A medical team led by a Korean doctor has succeeded in verifying the effectiveness and safety of an oral iron chelator in treating aplastic anemia a Seoul hospital said Wednesday.
According to Seoul St. Mary’s Hospital, it is the first study of a large number of aplastic anemia patients to find that Deferasirox, the first oral medication approved in the United States, 2005 is effective in treating related iron overland.
Patients who receive long-term blood transfusions for conditions such as beta-thalassemia and other chronic forms of anemia are at risk of developing chronic iron overload in their organs.
In order to prevent complications such as cirrhosis, heart failure and diabetes, common treatment has thus far been intravenous or hypodermic injections that cause severe pain in patients.
The team studied the level of serum ferritin, an indicator measuring iron overload in body, of 116 aplastic anemia patients for one year while treating them with Deferasirox, which is marketed as Exjade by its manufacturer Novartis.
Within one year of treatment, their serum ferritin level on average decreased significantly from 3,254 ng/mL to 1,854 ng/mL, the team said.
The study also found that lower doses of the medicine effectively reduced iron in people who receive blood transfusion less frequently, while a medically meaningful iron reduction was confirmed in long-term transfusion patients when they increased doses every three months.
The participants also did not report severe drug-related side effects other than common mild symptoms such as nausea and diarrhea.
“We have confirmed that iron can be effectively reduced in the body by controlling the frequency and doses of the medicine,” said Lee Jong-wook, hematology professor at Seoul St. Mary’s hospital, who led the international study.
“There have been no clear guidelines to treat aplastic anemia patients who suffer from iron overload. Our findings are expected to be used as standard guidelines for iron chelation therapy.”
The study results were also published in the November issue of Blood, a weekly journal published by the American Society of Hematology.
In Korea, the prevalence rate of aplastic anemia is 5.1 per 1 million people, more than double the 2 per million people in Europe.
