Sammut R, Fenwarth L, Pelissier A, Marceau A, Duployez N, Benachour S, Dadone B, Cluzeau T, Loschi M
Département d'Hématologie, Centre Hospitalier Universitaire de Nice, Nice, France.
Laboratoire d'Hématologie, Centre Hospitalier Universitaire de Lille, France.
J Cell Mol Med. 2025 Mar;29(6):e70520. doi: 10.1111/jcmm.70520.
Microtransplantation is a cellular therapy used in acute myeloid leukaemia and myelodysplastic syndromes as a maintenance therapy in patients ineligible for a regular allogeneic stem cell transplantation. We performed a monocentric retrospective study of acute myeloid leukaemia, myelodysplastic syndromes, and chronic myelomonocytic leukaemia patients who underwent microtransplantations at Nice University Hospital. We analysed the evolution of the disease mutational status after microtransplantation. We report 18 patients who underwent microtransplantation courses, with a total of 47 microtransplantations performed between February 2020 and June 2022. We observed long-term remissions even in high-risk patients. Founder mutations persisted throughout the follow-up, whereas it was more variable for other nonfounder mutations, with most of the nonfounder mutations variant allele frequency decreasing over time. Safety data were reassuring; no graft versus host disease was recorded, and cytokine release syndromes were manageable. Relapses or progressions were associated with the emergence or increase of a TP53 mutated clone. Microtransplantation is a promising therapy for patients ineligible for regular allogeneic stem transplantation. Further larger and randomised studies are required to establish its place as a maintenance therapy in myeloid malignancies.
微移植是一种细胞疗法,用于急性髓系白血病和骨髓增生异常综合征,作为不适于常规异基因干细胞移植患者的维持治疗。我们对在尼斯大学医院接受微移植的急性髓系白血病、骨髓增生异常综合征和慢性粒单核细胞白血病患者进行了一项单中心回顾性研究。我们分析了微移植后疾病突变状态的演变。我们报告了18例接受微移植疗程的患者,在2020年2月至2022年6月期间共进行了47次微移植。我们观察到即使是高危患者也实现了长期缓解。起始突变在整个随访过程中持续存在,而其他非起始突变则变化更大,大多数非起始突变的变异等位基因频率随时间下降。安全性数据令人放心;未记录到移植物抗宿主病,细胞因子释放综合征也可控。复发或进展与TP53突变克隆的出现或增加有关。微移植对于不适于常规异基因干细胞移植的患者是一种有前景的治疗方法。需要进一步开展更大规模的随机研究,以确立其作为髓系恶性肿瘤维持治疗方法的地位。