Background: Post-transplant cyclophosphamide (PTCy) is a standard graft-versus-host disease (GVHD) prophylaxis in allogeneic hematopoietic cell transplantation (allo-HCT). While effective, concerns remain about cyclophosphamide-related cardiotoxicity, especially in patients with pre-existing cardiac morbidity, a population often underrepresented in clinical trials. Objectives: To assess the incidence and outcomes of early (ECE, ≤100 days) and late (LCE, >100 days) cardiac events in acute myeloid leukemia (AML) patients with and without baseline cardiac morbidity undergoing allo-HCT with PTCy. Study Design: Retrospective multicenter study by the Grupo Español de Trasplante Hematopoyético y Terapia Celular (GETH-TC) including 461 AML patients (62 with cardiac morbidity) transplanted between 2012 and 2022. Cardiac morbidity was defined by documented cardiac disease or left ventricular ejection fraction < 45%. Cumulative incidence, overall survival (OS), and non-relapse mortality (NRM) were analyzed using competing risks models and adjusted with propensity score matching (PSM) and inverse probability weighting (IPW). Results: Cardiac events occurred in 13.2% of patients: 11% vs. 7% ECE (p= 0.93) and 8% vs. 5.3% LCE (p= 0.85) in those with vs. without cardiac morbidity. Most ECEs were arrhythmias or heart failure. Adjusted analyses confirmed no significant differences in CE incidence, OS, or NRM between groups. Two-year OS was 69% vs. 70% (p= 0.50); NRM was 18% vs. 17% (p= 0.20). ECE was associated with higher mortality in both groups. Conclusions: PTCy is feasible in AML patients with pre-existing cardiac morbidity when combined with comprehensive cardiovascular evaluation and cardio-oncology follow-up, supporting its safe use in broader patient populations with appropriate cardiologic support.
背景：移植后环磷酰胺（PTCy）已成为异基因造血细胞移植（allo-HCT）中预防移植物抗宿主病（GVHD）的标准方案。尽管其疗效明确，但环磷酰胺相关的心脏毒性问题仍受关注，尤其对于存在基础心脏疾病的患者，而此类人群在临床试验中常代表性不足。目的：评估接受PTCy方案allo-HCT的急性髓系白血病（AML）患者中，伴或不伴基础心脏疾病者发生早期（≤100天）和晚期（>100天）心脏事件的发病率及临床结局。研究设计：西班牙造血移植与细胞治疗协作组（GETH-TC）开展的多中心回顾性研究，纳入2012至2022年间接受移植的461例AML患者（其中62例伴心脏疾病）。心脏疾病定义为有明确心脏病史或左心室射血分数<45%。采用竞争风险模型分析累积发病率、总生存期（OS）及非复发死亡率（NRM），并通过倾向评分匹配（PSM）和逆概率加权（IPW）进行校正。结果：13.2%的患者发生心脏事件，伴与不伴心脏疾病患者的早期心脏事件发生率分别为11% vs. 7%（p=0.93），晚期心脏事件发生率为8% vs. 5.3%（p=0.85）。早期事件以心律失常或心力衰竭为主。校正分析证实两组间心脏事件发生率、OS及NRM均无显著差异。两年OS分别为69% vs. 70%（p=0.50），NRM为18% vs. 17%（p=0.20）。早期心脏事件与两组患者死亡率升高均相关。结论：在结合全面心血管评估及心脏肿瘤学随访的前提下，PTCy方案可用于存在基础心脏疾病的AML患者，这为在具备适当心脏支持条件的更广泛患者群体中安全应用该方案提供了依据。
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