Back to Search
Start Over
Using of deferasirox and deferoxamine in refractory iron overload thalassemia
- Source :
- Pediatrics international : official journal of the Japan Pediatric SocietyReferences. 63(4)
- Publication Year :
- 2020
-
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.
- Subjects :
- medicine.medical_specialty
Liver Iron Concentration
Iron Overload
Combination therapy
Thalassemia
030204 cardiovascular system & hematology
Deferoxamine
Iron Chelating Agents
Gastroenterology
Benzoates
Iron chelation
03 medical and health sciences
0302 clinical medicine
Refractory
030225 pediatrics
Internal medicine
medicine
Humans
Decreased serum ferritin
business.industry
Deferasirox
beta-Thalassemia
Triazoles
medicine.disease
Pediatrics, Perinatology and Child Health
business
medicine.drug
Subjects
Details
- ISSN :
- 1442200X
- Volume :
- 63
- Issue :
- 4
- Database :
- OpenAIRE
- Journal :
- Pediatrics international : official journal of the Japan Pediatric SocietyReferences
- Accession number :
- edsair.doi.dedup.....0ae4ef353710283393b0e18e3a6fc602