Efficacy of Deferasirox for the treatment of iron overload in Chinese thalassaemia major patients: results from a prospective, open‐label, multicentre clinical trial

Lai, Yongrong; Liu, R.-R.; Li, C.-F.; Huang, Siqi; Li, Q.; Habr, Dany; Martin, Nicolas; Shen, Zhi Xiang

doi:10.1111/tme.12077

Cited by 13 publications

(12 citation statements)

References 18 publications

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“…The median SF level was 4536 ng/ml and 165 patients (82.1%) had SF>2500 ng/ml. The data confirmed our previous finding that Chinese TM patients were younger and had greater burden of iron overload than those in western countries [31]. Myocardial iron overload was detected in 33.8% of our patients.…”

Section: Discussionsupporting

confidence: 91%

How Early Can Myocardial Iron Overload Occur in Beta Thalassemia Major?

et al. 2014

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BackgroundMyocardial siderosis is the most common cause of death in patients with beta thalassemia major(TM). This study aimed at investigating the occurrence, prevalence and severity of cardiac iron overload in a young Chinese population with beta TM.Methods and ResultsWe analyzed T2* cardiac magnetic resonance (CMR), left ventricular ejection fraction (LVEF) and serum ferritin (SF) in 201 beta TM patients. The median age was 9 years old. Patients received an average of 13 units of blood per year. The median SF level was 4536 ng/ml and 165 patients (82.1%) had SF>2500 ng/ml. Myocardial iron overload was detected in 68 patients (33.8%) and severe myocardial iron overload was detected in 26 patients (12.6%). Twenty-two patients ≤10 years old had myocardial iron overload, three of whom were only 6 years old. No myocardial iron overload was detected under the age of 6 years. Median LVEF was 64% (measured by CMR in 175 patients). Five of 6 patients with a LVEF<56% and 8 of 10 patients with cardiac disease had myocardial iron overload.ConclusionsThe TM patients under follow-up at this regional centre in China patients are younger than other reported cohorts, more poorly-chelated, and have a high burden of iron overload. Myocardial siderosis occurred in patients younger than previously reported, and was strongly associated with impaired LVEF and cardiac disease. For such poorly-chelated TM patients, our data shows that the first assessment of cardiac T2* should be performed as early as 6 years old.

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Section: Discussionsupporting

confidence: 91%

How Early Can Myocardial Iron Overload Occur in Beta Thalassemia Major?

et al. 2014

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“…The proportion of our patients reported to have deferasirox-related adverse events was similar to other studies [ 22 ]. The most common adverse events with a suspected relationship to deferasirox were also similar to other studies [ 15 , 22 , 23 ]. However, the incidences of common adverse events related to deferasirox were higher in our patients when compared with those in other studies.…”

Section: Discussionsupporting

confidence: 87%

The long-term efficacy and tolerability of oral deferasirox for patients with transfusion-dependent β-thalassemia in Taiwan

Chang

Peng

et al. 2015

Ann Hematol

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Deferasirox 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.Electronic supplementary materialThe online version of this article (doi:10.1007/s00277-015-2476-y) contains supplementary material, which is available to authorized users.

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“…We identified four RCTs, two NRCTs, five NCTs and two ROS reporting DFP‐related AEs in 667 patients using DFP doses of 50–75 mg/kg/d . Occurrence of mild‐to‐moderate neutropenia was reported in 425 patients with a pooled incidence of 7.1% (95% CI 2.3–13.7).…”

Section: Resultsmentioning

confidence: 99%

“…One RCT, one NRCT, 10 NCTs and three ROS were identified from the literature counting 37 patients (37,50,52,(58)(59)(60)(61)(62)(63)(64)(65)(66)(67)(68)(69)(70). Investigated DFX doses ranged from 10 to 40 mg/ kg/d.…”

Section: Deferasirox (Dfx) Monotherapymentioning

confidence: 99%

Safety profiles of iron chelators in young patients with haemoglobinopathies

Botzenhardt

Chan

et al. 2017

European J of Haematology

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Background: This review describes the safety of deferoxamine (DFO), deferiprone (DFP), deferasirox (DFX) and combined therapy in young patients less than 25 yr of age with haemoglobinopathies. Methods: Searches in electronic literature databases were performed. Studies reporting adverse events associated with iron chelation therapy were included. Study and reporting quality was assessed using AHRQ Risk of Bias Assessment Tool and McMaster Quality Assessment Scale of Harms. Prospective clinical studies were pooled in a random-effects meta-analysis of proportions. Results: Safety data of 2040 patients from 34 studies were included. Ninety-two case reports of 246 patients were identified. DFX (937 patients) and DFP (667 patients) possess the largest published safety evidence. Fewer studies on combination regimens are available. Increased transaminases were seen in all regimens (3.9-31.3%) and gastrointestinal disorders with DFP and DFX (3.7-18.4% and 5.8-18.8%, respectively). Therapy discontinuations due to adverse events were low (0-4.1%). Reporting quality was selective and poor in most of the studies. Conclusion: Iron chelation therapy is generally safe in young patients, and published data correspond to summary of product characteristics. Each iron chelation regimen has its specific safety risks. DFO seems not to be associated with serious adverse effects in recommended doses. In DFP and DFX, rare, but serious, adverse reactions can occur. Data on combined therapy are scarce, but it seems equally safe compared to monotherapy.

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Efficacy of Deferasirox for the treatment of iron overload in Chinese thalassaemia major patients: results from a prospective, open‐label, multicentre clinical trial

Abstract: Age and deferasirox exposure influenced study findings, supporting the need for longer-term treatment and dose escalation to ≥30 mg kg(-1) day(-1) to achieve neutral or negative iron balance in heavily iron overloaded and younger Chinese patients.

Cited by 13 publications

References 18 publications

How Early Can Myocardial Iron Overload Occur in Beta Thalassemia Major?

How Early Can Myocardial Iron Overload Occur in Beta Thalassemia Major?

The long-term efficacy and tolerability of oral deferasirox for patients with transfusion-dependent β-thalassemia in Taiwan

Safety profiles of iron chelators in young patients with haemoglobinopathies

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