Condition: Severe Aplastic Anemia · Sponsor: Boston Children's Hospital
Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow transplant. Regular treatment for patients with aplastic anemia who have a matched sibling (brother or sister), or family donor is a bone marrow transplant. Patients without a matched family donor normally are treated with immune suppressive therapy (IST). Match unrelated donor (URD) bone marrow transplant (BMT) is used as a secondary treatment in patients who did not get better with IST, had their disease come back, or a new worse disease replaced it (like leukemia). This trial will compare time from randomization to failure of treatment or death from any cause of IST versus URD BMT when used as initial therapy to treat SAA. The trial will also assess whether health-related quality of life and early markers of fertility differ between those randomized to URD BMT or IST, as well as assess the presence of marrow failure-related genes and presence of gene mutations associated with MDS or leukemia and the change in gene signatures after treatment in both study arms. This study treatment does not include any investigational drugs. The medicines and procedures in this study are standard for treatment of SAA.
This description comes directly from the study's public registry record.
No contact information is available for this study as per the public registry record.
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| University of Alabama at Birmingham | Birmingham, Alabama, United States | — |
| Phoenix Children's Hospital | Phoenix, Arizona, United States | — |
| Arkansas | Little Rock, Arkansas, United States | — |
| Loma Linda | Loma Linda, California, United States | — |
| Children's Hospital Los Angeles | Los Angeles, California, United States | — |
| UCLA | Los Angeles, California, United States | — |
| Children's Hospital & Research Center Oakland | Oakland, California, United States | — |
| Children's Hospital of Orange County | Orange, California, United States | — |
| Stanford | Palo Alto, California, United States | — |
| Rady Children's Hospital San Diego | San Diego, California, United States | — |
| University of California San Francisco | San Francisco, California, United States | — |
| Children's Hospital Colorado | Aurora, Colorado, United States | — |
+ 40 more locations — full list on the registry record.
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