Blood and Marrow Transplant for Sickle Cell Disease
Blood and marrow transplant (BMT) can cure sickle cell disease. BMT is not a surgery. BMT involves replacing stem cells in the bone marrow with new stem cells that will make healthy red blood cells. Children’s National offers all of the following types of BMT for sickle cell disease:
- Matched sibling donor transplant
- Alternative donor including haplo-identical (parent) transplant
- Gene therapy including CASGEVY™ and LYFGENIA™
Contact Information
For more information about BMT for sickle cell disease, please call us.
Stem Cell Transplant Timeline
We’ve created a step-by-step guide to give you an in-depth look at the transplant process: from diagnosis to treatment to long-term care.
How Blood and Marrow Transplant Works
This video provides an introduction to blood and marrow transplantation (BMT), including gene therapy, for families with sickle cell disease.
Choosing the Children’s National BMT Program for Your Care
The BMT Program for Sickle Cell Disease is one of the largest BMT centers for sickle cell disease and has been performing BMT for sickle cell disease for over 20 years. In fact, more than 150 children and adolescents with sickle cell disease have received transplants through our renowned program.
A Multidisciplinary Approach to Care
Our patients benefit from a highly specialized team with experience performing BMT for sickle cell disease. This team is a collaboration between our hematologists and blood and marrow transplant specialists. Patients receive comprehensive care from experts in heart (cardiologists), lung (pulmonologists), brain (neurologists), growth and development (endocrinologists) and emotional health (psychologists), who care for patients before, during, and years after BMT for sickle cell disease. In addition, given the potential impact of BMT on fertility, our pediatric gynecology and urology programs offer fertility preservation consultations and services.
Innovative Research on Transplant for Sickle Cell Disease
At Children's National, we lead research to advance curative therapy for sickle cell disease. Members of our team have published many articles on transplant for sickle cell disease. We continue to pioneer transplant therapies to cure sickle cell disease and currently offer participation in the below clinical trials:
- Minimizing Toxicity in HLA-identical Related Donor Transplantation for Children with Sickle Cell Disease (SUN)
- Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell AntibodY (SUN-RAY)
- Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation
- A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease
Demier's Story
Demier, born with sickle cell disease, received a cure – a stem cell transplant. "He's had a second chance at life," his mom says. "He does things he could never do before, playing football with his friends, running, going to the pool. Thanks to Children's National Hospital, he's a regular, active and happy kid."
