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news.Treating babies suffering with sickle cell anemia with oral liquid hydroxyurea appears to prevent the onset of long-term complications triggered by the disease, according to results of a preliminary study by investigators at St. Jude Children’s Research Hospital.
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The study’s findings are important because the onset of damage caused by sickle cell anemia (SCA) complications can occur as early as three months after birth. Starting treatment before those complications begin could dramatically reduce the chance of organ damage and premature death.
Hydroxyurea increases the production of fetal hemoglobin (HbF), the main oxygen transport protein in fetal red blood cells. Because HbF prevents red blood cells from “sickling”, clinicians have used hydroxyurea for about a decade to reactivate HbF production in adults and older children with SCA.
In addition, hydroxyurea reduces the severity of symptoms suffered by adolescents and adults with SCA, such as lung infections, organ damage, stunted growth, impaired brain development and acute chest syndrome (ACS).
The current St. Jude study was an extension of a previous clinical trial, Hydroxyurea Safety and Organ Toxicity (HUSOFT), which was the first in which young babies were treated with hydroxyurea. The original HUSOFT study, published in 2001, demonstrated that short-term oral liquid hydroxyurea therapy can be safe and effective in babies with SCA.
The aim of the HUSOFT Extension was to determine if this higher dose, given for an extended period of time, provided significant long-term benefits without causing unacceptable side effects in children ranging in age from 2.6 to 4.4 years (median age 3.4 years).
After four years of hydroxyurea therapy, the concentrations of Hb, HbF and the volume of red blood cells were significantly increased in the children receiving hydroxyurea. Moreover, the HbF level often exceeded 20% of the total amount of Hb.
Dr Jane Hankins, a physician at the St. Jude Comprehensive Sickle Cell Center and the study’s lead author said: “In children who weren’t treated with hydroxyurea, the level of HbF declined significantly. The fact that HbF levels rose in babies treated with hydroxyurea suggests that the drug is effective in babies, as well as in adolescents and adults.”
“Our results are promising and justify a larger multicenter clinical trial to confirm that treating babies with hydroxyurea is safe and effective,” said Dr Hankins added.