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Release: Jan. 10, 2001

UI researcher helps lead study of bone marrow transplantation complication

IOWA CITY, Iowa -- A University of Iowa Health Care researcher is serving as a co-principal investigator for a National Institutes of Health (NIH) grant to study Graft versus Host Disease (GVHD) in children. GVHD is a common and life-threatening complication that can follow bone marrow transplantation.

Frederick Goldman, M.D., UI associate professor of pediatrics and director of the UI Pediatric Bone Marrow Transplant Unit, received $460,000 of the $2.3 million, five-year NIH grant for his component of the study. The study's lead investigator is Andrew Gilman, M.D., of Children's Mercy Hospital in Kansas City, Mo.

The overall study goals are to evaluate the effectiveness of hydroxychloroquine, a new immunosuppressive drug, for treating GVHD and to better understand what causes the disease, Goldman said. More than 100 pediatric bone marrow transplant centers nationwide will participate in the trial, which is sponsored by the federal Children's Oncology Group. The study will ultimately involve more than 300 pediatric patients.

"It will be one of the largest multi-institutional studies related to treating children with GVHD," Goldman said.

Up to 50 percent of children who receive a bone marrow transplantation develop this potentially fatal disease that also can lead to permanent damage of the lungs, skin and liver. Bone marrow transplantation is currently the best treatment available to cure various forms of leukemia and diseases of the bone marrow or immune system in children. More than 1,000 bone marrow transplants are performed on children in the United States every year.

"Graft versus Host Disease leads to immune system abnormalities and susceptibility to infection," said Goldman, who recently contributed to a phase II trial that showed a beneficial effect of hydroxychloroquine in treating the disease.

For the current phase III trial, Goldman will analyze blood samples taken from the hundreds of children enrolled in the study. The data will help the team better understand the origins of GVHD and monitor the effects of the study drug on immune recovery following bone marrow transplantation. The UI also will enroll several pediatric patients in the study.

"We hope that information gained from these studies will improve the outlook for patients with GVHD and someday lead to the development of safer and less toxic strategies to treat GVHD," Goldman said.

University of Iowa Health Care describes the partnership between the UI College of Medicine and the UI Hospitals and Clinics and the patient care, medical education and research programs and services they provide.