Understanding how Eltrombopag helps with aplastic anemia
Official title: Eltombopag: Novel Mode of Action on Normal and Aplastic Anemia Hematopoietic Stem Cells
Funded at Cleveland Clinic Lerner Com-Cwru · NIH-11120856 · Data updated 2026-11-12
This research explores how the medication Eltrombopag helps patients with aplastic anemia by looking at its effects on blood-forming cells.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cleveland Clinic Lerner Com-Cwru NIH-funded |
| Lab location | 1 site (Cleveland, United States) |
| Project start | August 15, 2022 |
| Project end | May 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $658,812 |
| NIH project number | 5R01DK131923-04 |
| Project ID | NIH-11120856 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with aplastic anemia, particularly those who have received or are considering Eltrombopag, might find this research relevant to their condition.
✕Less likely to help
Patients without aplastic anemia or related blood disorders would not directly benefit from this specific research.
What this research studies
Aplastic anemia is a serious condition where the body's immune system attacks its own blood-forming cells, leading to low blood counts. While some treatments help, many patients still struggle. A medication called Eltrombopag has shown great promise, not just by increasing platelets but also by helping the body make other types of blood cells. This project aims to uncover the exact ways Eltrombopag works, especially how it helps blood-forming cells grow and recover, even beyond its known effects. We believe understanding these mechanisms could lead to better ways to use this drug and develop new treatments.
Why it matters
Potential benefit: If successful, this research could lead to a deeper understanding of Eltrombopag's effects, potentially improving treatment strategies and outcomes for patients with aplastic anemia.
How similar studies have gone: Eltrombopag is already a successful treatment for aplastic anemia, but this research explores a new, previously unknown mechanism of its action.
Where this research is happening
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Cleveland Clinic Lerner Com-Cwru
Cleveland, United States Active
Researchers
- Principal investigator: Maciejewski, Jaroslaw P. — Cleveland Clinic Lerner Com-Cwru
- Study coordinator: Maciejewski, Jaroslaw P.
Related research
Other NIH-funded projects on the same topics.
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Emapalumab for children with aplastic anemia
Doctors will give emapalumab to children with acquired aplastic anemia to try to stop the immune attack on the bone marrow and help it make blood cells.
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Clinical and immunologic study of pediatric severe aplastic anemia to personalize care
A pediatric-focused research effort will collect and study immune and clinical data to tailor treatments for children with severe aplastic anemia.
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Aplastic Anemia Registry to Understand How Immune and Genetic Discoveries Affect Disease
A collaborative registry study to learn how genetic background and immune factors influence aplastic anemia and related bone marrow failure conditions in patients.
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Eltombopag: a new way to boost blood stem cells in aplastic anemia
A new drug approach aims to help people with idiopathic aplastic anemia by stimulating blood-forming stem cells through mechanisms that may not depend on traditional pathways.
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Investigating a new treatment for pediatric aplastic anemia
This study is looking at how well a new treatment called emapalumab works for kids with acquired aplastic anemia, a rare blood disorder where the body stops making enough blood cells, to see if it can help them produce more blood cells and feel better.
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Comparing bone marrow transplantation with immune therapy for severe aplastic anemia in young patients
This study is looking at whether bone marrow transplants from unrelated donors or immune suppression therapy works better for kids and young adults with newly diagnosed severe aplastic anemia, especially since not everyone has a sibling who can donate bone marrow.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.