Bone Marrow Transplant

Hemoglobinopathies

Hemoglobinopathies are a group of blood diseases in which patients inherit a problem producing an essential protein called hemoglobin, which is found in red blood cells. Hemoglobin carries oxygen around our body.

Patients with hemoglobinopathies like sickle cell anemia and beta thalassemia have abnormal hemoglobin, making their red blood cells look and behave differently. Patients with sickle cell anemia and beta thalassemia require carefully monitored care such as chronic transfusion therapy. Our comprehensive Sickle Cell Center is a national leader in caring for children with these kinds of disorders.

Hematopoietic Stem Cell Transplantation

Some patients with sickle cell anemia or beta thalassemia may benefit from allogeneic (donor) stem cell transplantation. These are usually children who have had a complicated disease course that has not responded to other conventional non-transplant forms of therapy. Their complications may include recurrent episodes of stroke, worsening lung problems (like acute chest syndrome) or multiple hospitalizations for pain.

Sickle Cell Center hematologists work closely with members of the Bone Marrow Transplantation team to determine which patients might benefit from a hematopoietic stem cell transplant. Families then begin a discussion to learn about the risks of transplant.

Our team is working hard to develop new approaches to hemoglobinopathy patients who come to transplant as a curative option. Some of these endeavors have included new and exciting approaches at preventing graft-versus-host disease, an unwanted side effect of transplant.

Your bone marrow transplant attending doctor will determine if you are suitable for any available clinical or experimental trials.