What is the efficacy of hydroxyurea therapy for stroke prevention in patients with SCA?
The efficacy of hydroxyurea therapy for stroke prevention in patients with SCA is promising, but additional study is needed. Results from the ongoing Transcranial Doppler With Transfusions Changing to Hydroxyurea (TWiTCH) trial may offer further insights into the benefits of hydroxyurea in stroke prevention. In the SWiTCH study, a phase III noninferiority trial comparing standard treatment (transfusions/chelation) with alternative treatment (hydroxyurea/phlebotomy) for children with SCA, stroke, and iron overload, was stopped for safety reasons when adjudication reported no strokes in patients on transfusions/chelation but a 10% stroke rate in patients on hydroxyurea/phlebotomy. Furthermore, the phase III Pediatric Hydroxyurea Clinical Trial (BABY HUG) demonstrated significantly lower increases in TCD velocities in the hydroxyurea group, but neurocognitive testing of the infants was not statistically different between groups.