METHODS: A phase II clinical trial to assess the efficacy and safety of administering four sequential doses of ex-vivo expanded bone marrow MSCs from a third-party donor to patients with persistent severe cytopenias after allo-SCT was performed.
RESULTS: The overall response rate on day 90 was 75% among the 27 evaluable patients (comprising 12 complete responses, 8 partial responses, and 7 with no response). The median time to respond was 14.5 days. Responses were observed across different profiles, including single or multiple affected lineages, primary or secondary timing, and potential immune-mediated or post-infectious pathophysiology versus idiopathic origin. With a median follow-up for surviving patients of 85 months after MSC infusion, 53% of patients are alive. Notably, no adverse events related to MSC therapy were reported.
CONCLUSIONS: In summary, the sequential infusion of third-party MSCs emerges as a viable and safe therapeutic option, exhibiting potential benefits for patients experiencing cytopenias following allo-SCT.
方法:一项II期临床试验,旨在评估在进行allo-SCT后,对患有持续性严重血细胞减少症的患者给予来自第三方供体的4种连续剂量的离体扩增骨髓间充质干细胞的疗效和安全性。
结果:在27名可评估患者中,第90天的总体缓解率为75%(包括12个完全缓解,8部分反应,和7没有回应)。中位应答时间为14.5天。在不同的配置文件中观察到了反应,包括单个或多个受影响的谱系,主要或次要计时,和潜在的免疫介导或感染后病理生理学与特发性起源。MSC输注后存活患者的中位随访时间为85个月,53%的患者还活着。值得注意的是,未报告与MSC治疗相关的不良事件.
结论:总之,顺序输注第三方MSCs是一种可行且安全的治疗选择,在allo-SCT后出现血细胞减少症的患者表现出潜在的益处。