Sickle cell disease is an inherited blood disorder that affects the red blood cells to the point of changing the shape. Normal red blood cells are smooth, round and flexible, like the letter “O” and can move easily through the vessels in the body. A person with sickle cell disease has red blood cells that are stiff and sticky and “C” shaped, which tend to stick together, making it more difficult to move through the vessels. These clusters form blockages which causes damage throughout the body.

Blood and marrow transplant (BMT) is a treatment for sickle cell disease. BMT is not a surgery, it involves replacing stem cells in the bone marrow with new stem cells that will make healthy red blood cells.

The video below introduces blood and marrow transplantation and discusses the different treatment options such as match sibling donor transplant and gene therapy for patients with sickle cell disease.

Children’s National currently offers 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™

Parents with questions about BMT for treating Sickle Cell Disease should ask their child’s pediatrician or check out Children’s National’s Bone and Marrow Transplant Program.