About this study
Severe aplastic anemia (SAA) is a rare disease where the body stops making enough new blood cells. The patient’s immune system attacks their bone marrow. This makes it difficult for the bone marrow to make healthy blood cells.
If the patient has a sibling (brother or sister), or other family member whose bone marrow fully matches, they are usually treated with a bone marrow transplant (BMT), which is also known as a hematopoietic cell transplant or HCT. This transplant helps the patient make new, healthy cells.
If no family member is a match, the patient may get a treatment known as immunosuppressive therapy (IST). IST consists of 2 drugs (horse antithymocyte globulin and cyclosporine) that slow the immune system's attack on the bone marrow.
Another treatment approach is a BMT from a matched unrelated donor. This is typically used for patients who do not respond to IST or have disease relapse. But in recent years we have learned that BMT from matched unrelated donors given as upfront therapy offers excellent cure rates.
This clinical trial studies SAA patients who have no matched sibling donors. Some patients will be chosen at random to receive IST as their first treatment. The other group will get a matched unrelated donor HCT as their first treatment. Our aim is to see how well these treatments work and which gives fewer side effects.
Scientists will follow study participants for 5 years from the time they start the trial. Patients will have medical visits, lab tests, and be asked about any side effects and their quality of life.
Purpose of this clinical trial
- Find out whether IST or matched unrelated donor BMT leads to better survival and fewer problems
- Help scientists better understand the disease
Eligibility overview
- Severe aplastic anemia
- Up to 25 years old
- Does not have a fully matched sibling donor
- Has at least 2 matched, unrelated donors