Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP
Status:
Terminated
Trial end date:
2012-07-01
Target enrollment:
Participant gender:
Summary
Patients are being asked to participate in this study because they have severe sickle cell
anemia (SCD) with or without the beta thalassemia trait. Sickle cell anemia is an illness
where the red blood cells change shape and can clog up blood vessels. This keeps the body
from getting the oxygen it needs. Thalassemia is when the body does not make enough
hemoglobin, something that helps the oxygen get to the places it needs to go in the body. The
patient may or may not need to get regular blood transfusions (getting more blood) to improve
their quality of life (feel better) and prevent organ damage (problems with the brain, heart,
lung, kidney, and gonad, for example.). The transfusions can also cause problems, including
iron overload (too much iron in the blood), which can be fatal (patients can die) without
regular deferoxamine shots. Even with the best usual treatments, people with thalassemia or
SCD die sooner. There is no proven cure.
We would like to treat patients using bone marrow transplantation, a treatment that has been
used for people with SCD. The transplant uses healthy "matched" bone marrow. This comes from
a brother or sister who does not have sickle cell disease or severe thalassemia. If the
treatment works, the sickle cell disease or thalassemia may be cured. This treatment has been
used to treat patients with sickle cell disease or thalassemia. It has worked in most cases.
We hope, but cannot promise, that the transplanted marrow will make healthy cells, and
patients will not have sickle cell disease or severe thalassemia anymore.
We do not know what effect this treatment will have on the damage that has already been done
by the disease. Finding that out is the main reason for this study. Currently, very little
has been reported about organ function after bone marrow transplants in patients with sickle
cell anemia.
Phase:
N/A
Details
Lead Sponsor:
Baylor College of Medicine Tami D. John
Collaborators:
Baylor College of Medicine Texas Children's Hospital The Methodist Hospital Research Institute The Methodist Hospital System