Jane S. Hankins, MD, MS

    Jane S. Hankins, MD, MS

    Hydroxyurea safe and effective for sickle cell babies

    Treating babies who have sickle cell anemia (SCA) with oral liquid hydroxyurea appears to prevent the onset of long-term complications triggered by this disease, according to results of a preliminary study by St. Jude  investigators. In the United States, SCA is found mostly among African-Americans.

    The onset of damage caused by SCA complications can occur as early as three months after birth, the researchers said. Starting treatment at a younger age may prevent serious complications such as pain, anemia, lung complications and other organ damage. A report on the study appears in the June 14 online issue of Blood.

    Hydroxyurea increases the production of fetal hemoglobin (HbF), preventing the hallmark sickling of red blood cells by reactivating HbF production in adults and older children with SCA, the researchers noted.  In babies and young children with SCA, hydroxyurea may prevent the HbF levels from falling, thereby protecting the red blood cells from sickling.

    The current St. Jude study was an extension of a previous St. Jude clinical trial, known as the Hydroxyurea Safety and Organ Toxicity (HUSOFT) study.  This original study, published in 2001, demonstrated that short-term oral liquid hydroxyurea therapy can be safe and effective in babies with SCa. The extension study followed these infants for up to six years of therapy.

    “Our results are very promising and justify a larger multicenter clinical trial to confirm that this approach is safe and effective,” said Jane Hankins, MD, a physician at the St. Jude Comprehensive Sickle Cell Center and lead author of the study.

    “This study is particularly encouraging because it suggests that we can treat babies with hydroxyurea for several years without serious side effects,” said Winfred Wang, MD, director of the St. Jude Comprehensive Sickle Cell Center. Wang is the senior author of the paper in Blood.

    The aim of the HUSOFT Extension was to determine if higher doses, given over a prolonged period of time, provided significant long-term benefits without causing unacceptable side effects in very young children, who had a median age of 3.4 years at enrollment into the extension study.

    The study demonstrated the concentrations of Hb, HbF and the volume of red blood cells were significantly increased in the children receiving hydroxyurea. “These results are particularly important because we know that the level of HbF declines significantly in young children with sickle cell anemia who don’t receive hydroxyurea therapy,” said Hankins.

    “Treatment with hydroxyurea requires periodic checkups, but the medication is relatively inexpensive and could prove to be an effective way to improve the care of sickle cell anemia patients who live in underprivileged areas of the world,” said Russell Ware, MD, PhD, director of the Hematology division at St. Jude. Ware is a co-author of the paper.

    Other authors of the article include Lynn Wynn, Hematology-Oncology; Zora Rogers of the University of Texas in Dallas; Peter Lane of Emory University in Atlanta, Georgia; and J. Paul Scott of the Medical College of Wisconsin in Milwaukee.


    Last update: July 2005