Clinical and immunologic study of pediatric severe aplastic anemia to personalize care
Official title: Project 1: Clinical and immunologic studies of pediatric acquired severe aplastic anemia to inform precision medicine
Funded at Boston Children's Hospital · NIH-11453341 · Data updated 2026-11-12
A pediatric-focused research effort will collect and study immune and clinical data to tailor treatments for children with severe 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) | $479,554 |
| NIH project number | 1U54HD123051-01 |
| Project ID | NIH-11453341 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and adolescents diagnosed with acquired severe aplastic anemia who may receive immune suppression or transplant-based therapies.
✕Less likely to help
Patients not eligible for pediatric-specific analyses or not pursuing hematopoietic transplant or IST within the study framework may see limited benefit.
What this research studies
This project looks at how children with acquired severe aplastic anemia respond to different treatments and why outcomes differ from adults. It will harmonize data from multiple centers to track long-term health, development, and fertility after therapy. By studying immune and biological factors in paSAA, doctors hope to guide more personalized treatment choices in the future.
Why it matters
Potential benefit: If successful, the work could lead to more effective, safer treatment plans tailored for children and adolescents with paSAA.
How similar studies have gone: Previous natural-history and cross-center data efforts have helped clarify paSAA outcomes, but randomized comparisons remain limited; this study advances the data-needed approach.
Where this research is happening
-
Boston Children's Hospital
Boston, United States Active
Researchers
- Principal investigator: Pulsipher, Michael a — Boston Children's Hospital
- Study coordinator: Pulsipher, Michael a
Related research
Other NIH-funded projects on the same topics.
-
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.
-
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.
-
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.
-
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.
-
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.
-
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.
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.