Comparing bone marrow transplantation and immune suppression therapy for severe aplastic anemia in young patients
Official title: A 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 Boston Children's Hospital · NIH-11060911 · Data updated 2026-11-12
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.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Boston Children's Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | April 1, 2022 |
| Project end | March 31, 2029 |
| Funding year | 2025 |
| Award (this year) | $634,334 |
| NIH project number | 5UH3HL157564-04 |
| Project ID | NIH-11060911 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are pediatric and young adult patients diagnosed with severe aplastic anemia who do not have a matched sibling donor.
✕Less likely to help
Patients who have a matched sibling donor for bone marrow transplantation may not benefit from this research.
What this research studies
This research investigates the effectiveness of unrelated donor bone marrow transplantation (BMT) 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, especially since only a small percentage of patients have matched sibling donors available for BMT. Participants will be randomly assigned to receive either treatment, and their health outcomes will be monitored over time to assess the effectiveness and safety of each approach. This trial is crucial for establishing a standard treatment protocol for patients without a sibling donor.
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 their chances of recovery.
How similar studies have gone: Previous studies have shown promising results for unrelated donor BMT in similar conditions, suggesting that this approach may be effective, although this specific comparison is novel.
Where this research is happening
-
Boston Children's Hospital
Boston, United States Active
Researchers
- Principal investigator: Williams, David a — Boston Children's Hospital
- Study coordinator: Williams, David a
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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.
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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.