Kidney Disease Research Updates
Spring/Summer 2008

Research News

Effective Treatment for Sickle Cell Disease is Underutilized, NIH Panel Finds

An independent panel convened by the National Institutes of Health (NIH) concluded that hydroxyurea, an effective drug for treating pain and complications from sickle cell disease, is being underused and that its use should be increased among adults and teenagers with the illness.

The panel, which met for 3 days as part of the NIH Consensus Development Program process, stopped short of advocating the drug’s use in children, noting that evidence of its efficacy in this population is not as strong as in adults but that emerging data are positive.

“The compelling benefits of hydroxyurea warrant increased adoption of this drug as a frontline therapy in adults with sickle cell disease,” said Otis Brawley, M.D., the panel chair and Professor of Hematology, Oncology, Medicine, and Epidemiology at Emory University.

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Director Griffin P. Rodgers, M.D., M.A.C.P., a pre-eminent expert in sickle cell disease and the first hematologist to head the NIDDK, set the stage for the consensus development conference with introductory remarks. The NIDDK, which funds research on sickle cell and other blood diseases, was one of the co-sponsors of the consensus development conference.

“At present, hydroxyurea is the major medical modality with proven efficacy in patients with frequent symptoms related to sickle cell disease, although there is increasing evidence that hydroxyurea is prescribed to only a fraction of patients who may benefit from it,” said Rodgers. While gene therapy for sickle cell disease may eventually supplant the need for hydroxyurea treatment by correcting the underlying genetic defect that causes this disease, curative gene therapy has proven to be an “elusive therapeutic holy grail,” according to Rodgers. However, “current genomic studies should provide more insights on directing strategies to resolve the therapeutic challenges of sickle cell disease.”

Safety Concerns

Patient and health care provider concerns about the safety and side effects of hydroxyurea, an anti-cancer drug approved by the U.S. Food and Drug Administration in 1998 to treat adults with sickle cell anemia, have hindered its use despite evidence that the drug’s benefits outweigh the risks for adults with the disease. Side effects of the drug include possible lower sperm count in men; increased risk of bleeding, infection, and malignancies; and the potential for birth defects in babies born of women who take the drug during pregnancy.

Sickle cell anemia is a form of sickle cell disease, an inherited blood disorder that causes red blood cells to lose oxygen and become crescent- or sickle-shaped. The misshapen cells clump together and stick to blood vessel walls, restricting blood flow to limbs and vital organs and causing acute pain and permanent damage to the bones, brain, eyes, heart, lungs, kidneys, liver, and spleen.

Sickle cell disease affects an estimated 50,000 to 100,000 people in the United States and is most common among families from Africa, the Caribbean, Central or South America, India, the Mediterranean, and Saudi Arabia.

The NIH created the Consensus Development Program in 1977 to impartially judge controversial topics in medicine and public health. The panel’s statement on hydroxyurea is not a policy statement of the NIH or the Federal Government. For a copy of the panel’s complete consensus statement, go to www.consensus.nih.gov.

NIH Publication No. 08–4531
July 2008

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