Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease or Beta-Thalassemia and Falling Donor Myeloid Chimerism Levels
National Heart, Lung, and Blood Institute (NHLBI)
Summary
Background: Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some donor cells in their body. Objective: To cure people s sickle cell disease by giving a second treatment that makes more room in their bone marrow for donor cells. Eligibility: People ages 4 and older with sickle cell disease who had a transplant but the disease returned, and their donor relatives. Donors can be 2 years of age or older. Design: Participants will be screened with medical history, physical exam, and blood tests. Recipients will also be screened with heart and breathing tests, x-rays, a bone marrow sample, and teeth and eye exams. They must have a caregiver. Donors will have 7-8 visits. They will take a drug for 5-6 days to prepare them for the donation. For the donation, blood is taken from a vein in the arm or groin. The stem cells are collected. The rest of the blood is returned. This may be repeated. Recipients will get a long IV line in their arm or chest for about 1-2 months. They will take drugs to help their body accept the donor cells. They will get the donor cells and red blood cell transfusions through the line. They will stay in the hospital about 30 days after the transfusion of donor cells. In first 3 months after the infusion, recipients will have many visits. Then they will have visits every 6 months to 1 year for 5 years. During those visits they will repeat some of the screening tests....
Description
Nonmyeloablative allogeneic peripheral blood stem cell (PBSC) transplants are currently being investigated in phase I/II trials assessing engraftment, efficacy, and toxicity at a number of transplant centers. Our ongoing protocol for patients with severe congenital anemias, particularly sickle cell disease (SCD), and an HLA-matched sibling donor has had excellent preliminary results. None of the patients who engrafted had sickle-related events or any evidence of graft versus host disease (GVHD). There was no significant toxicity associated with the conditioning regimen. An additional protocol…
Eligibility
- Age range
- 2–80 years
- Sex
- All
- Healthy volunteers
- Yes
* INCLUSION CRITERIA: Inclusion criteria- recipient 1. Patient with history of SCD or beta thalassemia who previously underwent allogeneic hematopoietic stem cell transplantation (HSCT) 2. Patient with recurrent SCD defined as HbS greater than or equal to 50% for donors with sickle cell trait and greater than or equal to 10% for donors with HbAA with recurrent clinical manifestations (for example but not limited to recurrent painful crises, acute chest syndrome, priapism, or severe anemia) or patients with recurrent beta thalassemia defined as clinical manifestations such as transfusion-depe…
Interventions
- DeviceCliniMACS CD34 Reagent
Haploidentical recipients will receive CD34-selected cells using Miltenyi CliniMACS(R) CD34+ cell selection kits. The target CD34+ cell dose is at least 10 x 106/kg, and the minimum CD34+ cell dose is 5 x 106/kg. All of the cells collected during the apheresis procedure will be given. The cells will be cryopreserved and stored until the day of transplant.
Location
- National Institutes of Health Clinical CenterBethesda, Maryland