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Allogeneic stem cell transplantation (allo-SCT) was more efficacious than standard of care in pediatric patients with sickle cell anemia who had a history of abnormal cerebral artery velocities. Allo-SCT was associated with reduced hospitalization and vaso-occlusive crises, and an improved QOL after 10 years of follow-up.
The observational DREPAGREFFE-2 trial (NCT05053932) assessed 67 pediatric patients between 5 and 15 years of age with sickle cell anemia and a history of abnormal cerebral artery velocities1. All patients had at least one sibling without sickle cell anemia. The 32 patients with a matched sibling donor received a transplant, while the other 35 were maintained on chronic transfusion. If patients in the ‘chronic transfusion’ group reached normalized velocities and did not have stenosis, they were switched to hydroxyurea.
After 10 years of follow-up, the mean number of hospitalizations was 0.2 in the allo-SCT group and 6.1 in the standard-of-care group (P<0.001). Also, the mean number of vaso-occlusive crises was lower in the allo-SCT group than in the standard of care group (0.0% vs 3.8%; P=0.001). Francoise Bernaudin, MD, from the Center Hospitalier Intercommunal Creteil, in France, added that patients who switched to hydroxyurea had worse outcomes after hospitalization or vaso-occlusive crises than patients who remained on chronic transfusion. “Next, patients in the allo-SCT arm had significantly better functioning at school, physical functioning, and even social functioning than patients on standard of care,” mentioned Dr. Bernaudin. Finally, patients who were stroke-free at baseline (n=60) benefited from allo-SCT in terms of reduced intracranial stenosis, decreased ischemic lesions, and enhanced cognitive performance.
“In children with sickle cell anemia and a history of abnormal cerebral artery velocities, allo-SCT was more efficacious than standard of care across various important outcomes,” concluded Dr. Bernaudin. “Early allo-SCT is useful in patients with sickle cell anemia and matched sibling donors in case they have abnormal cerebral artery velocities, severe baseline hemolytic anemia, crises, or complications despite hydroxyurea treatment. In patients without matched sibling donors, haplo-identical SCT or gene therapy should be considered in case of persistent/worsening cerebral vasculopathy or disease progression, respectively.”
Medical writing support was provided by Robert van den Heuvel.
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