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Aplastic Anemia Research Studies


Stem Cell Transplant for Patients with Aplastic Anemia - Detailed Protocol Description

If you have been diagnosed with aplastic anemia or paroxysmal nocturnal hemoglobinuria (PNH), you may be able to participate in a stem cell transplant clinical trial designed to evaluate methods to decrease graft-versus-host disease, promote engraftment, and improve immune system recovery following a bone marrow stem cell transplant procedure. 

You must have an HLA-matched family member to participate. You will be given chemotherapy followed by a transfusion of stem cells and lymphocytes from your donor, which will replace your immune system with the immune system of your healthy donor. Post-transplant therapy is designed to reduce the risk of graft-versus-host disease. 

We do the blood testing free of charge to see if your family member is a match. We pay for all medical costs related to the transplant procedure. You must be available to live near NIH for approximately 3 months. We also provide a daily allowance to help with living expenses while you are on the study and living away from home. 

To inquire about eligibility, contact our research coordinator (301) 594-8013 or by e-mail at Please reference study # 99-H-0050.


Umbilical Cord Transplant to Treat Severe Aplastic Anemia - Detailed Protocol Information

If you (or someone you know) has been diagnosed with aplastic anemia, you may be able to participate in our clinical trial designed to investigate methods of making stem cell transplant procedures safer and more effective for a wide number of diseases and disorders. In this study, we will evaluate the safety and effectiveness using a different type of stem cell transplant procedure, specifically, co-infusion of peripheral blood stem cells from a family member and cord blood stem cells from an unrelated donor. 

You must have family member who is willing to participate as a stem cell donor as well as the availability of an HLA matched umbilical cord blood unit (the NIH will conduct the search and procurement) in order to participate in this study.

To find out if you qualify, please contact our research coordinator at (301) 594-8013 or by email at Please reference study # 08-H-0046. 


Last Updated: June 26, 2017