Details
The U.S. Food and Drug Administration (FDA) initiated this study to explore the feasibility of using real-world setting data (TriNetX Live™ platform) to characterize patients undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) and to evaluate the characteristics and adverse outcomes of patients who experience transplant associated thrombotic microangiopathy (TA-TMA) in the pediatric and adult populations. An additional objective of this study was to characterize the ability of TriNetX to capture clinical data necessary to determine whether TA-TMA cases are considered to be high risk.
Between July 1, 2009, and August 31, 2020, 12,350 adult patients (aged 18 years and older) and 1,300 pediatric patients (aged younger than 18 years) with allo-HSCT were observed. TMA occurring within 12 months of allo-HSCT was observed among 290 (2.3%) adults and 60 (4.6%) pediatric patients, with mean ages (SD) at TMA diagnosis of 39.2 (21.1) years and 3.1 (2.67) years, respectively. Among patients with allo-HSCT, mortality within 12 months following transplant was observed in 3,160 (25.6%) adults and 180 (13.8%) pediatric patients. Among patients with TA-TMA, mortality within 12 months following TMA diagnosis was higher in both populations: 130 (44.8%) in adults and 20 (33.3%) in pediatric patients.
The study findings informed the FDA about the possibility of identifying potential external controls for clinical programs. The study findings also facilitated the understanding of the characteristics and outcomes of allogeneic HSCT population, particularly among the TA-TMA pediatric and adult patients who are identified using diagnostic codes.