Ferriprox similar to Desferal in easing iron overload in children

Analysis of pediatric SCD patients given either iron chelator in FIRST trial

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by Steve Bryson, PhD |

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Ferriprox (deferiprone) showed comparable efficacy to Desferal (deferoxamine mesylate) in reducing blood transfusion-induced iron overload in children and adolescents with sickle cell disease (SCD) or other anemias, a recent study reported.

Ferriprox is an oral treatment, while Desferal is administered via a subcutaneous (under-the-skin) infusion. As such, Ferriprox is an option “that could improve adherence and outcomes in children,” according to its researchers.

The study, “Deferiprone versus deferoxamine for transfusional iron overload in sickle cell disease and other anemias: Pediatric subgroup analysis of the randomized, open-label FIRST study,” was published in the journal Pediatric Blood & Cancer.

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Oral Ferriprox was compared with Desferal infusion in the Phase 4 study

SCD is marked by genetic mutations affecting hemoglobin, the protein in red blood cells that carries oxygen. Such mutations alter hemoglobin’s structure, causing red blood cells to adopt a sickle-like shape and increasing the risks of blood vessel blockage, which can lead to a vaso-occlusive crisis and other complications.

Blood transfusion is a standard SCD treatment that delivers healthy red blood cells to patients. Still, this can lead to a buildup of iron in the body, or iron overload, that may damage organs.

Iron chelating therapies, which bind to iron and help eliminate it from the body, are often prescribed to patients receiving frequent blood transfusions.

A Phase 3b/4 clinical trial called FIRST (NCT02041299) compared the efficacy and safety of two different iron chelators, Ferriprox and Desferal, in 230 patients ages 3 and older with SCD and other anemias. Ferriprox is marketed by Chiesi Global Rare Diseases, while Desferal is sold by Novartis.

Data showed that Ferriprox was not inferior to Desferal in its ability to lower iron levels in the liver of all participating SCD patients and those with other transfusion-dependent anemias after 12 months.

A global research team led by scientists at Cairo University analyzed FIRST data focusing solely on children and adolescents, ages 3-17, who participated in the study. Among the 142 pediatric patients, 111 (78.2%) were diagnosed with SCD or hemoglobin C disease, and 31 (21.8%) with other anemias.

Most, 94, of these young patients randomly had been assigned to receive Desferal, administered for five to seven days each week as an eight- to 12-hour subcutaneous infusion, and 48 were given Ferriprox orally three times a day.

Iron concentrations in the children’s liver diminished over the study’s year

Across both pediatric groups, there were no significant differences in the ability of Ferriprox and Desferal to reduce iron concentration in the liver. After six months of treatment, the mean concentration dropped by 1.6 mg/g with Ferriprox and by 1.9 mg/g with Desferal. Corresponding changes after 12 months were similar in the two groups.

With Ferriprox, blood levels of ferritin, a protein that stores iron, increased slightly from the study’s start (baseline) to three and six months, and declined after that. By contrast, ferritin levels decreased with Desferal’s use from baseline to three months, and remained stable thereafter.

At three and six months, ferritin levels were significantly higher with Ferriprox than with Desferal, but after 12 months, there were no differences.

A similar proportion of patients experienced at least one adverse event in the Ferriprox and in the Desferal groups (88.3% vs. 87.5%). While no patients on Desferal withdrew due to side effects, five patients (5.3%) receiving Ferriprox did. No deaths were reported.

The most common treatment-related side effects across both groups were upper abdominal pain, vomiting, fever, lower immune neutrophil counts, and elevated levels of liver enzymes. Nearly all events were mild or moderate in severity, and all patients recovered fully.

Serious adverse events were more frequent with Ferriprox than with Desferal (22.3% vs. 14.6%). The most common was sickle cell anemia with pain crisis, which was observed in six Ferriprox group patients and one in the Desferal group. Other serious side effects included fever, low neutrophil count, and upper abdominal pain.

“Consistent with findings from the primary analysis of FIRST, this post hoc subgroup analysis of pediatric patients with SCD and other anemias demonstrated comparable efficacy outcomes between [Ferriprox] and [Desferal] and no new safety concerns,” the researchers wrote.

Four of this study’s 13 authors are Chiesi employees in Canada.