体外扩增自体外周血单个核细胞在肌萎缩侧索硬化症治疗中的应用-现代医学

体外扩增自体外周血单个核细胞在肌萎缩侧索硬化症治疗中的应用

单位：1. 南京医科大学第一附属医院神经内科, 江苏南京 210029;
2. 南京市第二医院神经内科, 江苏南京 210003

分类号：R744.8

出版年·卷·期（页码）：2022·50·第九期（1104-1108）

摘要：

目的：探索体外扩增自体外周血单核细胞（PBMC）治疗肌萎缩侧索硬化症（ALS）的临床效果和安全性。方法：选取2019年9月至2020年8月南京市第二医院神经内科收治的28例ALS患者，取外周血分离PBMC后在体外用植物血凝素-L 1.25 μg·ml^-1、白介素-2 5×10³IU·ml^-1刺激3 d后培养扩增42 d。5×10⁷PBMC细胞按输血规范静脉输入患者体内为1轮治疗，继续取上述PBMC培养扩增，如此连续6轮治疗为一疗程。应用ALS综合整体功能障碍量表（ROADS）、肌萎缩侧索硬化功能量表（ALSFRS-R）评定疗效。结果：治疗前和6次治疗后ROADS分值为31（29，40）分和36（32，42）分，治疗后分值显著增高（P<0.05）。28例患者中16例明显有效（有效率达57.1%）。患者自觉改善症状包括肌张力、肌力改善，言语不清、吞咽困难改善，呼吸较前顺畅，改善的持续时间为2周及以上。治疗3个月后ALSFRS-R分值下降，但与治疗前相比差异无统计学意义（P>0.05），治疗中未发现严重的不良反应。结论：体外扩增自体PBMC短期治疗ALS安全有效。做好呼吸支持，同时密切观察患者的生命体征及不良反应，是保障ALS患者自体PBMC回输治疗成功的关键。

Objective: To explore the efficacy and safety of treatment with expanded ex vivo autologous peripheral blood mononuclear cells(PBMC) in patients with amyotrophic lateral sclerosis(ALS). Methods: A total of 28 ALS patients were selected and admitted to the Department of Neurology, Nanjing Second Hospital from September 2019 to August 2020. PBMC was isolated from peripheral blood of the subjects and then cultured and amplified for 42 days after stimulated with phytohemagglutinin-L 1.25 μg·ml^-1 and IL-2 5×10³·IU·ml^-1 in vitro for 3 days. According to the standard of blood transfusion, 5×10⁷ PBMC cells were infused intravenously into patients for one round of treatment, and the above-mentioned PBMC culture and amplification were taken for 6 consecutive rounds of treatment as a course of treatment. The trial had total six cycles. The Rasch-Built Overall ALS Disability Scale(ROADS) and the Revised ALS Functional Rating Scale(ALSFRS-R) were used to determine outcomes. Results: There were also significant improvements when we compared the score of ROADS after six cycles treatment with that of before the treatment(baseline)(P<0.05). However, there was no difference in the score of ALSFRS-R after 3 months treatment than that of baseline(P>0.05) although the score decreased after the treatment. In 28 patients, 16 patients felt improvement(57.1%). The improved symptoms included muscle tone, muscle strength, speech loss, swallowing difficulties, smooth breathing, etc. No serious adverse reactions were found in the treatment. Conclusion: The autologous PBMC cell therapy is safe and effective for ALS patients. Good respiratory support,closely monitored for vital signs and adverse reactions are the key to ensure the success of autotransfusion of PBMC in patients with ALS.

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