Sickle cell disease is an inherited disorder that causes patients' bone marrow to produce red blood cells with defective hemoglobin, which in turn causes red blood cells to become sickle-shaped. These irregular cells have difficulty passing through blood vessels, causing many symptoms, including excruciating pain episodes, recurrent pneumonias, and strokes.
"The standard treatment for sickle cell is the drug hydroxyurea. Hydroxyurea reactivates fetal hemoglobin, and increased fetal hemoglobin production is known to decrease the complications of sickle cell disease," said Yogen Saunthararajah, M.D., of the University of Illinois at Chicago, the lead author of the study. "Not all patients respond to treatment with hydroxyurea, prompting our team to investigate whether the antimetabolite drug decitabine could be used effectively in place of hydroxyurea for these patients."
To be eligible for this study, patients had to be 18 years of age or older and have sickle cell disease which was symptomatic. In addition, patients had to be either resistant to or intolerant of hydroxyurea. Between November 2001 and February 2002, eight patients were enrolled in the study. All patients had multiple clinically significant complications of sickle cell disease. Three patients had been on hydroxyurea for more than one year and failed to demonstrate a significant increase in fetal hemoglobin or decreased symptoms, and the remaining patients were forced to discontinue use of hydroxyurea due to complications. Decitabine was administered at relatively low doses of 0.2 mg/kg one to three times per week in two six-week cycles, with a two-week interval between cycles. The drug was administered subc
Contact: Aislinn Raedy
American Society of Hematology