New drug combo shows promise for rare blood disorder

NCT ID NCT01623167

Ongoing Disease control Sponsor: National Heart, Lung, and Blood Institute (NHLBI) Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

acquired aplastic anemia aplastic anemia autoimmune disease pancytopenia thrombocytopenia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

SEVERE APLASTIC ANEMIA

Contacts and locations

Locations

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

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