A New Drug for Sickle Cell Disease

Decitabine may offer alternative treatment for people with blood disease

MONDAY, Nov. 24, 2003 (HealthDayNews) -- The drug decitabine may offer an alternative for people with sickle cell disease who can't tolerate current standard treatment with the drug hydroxyurea, says a study in the Dec. 1 issue of Blood.

Hydroxyurea reactivates fetal hemoglobin, which is known to decrease complications caused by sickle cell disease. However, not all patients respond well to the medication.

Sickle cell disease is an inherited disorder that causes bone marrow to produce red blood cells with defective hemoglobin, causing the red blood cells to become sickle-shaped.

These abnormally shaped red blood cells have difficulty passing through blood vessels. This results in a number of potential problems such as severe pain, recurrent pneumonia and stroke.

The study included eight patients with several clinically significant complications of sickle cell disease. Three of the patients had been taking hydroxyurea for more than a year without a significant increase in fetal hemoglobin or decreased symptoms. The remaining five patients had to stop using hydroxyurea due to complications.

During the study, the patients received 0.2 milligrams/kilogram of decitabine one to three times a week in two six-week cycles. There was a two-week interval between the cycles.

All the patients showed an increase in fetal homglobin levels and improvement in markers of disease activity.

"Decitabine induces the production of fetal hemoglobin, probably through a different mechanism than hydroxyurea. These results are exciting as patients who do not respond to hydroxyurea need alternative therapies that can increase the quality and quantity of their lives," study author Dr. Yogen Saunthararajah, of the University of Illinois at Chicago, says in a prepared statement.

"Further studies with decitabine should be performed to demonstrate if this agent, administered over prolonged periods of time, can improve the quality of life for patients with sickle cell disease," Saunthararajah adds.

More information

Here's where you can learn more about sickle cell disease.

SOURCE: American Society of Hematology, news release, November 2003
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