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

Abstract

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.

• Authors: Hsiu-Hao Chang, Meng-Yao Lu, Steven Shinn-Forng Peng, Yung-Li Yang, Dong-Tsamn Lin, Shiann-Tarng Jou, Kai-Hsin Lin
• Journal: Annals of hematology (Ann Hematol) Vol. 94 Issue 12 Pg. 1945-52 (Dec 2015) ISSN: 1432-0584 [Electronic] Germany
• PMID: 26404899 (Publication Type: Clinical Trial, Journal Article, Research Support, Non-U.S. Gov't)

Chemical References

• Benzoates
• Iron Chelating Agents
• Triazoles
• Deferasirox

Topics

• Adolescent
• Adult
• Benzoates (administration & dosage, adverse effects)
• Blood Transfusion
• Child
• Child, Preschool
• Deferasirox
• Female
• Follow-Up Studies
• Humans
• Iron Chelating Agents (administration & dosage, adverse effects)
• Iron Overload (blood, therapy)
• Male
• Taiwan
• Time Factors
• Triazoles (administration & dosage, adverse effects)
• beta-Thalassemia (blood, therapy)