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Target Ferritin Level Achieved With Long-Term Deferiprone in Thalassemia With Iron Overload

November 21, 2022

December 2022

Leah Lawrence

Leah Lawrence is a freelance health writer and editor based in Delaware.

Deferiprone (DFP) was an effective iron chelator for patients with thalassemia, with more than half achieving a target serum ferritin (SF) level with long-term use, according to a study of real-world data published in Hemoglobin. DFP is the frontline national essential drug in Thailand for iron overload therapy.

Teerajed Kittipoom, MD, of Chiang Mai University in Thailand, and colleagues wanted to determine the long-term effectiveness and adverse events (AEs) of DFP monotherapy in transfusion-dependent (TDT) and non-transfusion-dependent thalassemia (NTDT). To do this, they used data from a thalassemia registry database from November 2008 to October 2019 and studied the use of DFP in patients 15 and older with thalassemia. Those patients in the database who were able to achieve a target SF level were categorized as “achievable.” For patients with TDT, this was defined as less than 1,000 ng/mL for two consecutive visits; and in patients with NTDT, it was defined as less than 800 ng/mL for two consecutive visits.

The study included 105 patients, 60% of whom had TDT. The majority of patients (61.0%) were carriers of hemoglobin E beta thalassemia disease. Median initial SF level was 1,399 ng/mL, and median DFP monotherapy duration was 35.9 months.

Median DFP dose was 63 mg/kg/day. A little more than half of patients (55%) were in the achievable group. Overall, the median value for SF decreased from 1,250 to 814 ng/mL in 7.6 months. In the achievable group, the mean SF level changed from 1,298 to 896 ng/mL. In the non-achievable group, the mean SF increased from 1,895 to 1,911 ng/mL.

A multivariable analysis showed that the only significant factor associated with achieving the target SF level was an initial SF level of less than 1,350 ng/mL (p=0.002). Sex, age, thalassemia type, splenic status, and bodyweight did not significantly differ between those who were in the achievable group and those who were not.

Changes in tissue iron deposition measured by liver iron concentration and cardiac T2* on magnetic resonance imaging (MRI) were collected, but no significant reduction was found.

Ten patients had severe AEs that resulted in withholding DFP. The most common AE was nausea with vomiting (3.8%). Agranulocytosis occurred in three patients (2.8%). Dr. Kittipoom and colleagues wrote that careful monitoring of complete blood counts during the first year of therapy is warranted with use of DFP. Leukopenia, ototoxicity, and transaminitis each occurred in one patient. No serious complications occurred.

The researchers classified patients into four groups: stop group (discontinued DFP for more than three months; 25.7%), continue group (38.1%), combination group (DFP combined with another drug; 21.0%), and switch group (use of a new drug; 15.2%). Of the 58 patients who were in the achievable group, less than half were able to stop DFP. The majority of patients in the stop group (81.5%) had to restart treatment within a median of 13.6 months.

The study was limited by the fact that some non-serious AEs were not noted in the database and MRI was not routinely evaluated in all patients, according to researchers. They also noted interest in exploring whether factors other than SF level could predict response to DFP.

“Our study confirmed that DFP is an effective oral iron chelator in thalassemia patients with acceptable [AEs],” the researchers concluded.

Any conflicts of interest declared by the authors can be found in the original article.

Reference

Kittipoom T, Tantiworawit A, Punnachet T, et al. The long-term efficacy of deferiprone in thalassemia patients with iron overload: real-world data from the Registry Database [published online, 2022 Aug 18]. Hemoglobin. doi: 10.1080/03630269.2022.2072326.

 

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