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A 5'UTR polymorphism in NT5E gene but not fludarabine systemic exposure influences HCT outcome in patients with high-risk β-thalassemia major
Aim Although the fludarabine (F-araA)-treosulfan based toxicity reduced conditioning regimen has improved hematopoietic cell transplantation (HCT) outcome in patients with high-risk beta-thalassemia major (TM), rejection and regimen related toxicities (RRT) are still of major concern. This study aims to assess the role of F-araA pharmacokinetics (PK) and pharmacogenetics (PG) in a uniform cohort of patients with TM. Methods All patients with TM who receiving F-araA based regimen prior to HCTdoi:10.22541/au.161101014.47082569/v1 fatcat:4x322d3ofvagbasxu4ecpxivoy