Pediatric Bone Marrow Failure Network (pBMFN) to improve care for children with aplastic anemia
Official title: Pediatric Bone Marrow Failure Network (pBMFN)
Funded at Boston Children's Hospital · NIH-11453337 · Data updated 2026-11-12
A nationwide effort to understand and improve diagnosis, treatment, and long-term health for children with rare bone marrow failures like aplastic anemia.
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 | August 21, 2026 |
| Project end | June 30, 2031 |
| Funding year | 2026 |
| Award (this year) | $1,830,327 |
| NIH project number | 1U54HD123051-01 |
| Project ID | NIH-11453337 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children and adolescents with bone marrow failure conditions or related hematologic disorders who may contribute samples or data to the pBMFN.
✕Less likely to help
Some patients may not directly benefit if they do not have the specified pediatric BMF conditions or participate in data/sample collection.
What this research studies
This project creates a national Pediatric BMFN to unite pediatric and rare-disease research networks and focus on three conditions: acquired severe aplastic anemia, idiopathic bone marrow failure, and paroxysmal nocturnal hemoglobinuria in children. It will collect and analyze clinical data, biology samples, and outcomes to identify better ways to predict infections, relapse, and treatment-related toxicities. The goal is to tailor care for kids by understanding how pediatric bone marrow failure differs from adult disease. This work combines clinical expertise across leading centers to accelerate discoveries that could guide earlier, more precise therapies.
Why it matters
Potential benefit: Children with bone marrow failure could receive more accurate diagnoses and safer, more effective treatments.
How similar studies have gone: Prior networks and rare-disease collaborations have shown that coordinated data sharing improves understanding and management of complex pediatric diseases.
Where this research is happening
-
Boston Children's Hospital
Boston, United States Active
Researchers
- Principal investigator: Shimamura, Akiko — Boston Children's Hospital
- Study coordinator: Shimamura, Akiko
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.