Acute myeloid leukemia (AML) is an aging-related malignancy, with patients aged ≥60 years old facing significantly poorer prognosis. Umbilical cord blood (UCB) has emerged as a promising source with effective anti-aging roles. Here, we conducted a prospective, phase 2, single-arm trial of UCB infusion as an adjuvant consolidation therapy in elderly AML patients (ChiCTR-OPC-15006492). A total of 51 patients were enrolled (median age 66 years; range, 60–75) and received two cycles of consolidation chemotherapy combined with UCB infusion. At a median follow-up of 27.3 months (range, 9.3–100), the median overall survival (OS) was not yet reached and the median event-free survival (EFS) was 72.2 months (range, 5.4–100). The 2-year OS and EFS rates were 76.9% and 62.8%, respectively. No acute graft-versus-host disease (aGVHD) or toxicity-related death occurred in any patient. The median times to platelet and neutrophil recovery were 11.5 days (range, 6–17) and 12.2 days (range, 0–21), respectively. Single-cell RNA sequencing (scRNA-seq) identified enhanced anti-tumor and anti-aging properties of UCB, manifested through activation of immune responses and telomere synthesis/maintenance. These findings suggest that UCB infusion is an effective and safe post-remission adjuvant therapy for elderly AML patients. This study provides evidence that anti-aging therapy may serve as a new and promising dimension in combined cancer treatment.

急性髓性白血病 （AML） 是一种与衰老相关的恶性肿瘤，年龄≥ 60 岁的患者预后明显较差。脐带血 （UCB） 已成为一种具有有效抗衰老作用的有前途的来源。在这里，我们进行了一项前瞻性、2 期、单臂试验，将 UCB 输注作为老年 AML 患者的辅助巩固治疗 （ChiCTR-OPC-15006492）。共有 51 名患者入组 （中位年龄 66 岁;范围，60-75 岁），并接受了两个周期的巩固化疗联合 UCB 输注。中位随访 27.3 个月 （范围，9.3-100），中位总生存期 （OS） 尚未达到，中位无事件生存期 （EFS） 为 72.2 个月 （范围，5.4-100）。2 年 OS 和 EFS 率分别为 76.9% 和 62.8%。任何患者均未发生急性移植物抗宿主病 （aGVHD） 或毒性相关死亡。血小板和中性粒细胞恢复的中位时间分别为 11.5 天 （范围，6-17） 和 12.2 天 （范围，0-21）。单细胞 RNA 测序 （scRNA-seq） 确定了 UCB 增强的抗肿瘤和抗衰老特性，表现为激活免疫反应和端粒合成/维持。这些发现表明，UCB 输注是老年 AML 患者一种有效且安全的缓解后辅助治疗。这项研究提供了证据表明，抗衰老疗法可能成为联合癌症治疗的一个新的和有前途的维度。