What this study adds
- Dose-exposure-response relationship of F-araA was evaluated in a large
uniform cohort of patients with high-risk TM undergoing HCT.
- Wide Inter-individual variation in F-araA PK, partially explained by a
genetic polymorphism in NT5E gene.
- F-araA PK did not explain variability in HCT outcome while patients
carrying NT5E promoter variant had improved outcome and better
survival.
- Our results suggest that NT5E polymorphism could be a
predictive biomarker in F-araA based HCT setting in TM, however
extensive functional studies are warranted to validate the clinical
utility of this finding.