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

Abstract

OBJECTIVE:

To assess the efficacy and safety of deferasirox in Chinese thalassaemia major (TM) patients

BACKGROUND:

EPIC (Evaluation of Patients' Iron Chelation with Exjade(®)) was a large multi-national study and, notably, the first clinical trial of an iron chelator registered with the Chinese State Food and Drug Administration.

METHODS:

Efficacy and safety of deferasirox were compared in Chinese (n = 117) and non-Chinese (n = 998) TM patients. Deferasirox was initiated at 20 mg kg(-1)  day(-1), with titration increments of 5-10 mg kg(-1) day(-1), based on serum ferritin trends and safety parameters.

RESULTS:

At baseline, Chinese patients were younger than non-Chinese (mean age 6·8 versus 19·5 years), with higher median serum ferritin (4519 vs 3058 ng mL(-1)). Over 1 year, mean actual deferasirox dose was similar for Chinese and non-Chinese patients (24·6 and 24·0 mg kg(-1)  day(-1), respectively); median serum ferritin did not change significantly from baseline in Chinese patients (+340 ng mL(-1), P = 0·102) and significantly decreased in non-Chinese patients (-220 ng mL(-1); P < 0·001). In the 1-year extension in Chinese patients, (mean actual deferasirox dose 33·6 mg kg(-1)  day(-1)), median serum ferritin decreased (-756 ng mL(-1); P = 0·0397), with a numerically higher reduction in patients aged ≥6 to  < 12 than <6 years (-982 vs -457 ng mL(-1), respectively). The safety profile of deferasirox in Chinese patients was similar to the overall population with respect to clinically-relevant findings.

CONCLUSION:

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.

• Authors: Y-R Lai, R-R Liu, C-F Li, S-L Huang, Q Li, D Habr, N Martin, Z-X Shen
• Journal: Transfusion medicine (Oxford, England) (Transfus Med) Vol. 23 Issue 6 Pg. 389-96 (Dec 2013) ISSN: 1365-3148 [Electronic] England
• PMID: 24147622 (Publication Type: Clinical Trial, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
• Copyright: © 2013 The Authors. Transfusion Medicine © 2013 British Blood Transfusion Society.

Chemical References

• Benzoates
• Iron Chelating Agents
• Triazoles
• Iron
• Deferasirox

Topics

• Adolescent
• Adult
• Age Factors
• Aged
• Asian People
• Benzoates (administration & dosage, adverse effects)
• Child
• Child, Preschool
• China
• Deferasirox
• Female
• Humans
• Infant
• Iron (blood)
• Iron Chelating Agents (administration & dosage, adverse effects)
• Iron Overload (blood, drug therapy)
• Male
• Middle Aged
• Prospective Studies
• Thalassemia (blood, drug therapy)
• Triazoles (administration & dosage, adverse effects)