(WASHINGTON, September 19, 2005) -- A drug used for the treatment ofsickle cell anemia in adults has now been shown to cause significantimprovements in very young children with the disorder. The finding isan important one as these young patients are especially vulnerable toserious organ failure and even death at an early age. The study resultswill be published in the October 1, 2005, issue of Blood, the official journal of the American Society of Hematology.
Sickle cell anemia is a genetic blood disorder that can cause severepain, fatigue, and organ damage to the kidneys, spleen, and liver. Itoccurs in about one in every 500 African-Americans. In the new study,21 children from two to four years old who had sickle cell anemia weregiven the drug hydroxyurea orally as a flavored liquid formula. Amajority of the children took the drug for at least four years and morethan half of the participants completed all six years of the study.
The treatment was well-tolerated in the patients, with only one child'sdosage permanently reduced during the study due to adverse effects. Thedrug's primary function, to counteract the effects of the disease byincreasing and sustaining fetal hemoglobin production, was achieved inall study participants. Patients treated with hydroxyurea also weighedmore and were taller than untreated children with the disorder -- theirgrowth rates were even comparable to those of normal children.
Another measure of success of the therapy was that the study patientshad improved spleen function, an important finding as many childrenwith sickle cell anemia lose spleen function by two years of age.Participants also experienced significantly fewer incidents of acutechest syndrome, a potentially life-threatening disorder associated withsickle cell disease. During the study, one four-year-old girl died ofsepsis, a toxic bacterial infection, though no increased risk of sepsiswas found among the hydroxyurea-treated patients.
"This study demonstrates that hydroxyurea is an efficient and safetreatment option for young children with sickle cell anemia," said JaneHankins, M.D., M.S., of St. Jude Comprehensive Sickle Cell Center andlead study author. "As sickle cell anemia is a chronic disorder, havinga drug that can be started early and continued long-term with fewadverse effects is of significant importance to the way we treat thisdebilitating disease."
This work was supported by the American Lebanese Syrian AssociatedCharities and by General Clinical Research Center grants from theNational Institutes of Health.
The American Society of Hematology (www.hematology.org)is the world's largest professional society concerned with the causesand treatment of blood disorders. Its mission is to further theunderstanding, diagnosis, treatment, and prevention of disordersaffecting blood, bone marrow, and the immunologic, hemostatic, andvascular systems, by promoting research, clinical care, education,training, and advocacy in hematology.
Blood, the official journal of the American Society of Hematology, is the most cited peer-reviewed publication in the field. Blood is issued to Society members and other subscribers twice per month, available in print and online at www.bloodjournal.org.
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