Allo-SCT improves lives of children with sickle cell anaemia and abnormal cerebral artery velocities

Allogeneic stem cell transplantation (allo-SCT) was more efficacious than standard-of-care in paediatric patients with sickle cell anaemia who had a history of abnormal cerebral artery velocities. Allo-SCT was associated with a reduction in hospitalisation and vaso-occlusive crises (VOCs) and an improved quality-of-life after 10 years of follow-up.

The observational DREPAGREFFE-2 trial (NCT05053932) assessed 67 paediatric patients between 5 and 15 years of age with sickle cell anaemia and a history of abnormal cerebral artery velocities [1]. All patients had at least 1 sibling without sickle cell anaemia. The 32 patients with a matched sibling donor received a transplant, while the other 35 were maintained on chronic transfusion. Patients in the chronic transfusion group who reached normalised velocities and did not have stenosis were switched to hydroxyurea.

After 10 years of follow-up, the mean number of hospitalisations 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 VOCs was lower in the allo-SCT group than in the standard-of-care group (0.0% vs 3.8%; P=0.001). Dr Françoise Bernaudin (Centre Hospitalier Intercommunal Creteil, France) added that patients who switched to hydroxyurea had worse outcomes after hospitalisation or VOCs than those 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) benefitted from allo-SCT in terms of reduced intracranial stenosis, decreased ischaemic lesions, and enhanced cognitive performance (see Table).

Table: Cognitive performance in stroke-free patients [1]



“In children with sickle cell anaemia 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 anaemia and matched sibling donors in case they have abnormal cerebral artery velocities, severe baseline haemolytic anaemia, or 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.”

1. Bernaudin F, et al. Outcome of cerebral vasculopathy and cognitive performance a decade later in the DREPAGREFFE trial comparing allogeneic stem cell transplantation to standard of care in children with sickle cell anemia and history of abnormal cerebral velocities. Late-breaking Abstract 5, 66^th ASH Annual Meeting, 7–10 December 2024, San Diego, CA, USA.

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Posted on 29 January 202529 January 2025