Haploidentical PBMC Transplant for Severe Congenital Anemias
Status:
Active, not recruiting
Trial end date:
2021-09-10
Target enrollment:
Participant gender:
Summary
Background:
Bone marrow transplantation (BMT), which involves transplanting a donor's marrow stem cells,
is capable of curing some congenital anemias. BMT usually involves high-intensity treatment
with chemotherapy and radiation to kill abnormal cells, which affects all systems of the
body.
People with anemias often have damage to other organs such as the kidneys, which can be
further damaged by the chemotherapy. Only approximately 20 percent of patients have a
full-matched donor, making treatment for many people with anemias unavailable. However, 90
percent of patients may have a half-matched donor, but using a half-matched donor increases
the toxicity of BMT.
Objectives:
To determine if a research BMT with half-matched donor cells, low-intensity radiation,
immunosuppressant drugs, and no chemotherapy will be effective in patients with sickle cell
disease and Beta-thalassemia.
To determine the effectiveness of cyclophosphamide, an immunosuppressant drug, in preventing
rejection of the donor cells.
Eligibility:
Recipients are individuals at least 18 years of age who have been diagnosed with sickle cell
disease and Beta-thalassemia, and who have a family member who is a haploidentical (i.e.,
half match) tissue match.
Donors are healthy individuals between the ages of 2 and 80 who are found to be suitable
donors.
Design:
Donors will undergo apheresis, which involves withdrawing blood from one arm vein, passing it
through a machine that removes bone marrow stem cells, and returning the remaining blood
through the vein in the other arm. Donors will receive a drug that causes the stem cells to
be released into the bloodstream prior to the apheresis procedure.
Recipients will undergo routine physical and laboratory examinations, including bone marrow
sampling at the beginning of the study. After transplantation, physical and laboratory
examinations will occur on a weekly or twice weekly basis at the outpatient clinic.
Recipients will be examined every 6 months starting 100 days posttransplant for 5 years.
Recipients will receive low-dose radiation in two treatments 1 and 2 days before the
transplant. They will also be given immunosuppressant therapy with alemtuzumab and sirolimus.
Another immunosuppressant drug, cyclophosphamide, will be given in the future as needed to
subsets of the recipients to prevent rejection of donor cells.
Recipients will receive the donor stem cells through a previously inserted central line. The
process takes up to 8 hours.
Recipients will receive blood transfusions as necessary to prevent anemia and bleeding during
the posttransplant period. They may also receive intravenous antibiotics to prevent
infection.