Regenerative Medicine to Restore Normal Hematopoiesis in Aplastic Anemia
Johns Hopkins Kimmel Cancer Center in Baltimore
This research is being done to find out how to increase the effectiveness of a mini bone marrow transplant (BMT) from a relative or unrelated donor. Mini-transplants rely on drugs that suppress the immune system to allow the donors marrow to grow, or engraft, inside your body, however these drugs often carry risk of negative side effects. The main goal of this study is to learn whether people with previously untreated severe aplastic anemia (SAA) can safely receive a BMT.
Confirmed diagnosis of SAA with no prior treatment. Good physical condition with good lung, heart, and kidney functions. Donor must be identified, approved to donate, and willing to participate. **NOTE: Relatives who have donated blood for tranfusions to the patient in the past will not be considered eligible donors.
If you are found to be eligible, and you choose to join this study, a large intravenous catheter will be surgically placed to help with the administration of the medications (chemotherapy agents, antibiotics, blood transfusions) as well as obtaining blood samples. Prior to the BMT, you will be given several days of chemotherapy and one day of radiation. On the day after radiation, the bone marrow will be taken from your donor and given to you like a blood transfusion. Two more doses of cyclophosphamide will be given, one on the 3rd day after the marrow infusion and one on the 4th day after. On the 5th day after transplant you will begin tacrolimus. Other medications will be given after the marrow infusion (by vein and by mouth) to help prevent side effects of the BMT (such as infections, and to help your body from rejecting the donors marrow). During the follow up time, many blood samples and physician exams will be done. Most of the time, the chemotherapy, radiation, marrow infusion, and follow up period are done on an outpatient basis.
03/05/2019 05:03 AM