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Research Showcase: Pediatric bone marrow transplant research at UF gets boost

The mission of CMN Hospitals at UF Health Shands Children’s Hospital is to build awareness and raise funds for our pediatric patients. This research spotlight is the first in our Miracle Memo series to explore the impactful research completed thanks to CMN Hospitals funding.

Biljana Horn, M.D., is the director of the Pediatric Bone Marrow Transplant and Cell Therapy Program in the University of Florida’s Division of Hematology & Oncology. In 2018, Horn established the Florida Pediatric Bone Marrow Transplant and Cell Therapy Consortium (FPBCC).

“I came to the idea that pediatric bone marrow transplant (BMT) providers within the state of Florida should support each other,” Horn said. “To organize and improve care for children in Florida, we visited all pediatric [transplant] centers in Florida and actually got an agreement that they will participate in the consortium.”

The consortium’s mission is to improve the outcomes of all pediatric BMT patients in Florida. The first step toward that goal was to identify the ongoing trends in utilization and outcomes of pediatric hematopoietic cell (HTC) transplant in Florida.

“Outcomes research has a great impact,” Horn explained. “It has the potential of making the fastest and widest impact on patient care. Although we plan to conduct prospective trials, we started our collaboration by evaluating our outcomes first and then identifying areas for improvement.”

The FPBCC made several significant discoveries from its data research. For one, Horn and her team found that in Florida, due to genetic differences in the population, the number of human leukocyte antigen (HLA)-mismatched transplants is higher than in the US as a whole. HLA typing is used to match patients and donors for bone marrow or cord blood transplants.

HLA-mismatched transplants have historically been related to lower survival rates when compared to fully HLA-matched transplants.

The FPBCC plans on addressing the issue of HLA-mismatched transplants through a prospective trial which will introduce novel, precisely engineered grafts with a goal of reducing treatment-related mortality.

Two, the FPBCC’s analysis also showed no disparities by race in outcomes of children in Florida receiving HCT for cancers that affect the blood, bone marrow, and lymph nodes in Florida.

Throughout the pandemic, the FPBCC has met via monthly Zoom meetings. Participants share data and supply peer-to-peer advice. The collaboration allows physician-researchers to learn from one another and, ultimately, help their patients.

“It’s great to know who your peers are,” Horn said. “If you have a question, you can easily reach out to any of them. They always answer.”

CMN Hospitals funding plays a pivotal role in the FPBCC’s research. The grant supports the team’s statistician and a research coordinator. The funding also protects some of Dr. Horn’s time, which she can devote to data analyses and development of new studies.

“We are patient-centered,” said Horn. “We try to put all efforts into getting patients the best possible care in this state.”