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Deferasirox Retains Long-Term Efficacy in Iron Overload: Presented at ASH

By John Gever

ATLANTA, GA -- December 10, 2007 -- The oral iron chelator deferasirox (Exjade) remains effective against iron overload for several years, according to results from studies presented here at the 49th American Society of Hematology (ASH) Annual Meeting and Exposition.

Most notably, deferasirox showed long-term efficacy and safety in patients with sickle cell disease, including adults, and in children as young as 2 years old with various types of transfusion-treated anemia.

The sickle cell results were described in a poster presentation on December 8 at a press briefing by Elliott Vichinsky, MD, Professor of Pediatrics, University of California-San Francisco, San Francisco, California.

Dr. Vichinsky also participated in several additional studies of deferasirox reported in a poster session on December 9.

Deferasirox is administered orally once daily. It was approved in the United States and elsewhere for treating chronic iron overload in patients at least 2 years old based primarily on 1-year studies.

The sickle cell results reported were from a long-term, open-label extension of a 1-year, randomized study that involved 82 children under the age of 16 and 77 patients age 16 and older.

Dr. Vichinsky said it was especially important to include the older subjects in the trial. Sickle cell disease, he said, "is really becoming an adult disease" thanks to improved therapies that allow children to survive into adulthood.

Fifty-three of the patients had received deferoxamine, an infusion drug traditionally used for iron overload, in the randomized study and accepted open-label treatment with deferasirox in the follow-up study. Twenty-six patients assigned to deferasirox in the randomized study declined to participate in the follow-up.

Patients in the study received 5/10, 20, or 30 mg/kg/day of deferasirox. Median duration of open-label treatment was 2.7 years, with data collection still ongoing.

No major differences in serum ferritin levels between children and adults were seen, Dr. Vichinsky said, with both groups showing stable or gradually declining ferritin levels. There was a trend toward increased efficacy with higher doses.

Fifteen patients (9.4%) discontinued treatment: three for adverse effects, two for lack of efficacy, and 10 for other reasons. Nausea, vomiting, diarrhea, rash, and abdominal pain were the most common adverse effects. These were mild to moderate, except for two cases of severe diarrhea.

The adverse effect profile was similar to that seen in the randomized portion, Dr. Vichinsky said.

Another study presented at ASH reported similar results from extensions of four pediatric trials of deferasirox in patients with beta-thalassemia, myelodysplastic syndrome, sickle cell disease, and other types of anemia.

Pooled data from the trials indicated continued efficacy for up to 42 months. Patients receiving the highest dose level (30 mg/kg/day) showed declining levels of serum ferritin through the follow-up period, while ferritin remained stable in those receiving 5 to 20 mg/kg/day.

Another oral iron chelator, deferiprone, is available in numerous countries worldwide but not in the US or Canada.

The study was funded by Novartis, makers of deferasirox.


[Presentation title: Long-Term Efficacy and Safety of Deferasirox (Exjade, ICL670), a Once-Daily Oral Iron Chelator, in Patients With Sickle Cell Disease (SCD). Abstract 3395]

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