Efficacy and Safety of a Dispersible Tablet of GPO-Deferasirox Monotherapy among Children with Transfusion-Dependent Thalassemia and Iron Overload

Abstract

The study aimed to determine efficacy and safety of generic deferasirox monotherapy. Deferasirox was administered in transfusion-induced iron overloaded thalassemia. Efficacy was defined as responders and nonresponders by ≤ 15 reduced serum ferritin from baseline. Adverse events were also monitored. Fifty-two patients with mainly Hb E/β-thalassemia at the mean (SD) age of 8.7 (4.1) years, were enrolled. The mean (SD) daily transfusion iron load was 0.47 (0.1) mg/kg and maximum daily deferasirox was 35.0 (6.2) mg/kg. Altogether, 52, 40 and 18 patients completed the first, second and third years of study, respectively. The median baseline serum ferritin 2,383 ng/mL decreased to 1,478, 1,038 and 1,268 ng/mL at the end of first, second and third years, respectively, with overall response rate at 73.1% (38/52). Patients with baseline serum ferritin >2,500 ng/mL showed a change in serum ferritin higher than those ≤2,500 ng/mL starting from the 9th month of chelation. Adverse events were found in 5 of 52 patients (9.6%) including transaminitis (n = 2), one each of proteinuria, rash and proximal tubular dysfunction which resolved after transient stopping or decreasing the chelation dose. Generic deferasirox was effective and safe among pediatric patients with transfusion-induced iron overloaded thalassemia.

Keywords:

• Deferasirox
• dispersible tablet
• iron overload
• thalassemia
• transfusion-dependent thalassemia

Acknowledgements

The authors would like to thank the GPO for providing the dispersible tablets of deferasirox for the study.

Authors’ contributions

AC designed and conducted the study using data analysis, and composed the manuscript while DS and NS took care of the patients and analyzed data. PS took care of patients complicated with proximal tubular dysfunction and proteinuria while WS performed and interpreted MRI T2*. KK took care of the patients and monitored their compliance while PK and NT identified mutations of thalassemia and laboratory investigations. All authors approved the manuscript.

Disclosure statement

The authors report no conflicts of interest. The authors alone are responsible for the content and writing of this article.

Statement of ethics

The study protocol was reviewed and approved by the Faculty Ethics Committee, Faculty of Medicine Ramathibodi Hospital, Mahidol University (COA.MURA 2020/750) and was conducted according to Good Clinical Practice Guidelines and the Declaration of Helsinki. Written informed consent was obtained from patients and parents.

Data availability statement

The data from the findings of the study are available from the corresponding author upon reasonable request.

Additional information

Funding

The GPO, Thailand, provided the dispersible tablets of deferasirox for the patients enrolled in the study.