Extended hydroxyurea therapy boosts outcomes in young sickle cell anemia patients

Taking hydroxyurea over a longer period of time can lead to more significant improvements in patients with sickle cell anemia (SCA), according to new data published in Blood Advances.

Sickle cell disease, a common inherited red blood cell disorder, is seen in approximately 100,000 people in the United States alone. The study's authors evaluated data from 100 SCA patients between the ages of 3 and 22. All patients were treated at Cohen Children’s Medical Center in New York from 2010 to 2017. Half of the study's participants were found to have enlargement or thickening of the left ventricle (LV) of the heart.

Sixty of the study participants were already taking hydroxyurea. There was no difference in the level of LV enlargement for that group of patients compared with the group that was not taking hydroxyurea. However, cardiac outcomes were better among patients taking hydroxyurea for a longer period of time.  

According to the authors, 41.6% of the patients on hydroxyurea were treated for less than one year. That patient cohort had a significantly greater prevalence of LV dilation compared with patients who were treated for more than a year.

“This finding set the stage for the next phase of our research, where we discovered that cardiac measures were actually improving over time on hydroxyurea,” lead author Arushi Dhar, MD, a specialist at Cohen Children’s Heart Center, said in a press release.

An analysis of echocardiograms of patients who were already taking hydroxyurea found that LV dilation and hypertrophy improved significantly. Among the 34 patients who had an abnormal LV mass upon initial testing, 18 had a normal LV on follow-up.