Pinto Filipe R, Suárez-Lledó María, Guardia Laia, Charry Paola, Cid Joan, Lozano Miquel, Pedraza Alexandra, de Llobet Noemi, Corrius Gerard, Moreno Cristina, Esteve Jordi, Serra Carles, Carreras Enric, Rosiñol Laura, Fernández-Avilés Francesc, Rovira Montserrat, Martinez Carmen, Salas María Queralt
Clinical Hematology Department, Unidade Local de Saúde de Santo António-Hospital de Santo António, Oporto, Portugal.
Hematopoietic Transplantation Unit, Hematology Department, Clinical Institute of Hematology and Oncology (ICMHO), Hospital Clínic de Barcelona, Barcelona, Spain.
Eur J Haematol. 2025 Apr;114(4):650-662. doi: 10.1111/ejh.14378. Epub 2024 Dec 30.
Although post-transplant cyclophosphamide (PTCY)-based prophylaxis has become a widely adopted strategy for preventing graft-versus-host disease (GVHD) in 9 out of 10 HLA-mismatched unrelated donors (MMUDs), allogeneic hematopoietic cell transplants (allo-HCTs), data on the safety and efficacy of PTCY in this setting remain limited. This single-center study investigates the outcomes of 94 adults with hematological malignancies undergoing MMUD allo-HCT with PTCY and tacrolimus (Tac) (PTCY-Tac) between 2014 and 2023. The median age was 53 years, and 60.6% were male. Peripheral blood stem cells were infused in all cases. By Day +100, the cumulative incidence of Grades II-IV and Grades III and IV acute GVHD were 33.0% and 9.7%, with 2-year incidence of moderate-to-severe chronic GVHD at 12.6%. By Day +30, 40.8% of patients experienced bacterial bloodstream infections, and 52.4% had cytomegalovirus (CMV) reactivation before letermovir prophylaxis. With letermovir's introduction, CMV reactivation rates dropped significantly, with only one case reported. At 3 years, overall survival was 60.8%, non-relapse mortality was 23%, and the cumulative incidence of relapse was 24.5%. HLA Class I or II mismatches did not affect key outcomes or GVHD rates. These findings demonstrate that PTCY-Tac offers effective GVHD prevention and favorable outcomes in MMUD allo-HCT, supporting its application for patients without fully matched donors.
尽管基于移植后环磷酰胺（PTCY）的预防措施已成为在十分之九的人类白细胞抗原（HLA）不匹配无关供体（MMUD）的异基因造血细胞移植（allo-HCT）中预防移植物抗宿主病（GVHD）的广泛采用策略，但关于PTCY在此种情况下的安全性和有效性的数据仍然有限。这项单中心研究调查了2014年至2023年间94例接受MMUD allo-HCT并使用PTCY和他克莫司（Tac）（PTCY-Tac）治疗的血液系统恶性肿瘤成人患者的结局。中位年龄为53岁，60.6%为男性。所有病例均输注外周血干细胞。到第+100天，II-IV级和III-IV级急性GVHD的累积发生率分别为33.0%和9.7%，中度至重度慢性GVHD的2年发生率为12.6%。到第+30天，40.8%的患者发生细菌血流感染，52.4%的患者在使用来特莫韦预防前出现巨细胞病毒（CMV）再激活。随着来特莫韦的引入，CMV再激活率显著下降，仅报告了1例。3年时，总生存率为60.8%，非复发死亡率为23%，复发累积发生率为24.5%。HLA I类或II类不匹配不影响关键结局或GVHD发生率。这些发现表明，PTCY-Tac在MMUD allo-HCT中提供了有效的GVHD预防和良好的结局，支持其应用于没有完全匹配供体的患者。
