Adults should be treated with the maximum tolerated hydroxyurea dose, ideally before organ damage occurs, according to a study of 383 adults with homozygous sickle cell disease, including 59 who died during follow-up. Researchers found:

• Deceased patients had more hepatic dysfunction, kidney dysfunction, and cardiopulmonary dysfunction vs surviving patients.

• 66% of patients received hydroxyurea therapy, but only 66% of those received a dose within the recommended therapeutic range.

• Hydroxyurea use was associated with improved survival (HR=0.58); an effect most pronounced among those taking the recommended dose.

• Hydroxyurea use was not associated with changes in organ function over time.

• Patients with higher fetal hemoglobin responses to hydroxyurea were more likely to survive.

• Although alkaline phosphatase was lowest in patients with the best fetal hemoglobin response, other markers of organ damage were not consistently improved over time in patients with the highest fetal hemoglobin levels.

Citation: Fitzhugh CD, Hsieh MM, Allen D, et al. Hydroxyurea-increased fetal hemoglobin is associated with less organ damage and longer survival in adults with sickle cell anemia. PLoS One. 2015;10(11):e0141706. doi: 10.1371/journal.pone.0141706.