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Myelodysplastic Syndromes (MDS) Research Studies

Eltrombopag in Myelodysplastic syndrome (MDS) patients with low platelets - Protocol Description 

If you have been diagnosed with myelodysplastic syndrome, are18 years old or older, and have a low platelet count, you may be able to participate in a NIH sponsored clinical trial evaluating eltrombopag, a new FDA approved drug that has been shown to help the bone marrow produce more platelets in patients with another disorder that causes a low platelet count, immune thrombocytopenia (ITP). We are studying whether or not this drug can increase the platelet count and/or reduce the need for platelet transfusions in patients with myelodysplastic syndrome.

If you qualify for study participation, you will take eltrombopag pills by mouth once a day for three months. While you are taking eltrombopag and for 30 days after you discontinue the drug we will monitor you closely for side effects. You will need to been seen at least once a month at the NIH, and weekly for blood count monitoring either with your home physician or at the NIH. To find out if you qualify or for more information, please contact our study coordinator 301-594-4180 or email us at 


Non-myeloablative stem cell transplant for patients with myelodysplastic syndrome - Protocol Description

If you have been diagnosed with MDS, 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 at (301) 594-8013 or by e-mail


Umbilical Cord Transplant to Treat Myelodysplastic Syndrome (MDS) - Protocol Description

If you (or someone you know) has been diagnosed with myelodysplastic syndrome, 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 a 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

Last Updated: July 31, 2015