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Research shows compound reduces length of painful sickle cell crises

(Embargoed) CHAPEL HILL -- Using a novel compound known as Purified Poloxamer 188 (PP188), U.S. physicians have found a way to shorten the often-excruciating, sometimes days-long painful episodes known as "crises" that sickle cell disease patients suffer.

In a national cooperative study led by Dr. Eugene P. Orringer of the University of North Carolina at Chapel Hill School of Medicine, PP188 was shown to make the painful episodes end sooner than they otherwise would.

"While the effect wasn't profound, it clearly shortened crises," said Orringer, a sickle cell disease expert and executive associate dean of medicine. "The beneficial effect of PP188 was particularly apparent in two specific subgroups of patients. One group was patients who were already taking the drug hydroxyurea, and the other was children."

A report on the findings appears in the Nov. 7 issue of the Journal of the American Medical Association. About 40 U.S. medical centers participated in the research. Dr. Kenneth I. Ataga, instructor in medicine, helped run the project at UNC.

In 1995, Orringer was one of 20 U.S. researchers who carried out a clinical trial that tested hydroxyurea in sickle cell patients. Based on that study, which found a 50 percent reduction in the number of crises, the Food and Drug Administration made hydroxyurea the first therapy approved as a specific treatment for sickle cell disease.

"Results of this new study suggest combining PP188 with hydroxyurea could further help these patients," he said.

As they grow into adulthood, children with sickle cell disease eventually develop a variety of health problems including damage to major organs such as kidneys, liver and lungs.

"If we could reduce the frequency or severity of crises during childhood, we might be able to protect these patients from much of the organ damage that typically appears later in adult life," Orringer said. "That possibility is particularly exciting."
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Contact: David Williamson
david_williamson@unc.edu
919-962-8596
University of North Carolina at Chapel Hill
6-Nov-2001


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