One of the greatest frustrations for Sung Won Choi, M.D., M.S., is treating a child's cancer with a bone marrow transplant only for the young patient to become severely ill once more.

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Between 50 to 60 percent of patients who receive donated bone marrow or stem cells develop a major complication called graft-versus-host disease, or GVHD. This occurs when transplanted cells view the recipient's tissues and organs as foreign, prompting them to attack the body — primarily the skin, liver and gastrointestinal tract.

Symptoms can be life-threatening, with a mortality rate of 75 to 100 percent among those with the most severe cases of the disease.

"A bone marrow transplant may cure a patient's underlying cancer or immunodeficiency but then give them an entirely new chronic disease," says Choi, an associate professor of pediatric hematology and oncology at University of Michigan C.S. Mott Children's Hospital.

That unexpected development can be devastating during an already stressful time.

"When I meet patients and families, their biggest focus is eliminating the cancer or disease for which they are receiving the transplant," Choi says. "But then I have to tell them they have a 50 percent or higher chance of replacing their disease with another one. The complication could last weeks or years and is potentially fatal."

That's why Choi has devoted the past decade of her career to research intended not only to better treat GVHD but also to prevent it from occurring in the first place.

Targeting GVHD in advance

Choi is this year's recipient of Hyundai's Hope on Wheels Impact grant, which will support her team's work to detect the disease in patients by identifying new markers of disease before it occurs.

To do so, researchers will monitor patients' blood and stool to help identify signs of the disease, such as temperature changes and concerning heart rates. If the disease can be detected earlier, it's possible to start earlier treatment to prevent the more severe forms of GVHD.

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Additionally, Choi and colleagues have spent years studying how a novel drug called vorinostat may be associated with reduced GVHD. Based on her research findings in adults, her team plans to open up a clinical trial to examine the feasibility and effectiveness of using this therapy in children and adolescents.

Current treatment poses challenges

At present, high-dose steroids are the standard initial treatment for acute GVHD, but only half of patients have a complete response to steroids.

For other patients, despite prompt initiation of steroids, outcomes are often poor, with either no response or a reoccurrence while weaning off steroids.

Even if successful, high-dose steroids are a major source of morbidity and mortality because of increased infections, Choi notes.

"Over the past three decades, we have made enormous strides in our understanding of the development of acute GVHD," she says. "Still, prevention of the disease has remained largely unchanged.

"More effective preventive interventions are urgently needed to improve outcomes for children who undergo a bone marrow transplant. We hope our research helps lead to more successful stem cell transplants that eliminate the cancer without creating the burden of another life-threatening condition."

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