| Content Provider |
World Health Organization (WHO)-Global Index Medicus |
| Author |
Taher, Ali T. Cappellini, M. Domenica Aydinok, Yesim Porter, John B. Karakas, Zeynep Viprakasit, Vip Siritanaratkul, Noppadol Kattamis, Antonis Wang, Candace Zhu, Zewen Joaquin, Victor Uwamahoro, Marie José Lai, Yong-Rong |
| Description |
Author Affiliation: Taher AT ( Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon. Electronic address: ataher@aub.edu.lb.); Cappellini MD ( Universitá di Milano, Ca Granda Foundation IRCCS, Milan, Italy.); Aydinok Y ( Department of Paediatric Haematology, Ege University Hospital, Izmir, Turkey.); Porter JB ( Department of Haematology, University College London, London, UK.); Karakas Z ( Istanbul University, Istanbul Medical Faculty, Istanbul, Turkey.); Viprakasit V ( Department of Pediatrics and Internal Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand.); Siritanaratkul N ( Department of Pediatrics and Internal Medicine, Siriraj Hospital, Mahidol University, Bangkok, Thailand.); Kattamis A ( First Department of Pediatrics, University of Athens, Athens, Greece.); Wang C ( Novartis Pharmaceuticals, East Hanover, NJ, USA.); Zhu Z ( Novartis Pharmaceuticals, East Hanover, NJ, USA.); Joaquin V ( Novartis Pharmaceuticals, East Hanover, NJ, USA.); Uwamahoro MJ ( Novartis Pharma AG, Basel, Switzerland.); Lai YR ( The First Affiliated Hospital of Guangxi Medical University, Nanning, China. Electronic address: laiyongrong@263.net.) |
| Abstract |
Efficacy and safety of iron chelation therapy with deferasirox in iron-overloaded non-transfusion-dependent thalassaemia (NTDT) patients were established in the THALASSA study. THETIS, an open-label, single-arm, multicentre, Phase IV study, added to this evidence by investigating earlier dose escalation by baseline liver iron concentration (LIC) (week 4: escalation according to baseline LIC; week 24: adjustment according to LIC response, maximum 30mg/kg/day). The primary efficacy endpoint was absolute change in LIC from baseline to week 52. 134 iron-overloaded non-transfusion-dependent anaemia patients were enrolled and received deferasirox starting at 10mg/kg/day. Mean actual dose±SD over 1year was 14.70±5.48mg/kg/day. At week 52, mean LIC±SD decreased significantly from 15.13±10.72mg Fe/g dw at baseline to 8.46±6.25mg Fe/g dw (absolute change from baseline, -6.68±7.02mg Fe/g dw [95% CI: -7.91, -5.45]; P<0.0001). Most common drug-related adverse events were gastrointestinal: abdominal discomfort, diarrhoea and nausea (n=6 each). There was one death (pneumonia, not considered drug related). With significant and clinically relevant reductions in iron burden alongside a safety profile similar to that in THALASSA, these data support earlier escalation with higher deferasirox doses in iron-overloaded non-transfusion-dependent anaemia patients. |
| File Format |
HTM / HTML |
| ISSN |
10799796 |
| Volume Number |
57 |
| e-ISSN |
10960961 |
| Journal |
Blood Cells, Molecules, and Diseases |
| Language |
English |
| Publisher |
Elsevier |
| Publisher Date |
2016-03-01 |
| Publisher Place |
United States |
| Access Restriction |
One Nation One Subscription (ONOS) |
| Subject Keyword |
Discipline Hematology Benzoates Administration & Dosage Chelation Therapy Methods Iron Chelating Agents Iron Overload Drug Therapy Liver Drug Effects Thalassemia Triazoles Adolescent Adult Adverse Effects Blood Transfusion Child Diarrhea Chemically Induced Diagnosis Drug Administration Schedule Drug Dosage Calculations Female Follow-up Studies Humans Iron Metabolism Complications Pathology Male Middle Aged Nausea Treatment Outcome Clinical Trial, Phase Iv Journal Article Multicenter Study Research Support, Non-u.s. Gov't |
| Content Type |
Text |
| Resource Type |
Article |
| Subject |
Cell Biology Hematology Molecular Biology Molecular Medicine |
