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New drug combo shows promise for rare blood disorder

NCT ID NCT01623167

First seen Apr 23, 2026 · Last updated May 24, 2026 · Updated 4 times

Summary

This study tests whether adding eltrombopag to standard immune-suppressing drugs helps people with severe aplastic anemia, a rare condition where the immune system attacks bone marrow. About 207 people aged 2 and older with untreated disease will take eltrombopag for up to 6 months alongside standard therapy. The goal is to see if this combination improves blood cell counts and is safe.

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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.

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Contacts and locations

Locations

  • National Institutes of Health Clinical Center, 9000 Rockville Pike

    Bethesda, Maryland, 20892, United States

Conditions

Explore the condition pages connected to this study.