Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias
Status:
Recruiting
Trial end date:
2023-04-01
Target enrollment:
Participant gender:
Summary
People with severe congenital anemias, such as sickle cell anemia and beta-thalassemia, have
been cured with bone marrow transplantation (BMT). The procedure, however, is limited to
children younger than the age of 16 because the risks are lower for children than for adults.
The purpose of this study is to explore the use of a BMT regimen that, instead of
chemotherapy, uses a low dose of radiation, combined with two immunosuppressive drugs. This
type BMT procedure is described as nonmyeloablative, meaning that it does not destroy the
patient s bone marrow. It is hoped that this type of BMT will be safe for patients normally
excluded from the procedure because of their age and other reasons.
To participate in this study, patients must be between the ages of 18 and 65 and have a
sibling who is a well-matched stem-cell donor. Beyond the standard BMT protocol, study
participants will undergo additional procedures. The donor will receive G-CSF by injection
for five days; then his or her stem cells will be collected and frozen one month prior to
BMT. Approximately one month later, the patient will be given two immune-suppressing drugs,
Campath 1-H and Sirolimus, as well as a single low dose of total body irradiation and then
the cells from the donor will be infused.
Prior to their participation in this study, patients will undergo the following evaluations:
a physical exam, blood work, breathing tests, heart-function tests, chest and sinus x-rays,
and bone-marrow sampling.