A 26-site clinical trial in children with sickle cell anemia who had experienced a stroke that compares a new strategy to prevent a second stroke with the current standard of care has been ended early by the National Heart, Lung, and Blood Institute (NHLBI). It was halted because preliminary data suggested that the experimental therapy is unlikely to improve outcomes, according to a statement by the agency.
The Stroke With Transfusions Changing to Hydroxyurea (SWiTCH) trial sought to determine if treatment with hydroxyurea and phlebotomy (to reduce excess iron) would help to prevent future strokes in children with sickle cell anemia who had already experienced one. Study sites had enrolled 133 patients aged 5 through 18 years and randomized them to either the experimental therapy or the standard of care (blood transfusions plus the iron-removal drug deferasirox). However, after the data and safety monitoring board found in an interim analysis that phlebotomy did not reduce liver iron better than deferasirox, it recommended halting the trial, and the NHLBI did so on May 6. Based on the data collected to that point, no strokes occurred in the 66 patients receiving blood transfusions and deferasirox; however, 7 strokes occurred among the 67 patients receiving hydroxyurea and phlebotomy.
Kuehn BM. Sickle Cell Trial Halted. JAMA. 2010;304(4):398. doi:10.1001/jama.2010.968