New sickle cell unrelated transplant trial

Children's National Medical Center immunologist and blood and marrow transplant physician Naynesh Kamani, MD, will serve as the study co-chair for a new national clinical trial of unrelated donor marrow and umbilical cord blood transplants for severe sickle cell disease.

Children's National will join more than 20 other medical institutions in the first-ever Phase II clinical trial of this treatment. If successful, the Sickle Cell Unrelated Transplant (SCURT) trial has the potential to extend a promising and possibly curative treatment option to more severely affected patients. Sickle cell disease affects approximately 70,000 people in the United States.

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.