Publication: Using of deferasirox and deferoxamine in refractory iron overload thalassemia
Issued Date
2021-04-01
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ISSN
1442200X
13288067
13288067
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2-s2.0-85103260900
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Mahidol University
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SCOPUS
Bibliographic Citation
Pediatrics International. Vol.63, No.4 (2021), 404-409
Suggested Citation
Chayamon Takpradit, Vip Viprakasit, Nattee Narkbunnam, Nassawee Vathana, Kamon Phuakpet, Bunchoo Pongtanakul, Kleebsabai Sanpakit, Jassada Buaboonnam Using of deferasirox and deferoxamine in refractory iron overload thalassemia. Pediatrics International. Vol.63, No.4 (2021), 404-409. doi:10.1111/ped.14444 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/78310
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Title
Using of deferasirox and deferoxamine in refractory iron overload thalassemia
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Abstract
Background: Iron overload is a major complication of transfusion-dependent thalassemia (TDT) and requires iron chelation (IC) therapy. However, a combination therapy may be required for patients responding poorly to monotherapy. Methods: Nine TDT patients previously treated with IC were enrolled; five patients were previously treated with deferasirox (DFX) twice daily. The dose of DFX was 20–40 mg/kg/day, while the dose of deferoxamine (DFO) was 18–40 mg/kg/day for 3–6 days/week. Results: At the 6- and 12-month time points, six and eight patients demonstrated decreased serum ferritin levels, with median reductions of 707 ng/mL (range, 1,653–5,444 ng/mL) and 1,129 ng/mL (range, 1,781–7,725 ng/mL) compared to the baseline, respectively. Eight patients also had a reduced liver iron concentration (LIC), with a median reduction of 3.9 mg/g dry wt (range, 8.3–11.1 mg/g dry wt). Of the five patients treated with DFX twice daily, four responded to combination therapy. All responsive patients could finally stop DFO after the decline in LIC. Moreover, there were no treatment-related complications. Conclusion: The combination of DFX and DFO proved to be effective and without significant toxicities for TDT patients who had been unresponsive to standard IC therapy. Further studies with a larger cohort size and long-term follow-up are warranted to elucidate the efficacy of the combination.