Florida Pediatric Bone Marrow Transplant and Cell Therapy Consortium (FPBCC) Pediatric Outcomes Analysis Using Enhanced Data Back to Centers (eDBtC) Platform

Florida, with 4.1 million children, has six small pediatric BMT programs which are distributed across the state facilitating access to transplant. However, the literature indicates a survival advantage when transplants are done in a larger center with disease-specific expertise. In addition to lack of expertise across all diseases, it is more difficult for smaller centers to use their experience to improve patient outcomes as each center's analyses are skewed due to small number of transplants. In 2018, five Florida Pediatric BMT centers formed a consortium (Florida Pediatric BMT and Cell Therapy Consortium – FPBCC) with the goal of sharing data, clinical collaboration, and increasing patient base for retrospective and early stage clinical trials. The ultimate goal is to increase knowledge about utilization of pediatric BMT and its outcomes in a geographically defined population of children. This knowledge will help develop interventions for practice improvement. After signing a memorandum of understanding for FPBCC and data use agreements, and after obtaining IRB approvals for retrospective data analyses, we gathered data from all pediatric BMT transplants performed in 5 Florida Pediatric BMT centers between 2014 and 2016. We utilized the CIBMTR eDBtC (enhanced data back to center) platform to gather data reported to the CIBMTR. De-coded CIBMTR data were downloaded by each center and forwarded to the FPBCC for analysis. Four hundred and fifteen transplants were done in the five Florida centers over the 3-year study period. However, we present 377 transplants performed in 301 children who were ≤18 years at the time of transplant. The majority (58%) of first transplant recipients were male, 28% were ≤3 years of age and 68% were ≤10 years of age at time of transplant. By race, the majority of recipients were Caucasians (70%) followed by AA (21%). The majority of transplants (61%) were allogeneic with 81% of them being first, and 9% being second or a subsequent transplant. Unrelated donor was the most common donor type (64%). Among related transplant donors, 25% were mismatched. Bone marrow was used as a stem cell source in 58% of first allogeneic transplants, cord blood in 26%, and PBSCs in 16%. Among autologous transplants, 38% were second or subsequent transplant. The most common indications for allogeneic transplant were acute lymphoblastic leukemia (33%), myelogenous leukemia (20%) sickle cell disease (8%) and primary immune deficiencies (5%). In the autologous group, the most common indication for transplant was neuroblastoma (37%), followed by medulloblastoma and other brain tumors (18% each). We showed that data obtained through collaboration of programs using the eDBtC platform could lead to better understanding of activity and trends in transplant in a defined patient population. This consortium will continue with data validation and analyses in order to identify areas for improvement.