Study: Iron Chelator Deferiprone Has Similar Safety Profile in MDS as in Other Conditions

By Keightley Amen - December 10, 2022

A recent study evaluated the safety profile of deferiprone in patients with myelodysplastic syndromes (MDS) and found no unexpected adverse events or safety concerns. The authors said the findings may indicate that deferiprone is a viable option for patients with lower-risk MDS. Amer M. Zeidan, MD, of Yale University in New Haven, Connecticut, and colleagues presented their study at the 64th American Society of Hematology Annual Meeting & Exposition.

Many patients with MDS need frequent blood transfusions, which can cause iron overload. Some guidelines indicate that patients with lower-risk MDS and iron overload should receive iron chelation, but current options to remove iron from the blood have important limitations. For example, deferoxamine requires an infusion regimen that may affect treatment adherence; oral deferasirox may improve adherence, but it is contraindicated in patients with kidney impairment and requires close monitoring of renal and hepatic function.

Deferiprone is an oral iron chelator approved for transfusion-related iron overload in thalassemia, sickle cell disease, and other anemias. Its use is not limited by patients’ renal function, but it requires close monitoring for neutropenia, which may be severe in 1% to 2% of patients, according to studies in other disease states. The current study sought to explore the safety of deferiprone in MDS.

The researchers analyzed data from two sources: (1) a safety registry established to meet U.S. Food and Drug Administration requirements, which included 115 adult patients (mean age, 78 years; 62% male) treated between 2011 and 2021; and (2) a compassionate-use program for patients from the United States and Canada, which included 15 adults (mean age, 68 years; 60% male) who were treated between 1996 and 2015. All patients received doses ranging from 75 mg/kg to 99 mg/kg per day.

In the safety registry, patients had a mean deferiprone exposure of 1.1 ± 1.4 years. Reasons for treatment discontinuation included death (n=30, but none attributed to deferiprone), physician decision (n=26), adverse events (n=16), hospice care (n=12), nonresponse (n=6), insurance issues (n=4), and change to another iron chelator (n=3). The most common adverse events were diarrhea (n=34), nausea (n=32), vomiting (n=26), and fatigue (n=26). Severe neutropenia/agranulocytosis occurred in four patients; two cases resolved, one was lost to follow-up, and one case was ongoing.

In the compassionate-use program, mean deferiprone exposure was 1.2 ± 2.3 years. The most common adverse events were nausea (n=3) and decreased neutrophil count (n=3). Two patients experienced severe neutropenia/agranulocytosis, but both cases resolved.

The authors concluded that their findings “suggest the deferiprone safety profile in MDS is potentially consistent with that of thalassemia, sickle cell disease, and other anemias.”


Zeidan AM, Fradette C, Rozova A, et al. Safety of deferiprone in patients with myelodysplastic syndromes: results from the Deferiprone US Safety Registry and a Compassionate Use Program. Abstract #462. Presented at the 64th American Society of Hematology Annual Meeting, December 10-13, 2022; New Orleans, Louisiana.

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