dc.contributor.author | KATTAMIS, Antonis | |
dc.contributor.author | Karakas, Zeynep | |
dc.contributor.author | VIPRAKASIT, Vip | |
dc.contributor.author | CHUNCHARUNEE, Suporn | |
dc.contributor.author | SUTCHARITCHAN, Pranee | |
dc.contributor.author | SIRITANARATKUL, Noppadol | |
dc.contributor.author | GALANELLO, Renzo | |
dc.contributor.author | LAWNICZEK, Tomasz | |
dc.contributor.author | ROS, Jacqueline | |
dc.contributor.author | ZHANG, Yiyun | |
dc.contributor.author | HABR, Dany | |
dc.contributor.author | CAPPELLINI, Maria Domenica | |
dc.contributor.author | TAHER, Ali T. | |
dc.contributor.author | Porter, John | |
dc.date.accessioned | 2021-03-02T22:11:17Z | |
dc.date.available | 2021-03-02T22:11:17Z | |
dc.date.issued | 2012 | |
dc.identifier.citation | TAHER A. T. , Porter J., VIPRAKASIT V., KATTAMIS A., CHUNCHARUNEE S., SUTCHARITCHAN P., SIRITANARATKUL N., GALANELLO R., Karakas Z., LAWNICZEK T., et al., "Deferasirox reduces iron overload significantly in nontransfusion-dependent thalassemia: 1-year results from a prospective, randomized, double-blind, placebo-controlled study", BLOOD, cilt.120, sa.5, ss.970-977, 2012 | |
dc.identifier.issn | 0006-4971 | |
dc.identifier.other | vv_1032021 | |
dc.identifier.other | av_0b4b4075-551f-4a37-8824-d5e5524766a7 | |
dc.identifier.uri | http://hdl.handle.net/20.500.12627/13272 | |
dc.identifier.uri | https://doi.org/10.1182/blood-2012-02-412692 | |
dc.description.abstract | Nontransfusion-dependent thalassemia (NTDT) patients may develop iron overload and its associated complications despite receiving only occasional or no transfusions. The present 1-year, randomized, double-blind, placebo-controlled THALASSA (Assessment of Exjade in Nontransfusion-Dependent Thalassemia) trial assessed the efficacy and safety of deferasirox in iron-overloaded NTDT patients. A total of 166 patients were randomized in a 2:1:2:1 ratio to starting doses of 5 or 10 mg/kg/d of deferasirox or placebo. The means +/- SD of the actual deferasirox doses received over the duration of the study in the 5 and 10 mg/kg/d starting dose cohorts were 5.7 +/- 1.4 and 11.5 +/- 2.9 mg/kg/d, respectively. At 1 year, the liver iron concentration (LIC) decreased significantly compared with placebo (least-squares mean [LSM] +/- SEM, -2.33 +/- 0.7 mg Fe/g dry weight [dw], P = .001, and -4.18 +/- 0.69 mg Fe/g dw, P < .001) for the 5 and 10 mg/kg/d deferasirox groups, respectively (baseline values [means +/- SD], 13.11 +/- 7.29 and 14.56 +/- 7.92 mg Fe/g dw, respectively). Similarly, serum ferritin decreased significantly compared with placebo by LSM -235 and -337 ng/mL for the deferasirox 5 and 10 mg/kg/d groups, respectively (P < .001). In the placebo patients, LIC and serum ferritin increased from baseline by 0.38 mg Fe/g dw and 115 ng/mL (LSM), respectively. The most common drug-related adverse events were nausea (n = 11; 6.6%), rash (n = 8; 4.8%), and diarrhea (n = 6; 3.6%). This is the first randomized study showing that iron chelation with deferasirox significantly reduces iron overload in NTDT patients with a frequency of overall adverse events similar to placebo. (Blood. 2012;120(5):970-977) | |
dc.language.iso | eng | |
dc.subject | Sağlık Bilimleri | |
dc.subject | İç Hastalıkları | |
dc.subject | Hematoloji | |
dc.subject | Dahili Tıp Bilimleri | |
dc.subject | Tıp | |
dc.subject | Klinik Tıp (MED) | |
dc.subject | Klinik Tıp | |
dc.subject | HEMATOLOJİ | |
dc.title | Deferasirox reduces iron overload significantly in nontransfusion-dependent thalassemia: 1-year results from a prospective, randomized, double-blind, placebo-controlled study | |
dc.type | Makale | |
dc.relation.journal | BLOOD | |
dc.contributor.department | Mahidol University , , | |
dc.identifier.volume | 120 | |
dc.identifier.issue | 5 | |
dc.identifier.startpage | 970 | |
dc.identifier.endpage | 977 | |
dc.contributor.firstauthorID | 26936 | |