The Long-Term Efficacy of Deferiprone in Thalassemia Patients With Iron Overload: Real-World Data from the Registry Database

Abstract

Deferiprone (DFP) is an oral iron-chelating agent that is widely used in thalassemia patients with iron overload. This study aimed to investigate the long-term efficacy of DFP monotherapy on serum ferritin (SF) and adverse events. All thalassemia patients aged 15 years or older who received DFP monotherapy were identified from the thalassemia registry database between November 2008 and October 2019. After treatment, patients who achieved a target SF level, defined as <1000.0 ng/mL in transfusion-dependent thalassemia (TDT) and <800.0 ng/mL in non-TDT (NTDT) for two consecutive visits, were categorized as the achievable group. We used multivariate analysis to identify factors that contribute to differences between groups. One hundred and five patients were enrolled in the study with a median age of 28 (19–41) years and median initial SF level of 1399.0 (1141.0–2169.0) ng/mL. Of these, 61.0% carried Hb E (HBB: c.79G>A)/β-thalassemia (β-thal) and 60.0% were TDT patients. The median DFP dose was 63 (47–73) mg/kg/d and the median follow-up duration of treatment was 36 (20–54) months. A total of 58 (55.24%) patients were in the achievable group. The initial SF level <1350.0 ng/mL was significantly associated with achieving a targeted SF level (p = 0.002). Ten adverse events resulted in withholding DFP. The most common was gastrointestinal irritation in four patients and three patients with agranulocytosis. In conclusion, DFP is an effective iron chelator in thalassemia patients. Slightly more than half the patients (55.0%) achieved a target SF level. Lower SF levels at the beginning were an important factor.

Keywords:

• Adverse event
• agranulocytosis
• deferiprone (DFP)
• efficacy
• thalassemia

Acknowledgments

We would like to thank Ms. Antika Wongthanee, Former Head of Analytical & Statistical Data Unit, Research Institute for Health Sciences, Chiang Mai University, Chiang Mai, Thailand, for her suggestions regarding the statistical analyses in this study. The authors would also like to express their gratitude to Dr. Yotsapon Thewjitcharoen, Theptarin Hospital, Bangkok, Thailand, for his generous advice and to Dr. Tinapa Himathongkam and for English language editing.

Disclosure statement

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