Aplastic Anemia Registry to Understand How Immune and Genetic Discoveries Affect Disease
Official title: Aplastic Anemia Registry Approach to Decipher the Clinical Impact of Novel Immune and Genetic Discoveries
Funded at Cleveland Clinic Lerner Com-Cwru · NIH-11443431 · Data updated 2026-11-12
A collaborative registry study to learn how genetic background and immune factors influence aplastic anemia and related bone marrow failure conditions in patients.
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 | September 1, 2026 |
| Project end | May 30, 2030 |
| Funding year | 2026 |
| Award (this year) | $628,000 |
| NIH project number | 1R01HL188557-01 |
| Project ID | NIH-11443431 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with aplastic anemia or related bone marrow failure conditions who are enrolled in the clinical registry and have available genetic/immune data.
✕Less likely to help
Patients not represented in the registry or without linked genetic/immune data may not directly benefit from this study.
What this research studies
This project uses a clinical registry approach to collect and analyze patient data and laboratory findings from people with bone marrow failure syndromes such as aplastic anemia and related conditions. By linking genetic information with clinical outcomes, the team aims to identify patterns that explain why some patients have different disease trajectories. The study emphasizes correlative lab testing and real-world clinical observations to overcome challenges posed by rarity and limited animal models. Results could guide more personalized care and inform future research directions in human BMFS. The work is grounded in patient data from a major medical center, with a focus on translating findings to improve patient management.
Why it matters
Potential benefit: If successful, patients could benefit from more precise prognosis and tailored treatments based on their genetic and immune profiles.
How similar studies have gone: Previous registry-based studies have yielded meaningful insights in BMFS, supporting the potential value of this approach.
Where this research is happening
-
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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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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Understanding how Eltrombopag helps with aplastic anemia
This research explores how the medication Eltrombopag helps patients with aplastic anemia by looking at its effects on blood-forming cells.
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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.
-
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.