ASH: Oral drug offers alternative to lifelong transfusions in sickle cell

Abnormal transcranial Doppler findings in sickle cell disease indicates that the pediatric patient is at high risk of a primary stroke. To date, the standard of care has been lifelong transfusion for these patients.

This study indicates there may be a viable alternative to transfusions with less invasive therapy and reduced risk of iron loading. What is not addressed, however, is whether we use hydroxyurea as front line therapy for children with abnormal findings on transcranial Doppler. The study patients had been on transfusions for at least 12 months when they were enrolled in the study and had normalized velocities on transcranial Doppler.

Do we still need to screen sickle cell disease patient patients with transcranial Doppler for stroke risk? The answer is 'yes.' What if they are already on hydroxyurea? Still the answer is 'yes.'

Should transcranial Doppler screening also be linked with MRI/MRA to look at vessels? This study refers to a select group of patients with no significant vasculopathy noted on brain imaging.

So how does the community provider interpret these finding for the real-world care of a patient? How do we not over interpret or under interpret the data? In partnership with a hematologist each child should continue to get transcranial Doppler screening annually and transfusions initiated for abnormal transcranial Doppler and study methods should be followed with crossing over to hydroxyurea for patients who normalize their transcranial Doppler results at 12 months and have no evidence of significant vasculopathy on brain imaging.

Transcranial Doppler screening should continue annually per guidelines even if the patient is already on hydroxyurea for other indications or was switched to hydroxyurea from transfusions. This is a situation that is more likely to occur in the real world clinical setting as hydroxyurea use is advocated for children as early as 9 months of age.

What happens then if transcranial Doppler findings become abnormal while on hydroxyurea? If compliance is assured and the hydroxyurea dose is optimized, then we are back to square one. Primary stroke prevention with lifelong transfusion is currently the standard of care. At least until another randomized study is done to prove non inferiority.

Dr. Ifeyinwa Osunkwo is the medical director for the sickle cell program at the Levine Cancer Institute, Carolinas Healthcare Systems, Charlotte, NC. and a member of the editorial board for Hematology News.