OMRF research could yield more grants

Published: Wednesday, September 13, 2017 By: Sarah Terry-Cobo

The Journal Record

A local researcher and his staff discovered a marker that can improve diagnoses for patients with a rare bleeding disorder.

Dr. Lijun Xia at the Oklahoma Medical Research Foundation said the scientific study won't immediately lead to treatments for patients with low blood platelets. But the advancement could lead to additional national research grants for the nonprofit organization. He has a patent that can be used to advance additional research.

Nancy Potthast, marketing director with the Cleveland, Ohio-based Platelet Disorder Support Association, said any new advancements with the rare condition are important, in part because so few people are aware it exists. About 3.3 adults per 100,000 people are diagnosed each year and at any given time the prevalence of the disorder is about 9.5 adults per 100,000 people.

Platelets are critical in the blood clotting process. Xia, head of the OMRF cardiovascular biology research program, and his staff examined the mechanisms behind immune thrombocytopenic purpura, or ITP. In patients who have ITP, antibodies prematurely destroy platelets, leading to a bleeding disorder.

ITP can be a long-term condition that can reappear after long periods without symptoms, according to the U.S. National Library of Medicine. Treatments generally include autoimmune diseases. Another treatment is removing the spleen, one organ that prematurely destroys the platelets.

But immune suppression and splenectomies are effective for about 66 percent of patients, Xia said.

"That's significant because in a clinical setting, that is one in three patients who we have exhausted all available (treatments), even removal of the spleen, and still have a life-threatening bleeding problem," he said.

His work showed that 30 percent of patients' platelets were removed by the liver instead of by the spleen. They found a type of complex carbohydrates are missing from those patients. That sugar-like coating protects platelets from being destroyed too soon, he said.

That gives researchers a target so they can develop a more precise diagnosis and determine which treatments will work best. Better diagnoses could help some patients avoid an unnecessary splenectomy, he said.

His study was published in June in the Proceedings of the National Academy of Sciences.

Potthast said the disorder creates financial, emotional and physical tolls on patients. Some treatments have not been approved by federal regulators and often aren't covered by insurance plans. People often experience severe fatigue that can't be remedied with more sleep. Doctors recommend patients don't engage in contact sports that could cause injury and trigger a bleeding event. The psychological toll can be overwhelming, she said.

"Most people don't die from ITP; but you can die, you can bleed to death," Potthast said. "So a lot of them have anxiety knowing they could have a brain bleed, and if you get a headache, it may be too late."

Xia said the discovery could lead to additional funding from the National Institutes of Health, which provided two grants and about $2 million for research leading to his most recent publication. He has a patent for engineering a sugar molecule, which could help develop a therapy to block removal of that sugar that prematurely destroys platelets.

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