Stem Cell Transplant for Sickle Cell Anemia
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores a new method for treating sickle cell disease and Beta-thalassemia using stem cell transplants. Researchers aim to determine if a half-matched donor with less intense treatment—avoiding heavy chemotherapy and using low-dose radiation and immunosuppressant drugs—can be effective and safe. The trial compares different doses of the immunosuppressant drug cyclophosphamide to assess how well they prevent donor cell rejection. Suitable participants are those over 18 with sickle cell disease or Beta-thalassemia who have a family member as a half-match for stem cell donation. As a Phase 1/Phase 2 trial, this research focuses on understanding the treatment's effects in people and measuring its effectiveness in an initial, smaller group.
Do I need to stop my current medications for the trial?
The trial protocol does not specify if you need to stop your current medications. However, since the trial involves immunosuppressant therapy and other treatments, it's best to discuss your current medications with the trial team.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that Peripheral Blood Stem Cell (PBSC) transplants for sickle cell disease are generally safe. Studies indicate that using half-matched donors is well-tolerated and leads to good outcomes. For instance, a review found a high survival rate of 94% in patients with sickle cell disease who received these transplants.
Early findings suggest that collecting these stem cells is safe and well-tolerated by donors. Additionally, the preparation for the transplant did not cause severe side effects in patients.
Regarding cyclophosphamide, a drug used to suppress the immune system after the transplant, research shows it is safe. It helps lower the risk of graft-versus-host disease, where donor cells attack the recipient's body. This method has proven effective and safe.
Overall, past studies have found these treatments to be generally safe, with manageable side effects and promising results.12345Why are researchers excited about this trial's treatments?
Researchers are excited about these treatments for sickle cell anemia because they explore the potential of PBSC (Peripheral Blood Stem Cell) transplants, which could offer a more direct way to address the root cause of the disease. Unlike conventional treatments like hydroxyurea or blood transfusions, which primarily focus on managing symptoms, PBSC transplants aim to replace the faulty blood-forming stem cells with healthy ones. This trial examines different approaches to post-transplant care, including varying doses of cyclophosphamide, which could help reduce the risk of complications and improve success rates. By potentially offering a more effective and long-term solution, these treatments could significantly improve the quality of life for people with sickle cell anemia.
What evidence suggests that this trial's treatments could be effective for sickle cell anemia?
Research has shown that stem cell transplants can effectively treat sickle cell disease. In one study, 94% of patients survived after receiving this type of transplant. Another study found that using partially matched donors with a special preparation worked as well as using fully matched donors.
In this trial, participants will receive a PBSC transplant, with some cohorts receiving different dosages of post-transplant cyclophosphamide (PT-Cy). Cyclophosphamide, a drug that prevents the body from rejecting the transplant, has improved results. It helps the transplant take hold and reduces the risk of graft-versus-host disease, where donor cells attack the recipient's body. This suggests that combining stem cell transplants with cyclophosphamide may effectively treat sickle cell disease.12356Who Is on the Research Team?
Courtney D Fitzhugh, M.D.
Principal Investigator
National Heart, Lung, and Blood Institute (NHLBI)
Are You a Good Fit for This Trial?
Adults diagnosed with severe congenital anemias like sickle cell disease or Beta-thalassemia, who have a half-matched family donor. Candidates should be at high risk due to organ damage or complications not improved by other treatments. They must understand and consent to the trial, not be pregnant, and meet specific health criteria.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Pre-Transplant Conditioning
Recipients receive low-dose radiation and immunosuppressant therapy with alemtuzumab and sirolimus before the transplant.
Transplantation
Recipients receive donor stem cells through a central line, followed by blood transfusions and antibiotics as necessary.
Post-Transplant Monitoring
Physical and laboratory examinations occur weekly or twice weekly to monitor engraftment and manage complications.
Follow-up
Participants are monitored every 6 months for 5 years to assess long-term outcomes such as GVHD and survival.
What Are the Treatments Tested in This Trial?
Interventions
- Alemtuzumab
- Cyclophosphamide
- Low Dose Irradiation
- PBSC Transplant
- Sirolimus
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Heart, Lung, and Blood Institute (NHLBI)
Lead Sponsor