Françoise Bernaudin, MD, on alloSCT Superiority Over SOC for Sickle Cell Anemia

This interview originally appeared on our sister site, HCPLive®.

“It's important we have demonstrated that SCT does better than chronic transfusion and better than hydroxyurea, for the outcome of stenosis, to prevent SCI, and to give a better quality of life and higher cognitive performance."

In the DREPAGREFFE-2 clinical trial (NCT05053932) allogeneic stem cell transplantation (allo-SCT) showed improvements over standard of care (SOC) in silent cerebral infarct (SCI) incidence and social quality of life (QoL), working memory, and processing speed for patients with sickle cell anemia (SCA), over 10 years of follow-up. Françoise Bernaudin, MD, the head of the Referral Center for Sickle Cell Disease and Clinical Research Department at Hôpital Intercommunal de Créteil of the Université Paris Cité, recently presented these results at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 7-10, 2024, in San Diego, California. At the conference, CGTLive®'s sister site, HCPLive®, interviewed Bernaudin to learn more.

The original prospective DREPAGREFFE-1 trial (NCT01340404) showed modest improvements between allo-SCT and SOC arms, many of which have become significant over 10 years of follow-up. Notably, Bernaudin and colleagues found that on MRI and MRA, of the 31 SOC group patients, 11 had reported SCIs—an increase of 5 incidences from 6 at enrollment—while there were no additional instances in the 28 patients in the allo-SCT group, which had 12 at enrollment. SCI were either no more visible or under 3 mm in 1 patient in the standard of care group and 4 in the allo-SCT group (P = .010). Processing speed index (PSI) was higher for the allo-SCT group at 96.5 (SD, 21.9) compared with the standard of care group at 83.7 (SD, 14.4), which was significant (P = .035), as well as social functioning QoL (allo-SCT: 93.8 [SD, 10.7]; standard of care: 86.4 [SD, 12.9]; P = .003).

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REFERENCE
Bernaudin F, Verlhac S, Ducros-Miralles E, et al. Outcome of Cerebral Vasculopathy and Cognitive Performances 10 Years Post-Enrollment in the Drepagreffe Trial Comparing Allogeneic Stem Cell Transplantation to Standard-Care in Children with Sickle Cell Anemia and History of Abnormal Cerebral Velocities. Presented at: ASH 2024 Annual Meeting. December 7-10, 2024; San Diego, CA. Abstract LBA-5