Relapse and regimen-related toxicities remain major challenges in achieving long-term survival, particularly among older patients with high-risk myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT). Previous studies have demonstrated the feasibility of treosulfan-based conditioning, noting stable engraftment and low non-relapse mortality (NRM) in patients undergoing HLA-matched allo-HSCT. However, data on treosulfan-based conditioning in the HLA-haploidentical transplantation (HaploT) setting are limited. We retrospectively compared conditioning with fludarabine–cyclophosphamide (FC)–melphalan (110 mg/m2) and FC-treosulfan (30 g/m2) prior to HaploT using post-transplantation cyclophosphamide (PTCy) in patients with high-risk MDS/AML patients ≥ 50 years, transplanted from 2009–2021 at our institution (n= 80). After balancing patient characteristics by a matched-pair analysis, we identified twenty-one matched pairs. Two-year OS and LFS were similar among the groups (OS 66% and LFS 66%,p= 0.8 andp= 0.57). However, FC-melphalan was associated with a significantly lower probability of relapse compared to FC-treosulfan (0% vs. 24%,p= 0.006), counterbalanced by a higher NRM (33% vs. 10%,p= 0.05). Time to engraftment and incidences of acute and chronic graft-versus-host disease (GvHD) did not differ significantly. In conclusion, HaploT using FC-treosulfan in combination with PTCy in patients aged ≥50 years with MDS/AML appears safe and effective, particularly in advanced disease stages. We confirm the favorable extramedullary toxicity profile, allowing for potential dose intensification to enhance antileukemic activity.
复发及预处理方案相关毒性仍是实现长期生存的主要挑战，尤其对于接受异基因造血干细胞移植（allo-HSCT）的高危骨髓增生异常综合征（MDS）或急性髓系白血病（AML）老年患者。既往研究已证实基于曲硫仑预处理方案的可行性，指出在接受HLA全相合allo-HSCT的患者中可实现稳定植入且非复发死亡率（NRM）较低。然而，在HLA单倍体相合移植（HaploT）中应用曲硫仑预处理方案的数据仍有限。本研究回顾性比较了2009-2021年间在本中心接受HaploT的80例≥50岁高危MDS/AML患者，在移植后环磷酰胺（PTCy）方案背景下，采用氟达拉滨-环磷酰胺（FC）-美法仑（110 mg/m²）与FC-曲硫仑（30 g/m²）两种预处理方案的效果。通过配对分析平衡患者特征后，共纳入21组配对病例。两组患者的两年总生存率（OS）和无白血病生存率（LFS）无显著差异（OS均为66%，p=0.8；LFS均为66%，p=0.57）。但FC-美法仑组相比FC-曲硫仑组显示出显著更低的复发率（0% vs. 24%，p=0.006），同时伴随更高的NRM（33% vs. 10%，p=0.05）。两组在造血重建时间、急慢性移植物抗宿主病（GvHD）发生率方面均无显著差异。结论表明，对于≥50岁MDS/AML患者，采用FC-曲硫仑联合PTCy的HaploT方案安全有效，尤其在疾病晚期阶段更具优势。本研究证实该方案具有较好的髓外毒性特征，为通过剂量强化增强抗白血病活性提供了可能。
肿瘤(癌症)患者之家