New drug combo shows promise for rare blood disorder
NCT ID NCT01623167
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests whether adding eltrombopag to standard immunosuppressive therapy can improve blood cell counts in people with untreated severe aplastic anemia. Participants receive horse-ATG and cyclosporine plus eltrombopag for up to 6 months. The goal is to see if this combination leads to better outcomes than standard therapy alone.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Eltrombopag (a drug that helps the body make more blood cells)
What this could lead to
If it works, this could improve blood cell counts and reduce the need for transfusions in people with severe aplastic anemia.
What could go wrong
This is an early-phase trial, so results are not yet proven. The drug may cause side effects or not work for everyone.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892, United States