothesis is that 400 mg/kg of I.V. immunoglobulin administered every 28 days will significantly reduce bacterial infections and death. The study participants will be permitted to receive antiretroviral treatment. The second trial is a National Institute of Allergy and Infectious Diseases study of AZT with and without I.V. IG. Among other recommendations, the panel concluded that I.V. IG combined with ganciclovir "is useful" for treating interstitial pneumonia in bone marrow transplant recipients, and may be protective against septicemia and local infections for these patients. In addition, the panel suggested that chronic inflammatory demyelinating polyneuropathies, Guillain-Barre syndrome, and certain childhood intractable seizure disorders also warrant clinical study.

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