Françoise Bernaudin, MD: A Decade of Follow-up Reveals allo-SCT Superiority Over SOC for Sickle Cell Anemia

Over 10 years of follow-up, allogeneic stem cell transplantation (allo-SCT) demonstrated improvements compared with standard of care (SOC) for patients with sickle cell anemia (SCA) in silent cerebral infarct (SCI) incidence and social quality of life (QoL), working memory, and processing speed.

These findings, from the DREPAGREFFE-2 study (NCT05053932), were presented at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 7-10, 2024, in San Diego, California, by 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.

“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,” Bernaudin told HCPLive® during the meeting.

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).

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