Study at Howard University and Other Institutions Leads to Breakthrough in Treatment of Sickle Cell Anemia


WASHINGTON – Clinical trials conducted at Howard University College of Medicine and 12 other medical institutions across the nation have discovered a treatment that dramatically reduces pain, inflammation and hospitalization time and eases other symptoms in young children suffering from sickle cell anemia.

Doctors and medical clinicians in a four-year study found that using the drug hydroxycarbamide (also called hydroxyurea) in very young children with sickle cell anemia was safe and markedly reduced episodes of pain and other problems.

Hydroxycarbamide has been used in adults for several decades for a variety of cancer treatments and is approved for use in adults with sickle cell anemia.

The findings, which will be published in this week’s edition of The Lancet, the world’s leading medical journal, suggest that use of hydroxycarbamide should now be the standard of care for all children with sickle cell disease.

Dr. Sohail Rana, professor of pediatrics in the Department of Pediatrics at Howard University College of Medicine and director of pediatric hematology at Howard University Hospital, was one of the investigators on the study.

“This is a significant breakthrough in the treatment of this disease, particularly for our very young patients,” Rana said. “To be able to dramatically diminish the pain that these young children suffer because of this disease is truly an important step forward. We hope all clinicians will now adopt this treatment for their patients.”

Howard was one of the leading partners in the study, which was named BABY HUG trial. The study continues to observe children for long term benefits.

Also participating in the study were Children’s National Medical Center in Washington, Johns Hopkins University School of Medicine in Baltimore, St. Jude Children's Research Hospital in Memphis, SUNY Downstate Medical Center, University of Mississippi Medical Center, St. Duke University Medical Center, University of Texas Southwestern Medical Center at Dallas, Medical University of South Carolina, University of Miami, Emory University School of Medicine,  Children’s Hospital of Michigan, University of Alabama at Birmingham, Medical College of Georgia, the National Heart, Lung and Blood Institute and the Clinical Trials & Surveys Corp.

In sickle cell anemia patients, the red blood cells develop abnormal shapes and are unable to pass through small blood vessels and cause blockage. These cells also break down faster than normal blood cells, leading to anemia. Complications include severe pain, stroke, damage to different organs of body and premature death.

The study was conducted between October 2003 and early September 2010 with 193 patients. Half of the patients, ages 9 to 18 months, were given liquid hydroxycarbamide to see if it would safely prevent early organ damage, particularly the spleen and kidney. The others were assigned a placebo, and the patients’ progress was monitored for two years.

Children who received the hydroxycarbamide therapy were half as likely to experience pain, five times less likely to have painful inflammation of the hands or feet and three times less likely to experience acute chest syndrome than the children who did not.?
The doctors and clinicians reported some benefit for spleen, kidney and neurological function, but the results were not conclusive.

They also reported the hydroxycarbamide therapy was well tolerated, with mild-to-moderate decrease in white cells being the only adverse effect associated with treatment.

“The laboratory and clinical benefits of hydroxycarbamide for children and adolescents with sickle-cell anaemia, coupled with an excellent short-term and long-term safety profile, suggest that hydroxycarbamide should become the standard treatment in young patients,” the authors said in their report. “The results of the BABY HUG study should have a major effect on guidelines for the management of children with sickle-cell anaemia… Hydroxycarbamide therapy can now be considered for all very young children with sickle-cell anaemia whether or not they have clinical symptoms.”

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