The sickle cell program at the Aflac Cancer & Blood Disorders Center of Children’s Healthcare of Atlanta has been named the lead coordinating center for a National Institute of Health study to determine the safety and effectiveness of bone marrow transplants compared to standard care therapies to cure sickle cell disease in young adult patients.
Dr. Lakshmanan Krishnamurti, director of the Blood and Marrow Transplant Program at Children’s, will lead the STRIDE study (Sickle Cell Transplant to Prevent Disease Exacerbation) with 26 institutions around 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 1980’s, advances in research and technology have led to improved outcomes for children receiving bone marrow transplants,” said Dr. Krishnamurti. “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 bone marrow transplant 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 bone marrow transplants (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,” said Dr. Krishnamurti. “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 patients’ “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 says 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. And this time, we might be able to offer a cure,” he said.