Providing Pediatric Transplant Access and Personalized Conditioning for Children
Official title: BMT Core - Pediatric Transplantation and Cellular Therapy Consortium (PTCTC): Providing Clinical Trial Access For Children With Life Threatening Diseases
Funded at National Marrow Donor Program · NIH-11392701 · Data updated 2026-11-12
A national program helps kids with life-threatening blood diseases access allogeneic hematopoietic cell transplantation and tailors pre-transplant chemotherapy to each child.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | National Marrow Donor Program NIH-funded |
| Lab location | 1 site (Minneapolis, United States) |
| Project start | August 15, 2024 |
| Project end | June 30, 2031 |
| Funding year | 2026 |
| Award (this year) | $164,412 |
| NIH project number | 5UG1HL174426-03 |
| Project ID | NIH-11392701 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with life-threatening hematologic diseases who qualify for allogeneic hematopoietic cell transplantation and whose dosing may be influenced by body size or metabolic differences.
✕Less likely to help
Children with conditions not requiring allogeneic transplantation or where transplantation is not appropriate may not benefit.
What this research studies
This grant supports pediatric patients with serious blood disorders who may benefit from a donor-based stem cell transplant. It aims to understand how to personalize pre-transplant chemotherapy dosing by considering body size and individual metabolism to improve safety and effectiveness. The effort leverages the National Marrow Donor Program to facilitate access to transplant services and to optimize treatment plans for children. Results could guide more precise, patient-centered conditioning regimens in pediatric transplantation.
Why it matters
Potential benefit: Better survival and quality of life for children needing life-saving transplants through personalized dosing and improved access to care.
How similar studies have gone: Prior work shows that personalized conditioning and dosing can reduce toxicity and improve outcomes in transplantation, though pediatric-specific data remain limited and further confirmation is needed.
Where this research is happening
-
National Marrow Donor Program
Minneapolis, United States Active
Researchers
- Principal investigator: Stefanski, Heather E — National Marrow Donor Program
- Study coordinator: Stefanski, Heather E
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.