Lopez-Corral L, Blázquez-Goñi C, Pérez-López E, Martín-Domínguez F M, Cabero Martínez A, Rodríguez-Torres N, Cabrero M, Espigado-Tocino I, Martín López A A, Parody-Porras R, Baile González M, Caballero-Velázquez T, Cortés-Rodríguez M, Soria-Saldise E, Avendaño Pita A, Alcalde-Mellado P, García Bacelar A, Rodríguez-Arbolí E, López Parra M, Falantes-González J F, Navarro-Bailón A, Vázquez López L, Escamilla-Gómez V, Sánchez-Guijo F, Pérez-Simón J A
Hematology Department, Complejo Asistencial Universitario de Salamanca-IBSAL; Departamento de Medicina, Universidad de Salamanca, Centro de Investigación del Cáncer-IBMCC, CIBERONC, Salamanca, Spain.
Hematology Department, Hospital Universitario Virgen del Rocío, Instituto de Biomedicina de Sevilla (IBiS)/CSIC, Universidad de Sevilla, de Sevilla, Spain.
Bone Marrow Transplant. 2025 Apr 8. doi: 10.1038/s41409-025-02562-w.
We present the largest prospective real-world experience in 159 patients who received the triple combination of tacrolimus/sirolimus/mycophenolate mofetil after reduced intensity conditioning allogeneic hematopoietic stem cell transplantation (RIC-alloHSCT) from matched-related (MRD), matched-unrelated (MUD) or mismatched-unrelated donors (MMURD). Despite the high-risk and elderly population, non-relapse mortality (NRM) at day +100 and 1 year was 5.1% and 8.6%. Grades 2-4 and 3-4 acute Graft-versus-host disease (GvHD) at day +180 was 30.3% and 13%, respectively. Chronic GvHD at 1 and 3 years was 23.2% and 41% and for moderate/severe was 13.2% and 26.6%, respectively. With a median follow-up of 20 months, the 1- and 3-year progression-free survival was 60% and 49%, the GvHD-free relapse-free survival was 44% and 32%, and the overall survival was 70.3% and 61%, respectively, for the entire cohort. Patients receiving allo-HSCT from MMURD showed a higher incidence of aGvHD with impact on survival endpoints. GvHD prophylaxis with the triple-drug combination tacrolimus/sirolimus/mycophenolate mofetil showed excellent results in terms of NRM, GvHD and survival in a high-risk, frail and elderly population in the context of RIC-HSCT from MRD and MUD. The subgroup of patients receiving RIC-HSCT from MMURD might probably benefit from other prophylaxis strategies.
我们展示了159例患者的最大规模前瞻性真实世界经验，这些患者在接受来自匹配相关（MRD）、匹配无关（MUD）或不匹配无关供者（MMURD）的减低强度预处理异基因造血干细胞移植（RIC-alloHSCT）后，接受了他克莫司/西罗莫司/霉酚酸酯三联组合治疗。尽管患者为高危和老年人群，但在+100天和1年时的非复发死亡率（NRM）分别为5.1%和8.6%。在+180天时，2-4级和3-4级急性移植物抗宿主病（GvHD）分别为30.3%和13%。1年和3年时慢性GvHD发生率分别为23.2%和41%，中度/重度慢性GvHD发生率分别为13.2%和26.6%。中位随访20个月时，整个队列的1年和3年无进展生存率分别为60%和49%，无GvHD无复发生存率分别为44%和32%，总生存率分别为70.3%和61%。接受MMURD的allo-HSCT患者的aGvHD发生率较高，对生存终点有影响。在来自MRD和MUD的RIC-HSCT背景下，他克莫司/西罗莫司/霉酚酸酯三联药物组合预防GvHD在高危、体弱和老年人群的NRM、GvHD和生存方面显示出优异结果。接受MMURD的RIC-HSCT患者亚组可能会从其他预防策略中获益。
