ATLANTA – The Sickle Cell Disease Program at the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta has been named the lead coordinating center for a National Institutes of Health (NIH) study to determine the safety and effectiveness of blood and marrow transplants (BMTs) compared to standard care therapies to help treat sickle cell disease in young adult patients.
Lakshmanan Krishnamurti, MD, Director of BMT at Children’s and a Professor in the Department of Pediatrics at Emory University School of Medicine, will lead the Sickle Cell Transplant to Prevent Disease Exacerbation (STRIDE) study with 26 institutions across the country. Dr. Krishnamurti developed the study hoping to bolster medicine’s support of BMTs for young adults, determining its safety and feasibility in treating sickle cell disease.
“Since the 1980s, advances in research and technology have led to improved outcomes for children receiving bone marrow transplants,” Dr. Krishnamurti said. “With pediatric patients responding better than ever before, we want to study the long term effects of BMTs to determine whether they will be as effective in curing young adults of sickle cell disease.”
Dr. Krishnamurti was the first physician in the world to perform a reduced-intensity BMT in a patient with sickle cell disease while at the University of Minnesota in 1999.
More than two decades ago, young patients with severe cases of sickle cell disease received a BMT as researchers searched for a cure. The procedure came with high risks of negative effects and life-threatening complications at the time.
“It resulted in the research community abandoning the idea of transplants for young adults,” Dr. Krishnamurti said. “But today, with more refined procedures and better knowledge, transplants could be a viable option for young adults suffering from severe sickle cell disease.”
Sickle cell disease is estimated to affect up to 100,000 Americans. It is a hereditary blood disorder, which disproportionally affects African Americans and causes a host of acute and chronic conditions, including debilitating pain.
Success will be measured by a patient's event-free survival for at least a year. In addition, Dr. Krishnamurti hopes that the study could show how BMTs help reverse some of the damage caused by sickle cell disease in a patient’s organs.
More importantly, Dr. Krishnamurti said he hopes to see as much success with the young adults as he has with the children. “It’s very exciting to work with the young adult population on this," he said. "And this time, we might be able to offer a cure.”