Landmark Sickle Cell Disease Trial To Investigate Unrelated Donor Marrow And Umbilical Cord Blood Transplants

For many years, doctors have used blood or marrow transplants to treat severe cases of sickle cell disease. However these treatments have been available only to patients with a matched family member to donate blood or marrow stem cells. Only 14 percent of severe sickle cell cases have a matched donor within their family. Successful transplants using unrelated donors could create a viable treatment option for more sickle cell patients.

"Blood and marrow transplantation from unrelated donors has been routinely utilized to treat treatment-refractory leukemia and lymphoma for almost two decades," said Dr. Kamani, co-principal investigator of the study. "Preliminary results from the few unrelated donor transplants that have been performed for other non-malignant conditions show great promise for those who suffer a severely diminished quality of life due to sickle cell disease. Participants in this large-scale study will help us understand how to better treat, and possibly even cure, sickle cell disease for more people."

Starting in late August, the clinical trial will enroll 45 patients ages 3 to 16 with severe symptoms of sickle cell disease, including stroke, recurring acute chest syndrome, or frequent pain crises. For all marrow and cord blood transplants, patients must undergo intense chemotherapy to prevent their immune systems from rejecting donor cells. This study will use a reduced-intensity chemotherapy to minimize side effects and increase the likelihood that the donor cells will be accepted by the body. After the transplant, participants will be followed for two years to gauge the effectiveness of the transplant at minimizing the ravages of sickle cell disease.

The trial is facilitated by the Blood and Marrow Transplant Clinical Trials Network, and is funded by the National Heart, Lung, and Blood Institute, the National Cancer Institute, the National Marrow Donor Program, the Sickle Cell Disease Clinical Research Network, and the National Center on Minority Health and Health Disparities.