Pitt / UPMC researchers will conduct a large clinical trial to test the exchange of red blood cells in sickle cell disease

Advances in medicine have significantly extended the life expectancy of sickle cell patients, but as patients live longer, they suffer more and more organ damage leading to premature death. Researchers from the University of Pittsburgh and UPMC, supported by a $ 19.2 million grant from the National Institutes of Health, will conduct the largest clinical trial of its kind aimed at testing a technique called Exchange transfusion of red blood cells to prolong life and slow down or reverse organic lesions.

At present, there is no standard of treatment for patients with sickle cell disease who are at high risk for organic lesions. We are proud to lead this collaborative effort among the leading centers of excellence to address an important open-ended question about how we treat and manage the disease. "

Mark Gladwin, M.D., Professor Jack D. Myers and Chair of Medicine at the Pitt's School of Medicine and Director of the Pittsburgh Institute of Cardiac, Pulmonary, Blood and Vascular Medicine

Nationwide, about 100,000 people live with sickle cell disease, a genetic disorder that disproportionately affects people of African descent. About 30% of MSC patients develop severe organ damage, such as cardiopulmonary complications, renal or hepatic impairment, with risks gradually increasing with age.

Although blood transfusions are commonly used to treat acute complications of MSC, they can lead to iron toxicity because of the excessive presence of red blood cells. In red cell exchange transfusion, sickle-cell red cells are removed and replaced with normal red blood cells. However, the exchange process is longer, requires more donor blood and costs more than standard blood transfusions.

"Due to the lack of data, doctors today must decide whether they should administer an exchange of red blood cells to their patients," said co-principal investigator Darrell Triulzi, MD, professor of pathology and Director of the Department of Transfusion Medicine. at the Pitt School of Medicine. "Physicians' opinions on the effectiveness of red blood cell exchange in the prevention or reversal of organ lesions are also shared, suggesting the real need for a clinical trial definitive such as this one to determine whether the exchange of red blood cells should be added to the standard treatment of these patients. "

In the new trial, called the Sickle Cell Disease and Cardiovascular Risk Red Cell Exchange (SCD-SQUARE) trial, patients will randomly receive the basic treatments alone or in combination with monthly treatments. exchange of red blood cells for one year. . The objective of the study is to determine whether red blood cell exchange can reduce the number of deaths, reduce the number of hospitalizations, and slow or reverse the development of major organic lesions.

The Pitt Graduate School of Public Health will serve as the data coordination center for the test. The lead researcher, Maria Mori Brooks, Ph.D., professor of epidemiology, will lead the development and administration of data collection and statistical badysis.

In designing the protocol and designing the study, Pitt's researchers collaborated with researchers at the University of Illinois at Chicago (UIC). The SCD-CARRE trial is expected to last seven years and bring together 150 adult patients in 22 centers. The trial will begin with the enrollment of patients in eight "avant-garde" clinical sites, international centers of excellence in the comprehensive treatment and research of sickle cell disease, within the next 12 years month. In addition to Pitt / UPMC and UIC, vanguard sites include Duke University, Emory University, UCSF Benioff Children's Hospital, the University of New York, and the University of California. Alabama in Birmingham, Imperial College London, London, UK, and Henri Mondor Hospital in Paris, France.