Comparing bone marrow transplantation with immune therapy for severe aplastic anemia in young patients
Official title: 1/2A Phase III Randomized Trial Comparing Unrelated Donor Bone Marrow Transplantation with Immune Suppressive Therapy for Newly Diagnosed Pediatric and Young Adult Patients with Severe Aplastic Anemia
Funded at Medical College of Wisconsin · NIH-11060990 · Data updated 2026-11-12
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.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Medical College of Wisconsin NIH-funded |
| Lab location | 1 site (Milwaukee, United States) |
| Project start | April 1, 2022 |
| Project end | March 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $1,165,286 |
| NIH project number | 5U24HL157560-04 |
| Project ID | NIH-11060990 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are pediatric and young adult patients newly diagnosed with severe aplastic anemia.
✕Less likely to help
Patients who have already undergone treatment for severe aplastic anemia or those with other significant health conditions may not benefit from this research.
What this research studies
This research investigates the effectiveness of bone marrow transplantation (BMT) from unrelated donors compared to immune suppression therapy (IST) for treating newly diagnosed severe aplastic anemia (SAA) in pediatric and young adult patients. The study aims to determine which treatment provides better outcomes, considering that many patients do not have a suitable sibling donor for BMT. Participants will be randomly assigned to receive either treatment, and their health outcomes will be monitored over time. The research is being conducted by a consortium of pediatric specialists to ensure comprehensive care and support for the patients involved.
Why it matters
Potential benefit: If successful, this research could provide a more effective treatment option for young patients with severe aplastic anemia, potentially improving survival rates and quality of life.
How similar studies have gone: Previous research has shown promising outcomes for unrelated donor bone marrow transplantation in similar patient populations, indicating that this approach may be effective.
Where this research is happening
-
Medical College of Wisconsin
Milwaukee, United States Active
Researchers
- Principal investigator: Shaw, Bronwen — Medical College of Wisconsin
- Study coordinator: Shaw, Bronwen
Related research
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Clinical and immunologic study of pediatric severe aplastic anemia to personalize care
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Comparing bone marrow transplantation and immune suppression therapy for severe aplastic anemia in young patients
This study is looking at whether getting a bone marrow transplant from an unrelated donor or using immune suppression therapy works better for kids and young adults who have just been diagnosed with severe aplastic anemia, especially since not everyone has a matched sibling donor.
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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.