Optimising iron chelation therapy with deferasirox for non-transfusion-dependent thalassaemia patients: 1-year results from the THETIS study
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Academic Press Inc.
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 30 mg/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 10 mg/kg/day. Mean actual dose ± SD over 1 year was 14.70 ± 5.48 mg/kg/day. At week 52, mean LIC ± SD decreased significantly from 15.13 ± 10.72 mg Fe/g dw at baseline to 8.46 ± 6.25 mg Fe/g dw (absolute change from baseline, -6.68 ± 7.02 mg 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. © 2015 The Authors.
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Dose escalation, Iron chelation therapy, Iron overload, Non-transfusion-dependent thalassaemia, Ntdt, Adolescent, Adult, Benzoates, Blood transfusion, Chelation therapy, Child, Diarrhea, Drug administration schedule, Drug dosage calculations, Female, Follow-up studies, Humans, Iron, Iron chelating agents, Liver, Male, Middle aged, Nausea, Thalassemia, Treatment outcome, Triazoles, Alanine aminotransferase, Chromium chloride, Creatinine, Deferasirox, Deferiprone, Ferritin, Hydroxyurea, Protein, Benzoic acid derivative, Iron chelating agent, Triazole derivative, Abdominal discomfort, Abdominal pain, Alanine aminotransferase blood level, Article, Cataract, Chromium blood level, Concentration (parameters), Conduction deafness, Creatinine blood level, Creatinine urine level, Drug dose escalation, Drug dose increase, Drug efficacy, Drug safety, Dyspepsia, Ferritin blood level, Heart failure, Human, Insomnia, Liver iron concentration, Major clinical study, Medication compliance, Multicenter study, Neutrophil count, Non transfusion dependent thalassaemia, Open study, Pancreatitis, Perception deafness, Phase 4 clinical trial, Pneumonia, Priority journal, Protein urine level, Rash, Thrombocyte count, Transfusion, Vomiting, Chemically induced, Clinical trial, Complication, Dose calculation, Drug administration, Drug effects, Follow up, Metabolism, Pathology, Procedures