What are the AHA/ASA treatment guidelines for children with sickle cell disease at high risk for stroke if RBC transfusion is contraindicated?

Updated: May 12, 2021
  • Author: Joseph E Maakaron, MD; Chief Editor: Emmanuel C Besa, MD  more...
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According to the AHA/ASA primary prevention guidelines, hydoxyurea or bone marrow transplantation might be an option for children at high risk for stroke in whom RBC transfusion is contraindicated. [98] For children with a human leukocyte antigen (HLA)–matched sibling, a consortium has demonstrated that the risk of recurrent stroke can be greatly reduced with allogeneic bone marrow transplantation. This offers an alternative to long-term transfusion and iron chelation. [99]

The Stroke With Transfusions Changing to Hydroxyurea (SWiTCH) trial documented no strokes in patients with SCD (n=66) who received monthly transfusions plus daily deferasirox iron chelation but seven strokes in patients (n=67) treated with hydroxyurea plus overlap transfusions during dose escalation to maximum tolerated dose, followed by monthly phlebotomy. Although the stroke percentage with hydroxyurea/phlebotomy was within the noninferiority stroke margin, the National Heart, Lung, and Blood Institute closed SWiTCH after interim analysis revealed equivalent liver iron content in the two groups, indicating futility for the composite primary end point. The SWiTCH investigators concluded that “transfusions and chelation remain a better way to manage children with SCA, stroke, and iron overload”. [100]

In the TCD With Transfusions Changing to Hydroxyurea (TWiTCH) trial, hydroxycarbamide treatment was found to be noninferior to transfusion therapy for maintaining TCD velocities and helping to prevent primary stroke. TWiTCH was conducted in high-risk children with sickle cell anemia and TCD velocities ≥200 cm/s who had received at least 1 year of transfusions and had no severe vasculopathy identified on magnetic resonance angiography. [101]

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