HSIU-HAO CHANGMENG-YAO LUSTEVEN SHINN-FORNG PENGYUNG-LI YANGLin D.-T.SHIANN-TANG JOULin K.-H.2020-09-222020-09-2220150939-5555https://scholars.lib.ntu.edu.tw/handle/123456789/514113Deferasirox is a novel once-daily, oral iron chelator. The aim of this study was to evaluate the long-term efficacy and tolerability of deferasirox in Taiwanese patients with transfusion-dependent β-thalassemia who have been treated with deferasirox for 7?years. Taiwanese patients aged ?2?years with transfusion-dependent β-thalassemia whose serum ferritin levels were ?1000?ng/mL and had started deferasirox treatment since December 2005 at the National Taiwan University Hospital were enrolled. Sixty patients were recruited for analysis, and 11 (18.3?%) patients discontinued deferasirox during the study. In the 42 patients included in the efficacy analysis, the mean serum ferritin levels decreased significantly by 2566?ng/mL after 7?years of treatment (P < 0.001). Forty-one of these patients received a cardiac T2* evaluation after 3?years of deferasirox treatment, and the mean cardiac T2* value increased significantly from 30.6 ± 16.6 to 45.9 ± 22.6?ms after 7?years of deferasirox treatment (P < 0.001). Deferasirox-related adverse events assessed by investigators were reported in 46 (76.7?%) patients. The most common adverse events related to deferasirox were skin rashes (n = 29, 48.3?%), followed by abdominal pain (n = 23, 38.3?%) and diarrhea (n = 16, 26.7?%). Most adverse events were manageable. This study demonstrated that long-term treatment with deferasirox was effective in improving iron overload, including cardiac iron overload, in patients with transfusion-dependent β-thalassemia. Deferasirox was well tolerated; however, the incidences of common adverse events related to deferasirox appeared higher in our Taiwanese patients than other studies. ? 2015, The Author(s).[SDGs]SDG3deferasirox; benzoic acid derivative; deferasirox; iron chelating agent; triazole derivative; abdominal pain; adolescent; adult; Article; beta thalassemia; child; diarrhea; drug efficacy; drug tolerability; drug withdrawal; Fanconi renotubular syndrome; female; ferritin blood level; human; hypertransaminasemia; iron chelation; iron overload; long term care; major clinical study; male; priority journal; rash; Taiwan; Taiwanese; transfusion; beta-Thalassemia; blood; blood transfusion; clinical trial; follow up; iron overload; preschool child; time factor; Adolescent; Adult; Benzoates; beta-Thalassemia; Blood Transfusion; Child; Child, Preschool; Female; Follow-Up Studies; Humans; Iron Chelating Agents; Iron Overload; Male; Taiwan; Time Factors; Triazolesjournal article10.1007/s00277-015-2476-y264048992-s2.0-84944075644