Allogeneic stem cell transplantation (allo-SCT) has demonstrated long-term superiority over standard of care (SOC) in patients with sickle cell anemia (SCA), according to 10-year follow-up data from the DREPAGREFFE-2 clinical trial (NCT05053932). The study, presented at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition, highlighted significant improvements in silent cerebral infarct (SCI) incidence and social quality of life (QoL) among SCA patients receiving allo-SCT compared to those receiving standard care.
Key Findings from DREPAGREFFE-2
Françoise Bernaudin, MD, head of the Referral Center for Sickle Cell Disease and Clinical Research Department at Hôpital Intercommunal de Créteil of the Université Paris Cité, presented the findings, emphasizing the importance of these long-term outcomes. The original prospective DREPAGREFFE-1 trial (NCT01340404) showed modest improvements between allo-SCT and SOC arms, many of which became significant over the 10-year follow-up period.
Notably, MRI and MRA results indicated that in the SOC group, 11 out of 31 patients reported SCIs, an increase of 5 incidences from the initial 6 at enrollment. In contrast, the allo-SCT group showed no additional instances of SCI, maintaining 12 cases at enrollment. Furthermore, SCI visibility improved in 1 patient in the standard of care group and 4 in the allo-SCT group (P = .010).
Cognitive and Social Benefits
Cognitive performance, specifically processing speed index (PSI), was significantly higher in the allo-SCT group, with a score of 96.5 (SD, 21.9) compared to 83.7 (SD, 14.4) in the standard of care group (P = .035). Social functioning QoL also favored the allo-SCT group, with a score of 93.8 (SD, 10.7) versus 86.4 (SD, 12.9) in the standard of care group (P = .003).
Clinical Implications
"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," said Dr. Bernaudin. These results suggest that allo-SCT offers a more effective long-term solution for managing SCA, particularly in preventing cerebral complications and enhancing overall patient well-being.
