Bone Marrow Transplantation Offers Hope for Extreme Cases of Sickle Cell Disease

Two-thirds of the patients who receive bone marrow transplants (BMT) at Columbus Children’s Hospital are treated for cancer conditions. The remaining BMT patients undergo the procedure for a variety of genetic disorders, including sickle cell disease. Children’s Hospital has completed three successful BMT for sickle cell disease over the past year. In the United States, there have been approximately 100 cases in which BMT was used for sickle cell disease, and about 150 cases in the world.

Sickle cell disease affects a person’s ability to make normal red blood cells. Their cells form rigid structures that look like sickles and clog up small blood vessels depriving tissues of oxygen. This produces extreme pain and can cause damage to the body’s organs.

BMT works as a cure for sickle cell disease because red blood cells come from mother cells that originate in bone marrow. BMT wipes out the bone marrow a child is born with, and replaces it with a healthy sibling’s who has the same tissue type. After the procedure, normal red blood cells are produced from the new bone marrow and the disease is cured.

Amanda Termuhlen, MD, associate chief of Hematology/Oncology at Columbus Children’s Hospital, and a faculty member at The Ohio State University College of Medicine, explained that BMT is only used for the most severe sufferers of sickle cell because of the risk factors associated with transplantation.

“The procedure itself has about a five percent mortality rate,” Termuhlen said. “Not every child with sickle cell is the same. Some children will have very few problems; some will have chronic and life-threatening problems. Our current approach is to pick children who are severely affected with multiple pain episodes, and are at risk of stroke, or are at risk for dying from sickle cell disease at a very young age.”

Only non-afflicted, full siblings are suitable for transplantation. Less than 19 percent, or one-in-five children with sickle cell, will find a suitable donor.

At Columbus Children’s Hospital, there is new hope for a young girl Kimmi Desir. Kimmi became the first sickle cell BMT recipient at Children’s when she received a transplant from her sister Melissa Desir. Transplants are considered successful if the recipient is healthy a year after the procedure. While Kimmi has passed that mile marker with flying colors, she continues to be monitored by specialists at Children’s Hospital.

“To be able to help those children who are at a higher risk of having complications, and being able to offer a cure for their disease is critical,” Termuhlen said. “It provides a service from toddlers to older teenagers with sickle cell disease.”