A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched and HLA-Matched Bone Marrow for Patients with Sickle Cell Anemia and Other Hemoglobinopathies
This research is being done to find out how effective a ?mini? Bone Marrow Transplant (BMT), from a relative who is not a perfect tissue match, is in treating diseases like Sickle Cell Anemia. Mini-transplants rely on drugs that suppress the immune system to allow the donor?s marrow to grow, or engraft, inside your body. We have found that using cytoxan after the BMT allow the cells from the donor to grow in patients' bodies. We will be studying the side effects of this treatment and hope to better understand how well mini-transplants work when done this way for Sickle Cell Anemia.
- Patients who are not eligible for a bone marrow transplant because of not having a sister or brother whose blood cells match - Age 2 to 70 years old - Good physical condition with good lung, heart, and kidney functions - Donor must be willing to participate and be approved to donate - Eligible diagnoses: Most patients with sickle cell anemia who have a severe risk feature - Patients who have received a blood transfusion from the donor will not be eligible
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. You will be given five days of chemotherapy (fludarabine) through the catheter and one day of radiation. You will also receive 2 days of cyclophosphamide through the catheter on the first 2 days for fludarabine. 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 of the 3rd day after the marrow infusion and one on the 4th day after. 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 donor?s 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 is done on an outpatient basis. It is required that you and a caregiver stay near Johns Hopkins Hospital throughout the whole process which is about 2-3 months. You will be discharged back to your local oncologist after the 60 day post transplant evaluation. Additional long term follow up at Hopkins will take place around 6 months post transplant and yearly for 5 years.
07/22/2014 04:02 AM