Yazarlar : Taher A, Porter J, Viprakasit V et al
Yayın : Blood.
Yayın Yılı : 2012
Pubmed Linki : http://www.ncbi.nlm.nih.gov/pubmed/22589472
Konu : Talasemi
Literatür İçeriği :
Abstract
Non-transfusion-dependent thalassemia (NTDT) patients may develop iron overload and associated complications, despite occasional/no transfusions. The 1-year, randomized, double-blind, placebo-controlled THALASSA trial assessed efficacy and safety of deferasirox in iron-overloaded NTDT patients. 166 patients were randomized in a 2:1:2:1 ratio to starting doses 5 mg/kg/day deferasirox/placebo, or 10mg/kg/day deferasirox/placebo. Mean±SD actual deferasirox dose received over the study in the 5 and 10 mg/kg/day starting dose cohorts was 5.7±1.4 and 11.5±2.9mg/kg/day, respectively. At 1 year, LIC significantly decreased versus placebo (least-squares mean [LSM]±SE): -2.33±.7 (P = .001) and -4.18±.69 mg Fe/g dw (P < .001) for deferasirox 5 and 10mg/kg/day groups, respectively (baseline values [mean±SD]: 13.11±7.29 and 14.56±7.92 mg Fe/g dw). Similarly, serum ferritin significantly decreased versus placebo by LSM -235 and -337 ng/mL for deferasirox 5 and 10 mg/kg/day groups, respectively (P < .001). In placebo patients, LIC and serum ferritin increased from baseline by .38 mg Fe/g dw and 115 ng/mL (LSM), respectively. Most common drug-related AEs were nausea (n = 11, 6.6%), rash (n = 8, 4.8%) and diarrhea (n = 6, 3.6%). This is the first randomized study to show iron chelation with deferasirox significantly reduces iron overload in NTDT patients, with a similar frequency of overall AEs as placebo.
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