Publication: Long-term effectiveness, safety, and tolerability of twice-daily dosing with deferasirox in children with transfusion-dependent thalassemias unresponsive to standard once-daily dosing
Issued Date
2021-01-01
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ISSN
20353006
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2-s2.0-85119477395
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Mahidol University
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SCOPUS
Bibliographic Citation
Mediterranean Journal of Hematology and Infectious Diseases. Vol.13, No.1 (2021)
Suggested Citation
Jassada Buaboonnam, Chayamon Takpradit, Vip Viprakasit, Nattee Narkbunnam, Nassawee Vathana, Kamon Phuakpet, Kleebsabai Sanpakit, Bunchoo Pongtanakul Long-term effectiveness, safety, and tolerability of twice-daily dosing with deferasirox in children with transfusion-dependent thalassemias unresponsive to standard once-daily dosing. Mediterranean Journal of Hematology and Infectious Diseases. Vol.13, No.1 (2021). doi:10.4084/MJHID.2021.065 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/78565
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Title
Long-term effectiveness, safety, and tolerability of twice-daily dosing with deferasirox in children with transfusion-dependent thalassemias unresponsive to standard once-daily dosing
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Abstract
Background: Patients with transfusion-dependent thalassemia (TDT) risk iron overload and require iron chelation therapy. Second-line therapy is warranted for patients demonstrating poor chelation responses. Patients and methods: We retrospectively studied the serum-ferritin (SF), and liver-iron-concentration (LIC) outcomes of patients with TDT treated with twice-daily dosing of deferasirox (TDD-DFX) > 24 months, after failing to respond to once-daily deferasirox (OD-DFX). Results: We enrolled 22 OD-DFX nonresponders (14 males and eight females; median age, 9.2 [3-15.5] years). The median blood transfusion was 216 (206-277) ml/kg/year. The median TDD-DFX treatment period was 30 (24-35) months. Before initiating TDD-DFX, the median SF level was 2,486 (1,562-8,183) ng/ml, while the median LIC was 6.6 (3.2-19) mg/g dry wt. There were 18 TDD-DFX responders (81.8%) and 4 TDD-DFX nonresponders. The median SF-level change was -724 (-4,916 to 1,490) ng/mL. The median LIC change was -2.14 (-13.7 to 6.8) mg/g dry wt. The 1-year and 2-year SF levels and LICs were statistically significant (SF, P = 0.006/0.005; and LIC, 0.006/0.005, respectively). There were no treatment interruptions secondary to adverse events. In the follow-up of the TDD-DFX responder group, 11 of the 18 had a reduced dose, whereas the remaining seven continued with the same dose. Conclusions: TDD-DFX appears to be an alternative treatment approach for patients refractory to OD-DFX, with a favorable long-term safety profile. Further studies with larger groups and pharmacogenetic analyses of OD-DFX responders are warranted to determine the efficacy and safety profile of TDD-DFX.