Specific dosage of sickle cell drug increases survival rate

WHAT: An analysis by National Institutes of Health researchers has shown that people with sickle cell anemia who took the drug hydroxyurea at the recommended dose had higher survival rates than those who took less than the recommended dose. The findings appear in the journal PLOS ONE.

Researchers at the National Heart, Lung and Blood Institute (NHLBI) and the National Institute of Diabetes and Digestive and Kidney Diseases reviewed data from 383 people who came to NIH for treatment or evaluation for sickle cell anemia between 2001 and 2010. The study found that 66 percent of people were taking hydroxyurea. Of the group taking hydroxyurea, only two-thirds (or 44 percent of all patients) were using doses high enough to fall within the recommended range.  People taking the recommended dose were 64 percent less likely to die from sickle cell anemia compared to those not taking hydroxyurea.  This survival benefit was not observed in those taking less than the recommended dose of hydroxyurea.  Hydroxyurea is the only FDA-approved drug to treat sickle cell anemia, a rare blood disorder.

Though previous research has shown hydroxyurea to be highly effective in managing the complications of sickle cell anemia, many people with the disease are not consistently receiving this treatment. The analysis suggests that many patients who take hydroxyurea should gradually increase their dose levels as tolerated based upon the desired effect and side effects.

About 70,000 to 100,000 Americans have sickle cell anemia, which can lead to complications including organ damage and intense pain. African Americans make up the majority of those with the disease, though there are also many people with this disease who come from Hispanic, southern European, Middle Eastern, or Asian Indian backgrounds. There is no widely available cure, though NIH researchers have made major advances toward this goal.

WHO:  James G. Taylor VI, M.D. and Courtney Fitzhugh, M.D., Division of Intramural Research, NHLBI, NIH, are available to comment on the findings and implications of this research.

CONTACT: For more information or to schedule an interview, please contact the NHLBI Office of Science Policy, Engagement, Education, and Communications at 301-496-4236 or nhlbi_news@nhlbi.nih.gov (link sends e-mail).