2024 年第 6 期 第 0 卷

普乐沙福对系统性轻链型淀粉样变性行自体外周血干细胞移植效果的影响

The effects of plerixafor on systemic light chain amyloidosis by autologous peripheral blood for stem cell transplantation

作者：艾燕董艳杨洪霞付微微

英文作者：Ai Yan Dong Yan Yang Hongxia Fu Weiwei

单位：东部战区总医院国家肾脏疾病临床医学研究中心细胞治疗中心，南京210002

英文单位：Cell Therapy Center General Hospital of Eastern Theater Command National Clinical Research Center for Kidney Disease Nanjing 210002 China

关键词：系统性轻链型淀粉样变性；普乐沙福；自体外周血干细胞移植；干细胞动员

英文关键词：Systemiclightchainamyloidosis;Plerixafor;Autologousperipheralbloodforstemcelltransplantation;Stemcellmobilization

• 摘要：

• 目的 探讨普乐沙福联合粒细胞集落刺激因子(G-CSF)对系统性轻链型淀粉样变性（AL型淀粉样变性）行自体外周血干细胞移植（ASCT）效果的影响。方法 回顾性选取2021年7月至2023年7月于东部战区总医院国家肾脏疾病临床医学研究中心诊断为AL型淀粉样变性后行ASCT的患者共46例，采用单独G-CSF动员方案的23例患者纳入对照组，采用普乐沙福联合G-CSF动员方案的23例患者纳入观察组。比较2组患者干细胞动员不良反应发生率、干细胞采集及回输情况、移植并发症及造血重建等临床资料。结果 观察组干细胞动员相关不良反应腹泻发生率高于对照组（P＜0.01）。观察组采集前CD+34细胞计数及采集物CD+34细胞数均高于对照组［（135±97）个/μl比（42±21）个/μl、（6.9±3.5）×106/kg比（3.2±1.7）×106/kg］，采集费用低于对照组（均P＜0.01）。观察组干细胞回输量、回输费用均低于对照组（均P＜0.01）。2组移植粒缺期各项并发症发生率比较差异均无统计学意义（均P＞0.05）。观察组粒缺时长、粒系植入时间、血小板植入时间及住院天数均短于对照组，住院费用低于对照组（均P＜0.05）。结论 普乐沙福联合G-CSF动员方案应用于AL型淀粉样变性行ASCT中是安全有效的，可减少采集次数的同时提高采集质量，有效促进造血重建，缩短住院天数，减少住院费用。

• Objective To investigate the effect of plerixafor combined with granulocyte colony stimulating factor (G-CSF) on systemic light chain amyloidosis (AL amyloidosis) by autologous peripheral blood for stem cell transplantation (ASCT). Methods A total of 46 patients diagnosed with AL amyloidosis and undergoing ASCT from July 2021 to July 2023 in General Hospital of Eastern Theater Command, National Clinical Research Center for Kidney Disease were enrolled retrospectively. Among them, 23 patients with G-CSF mobilization regimen alone were included in the control group, and 23 patients with plerixafor combined G-CSF mobilization regimen were included in the observation group. The incidence of adverse reactions of stem cell mobilization, stem cell collection and reinfusion, transplantation complications and hematopoietic reconstitution were compared between the two groups. Results The incidence of diarrhoea related to stem cell mobilization in the observation group was higher than that in the control group (P<0.01). The number of CD+34 cells before collection and CD+34 cells in the collection in the observation group were higher than those in the control group［（135±97）cells/μl vs （42±21）cells/μl,（6.9±3.5）×106/kg vs （3.2±1.7）×106/kg］, and the collection cost was lower than that of the control group (all P＜0.01). The amount and cost of stem cell reinfusion in the observation group were lower than those in the control group (both P<0.01). There was no significant difference in the incidence of complications in the neutropenia period between the two groups (all P>0.05). The duration of granule deficiency, vertical implantation time, platelet implantation time and length of hospital stay in the observation group were shorter than those in the control group, and the hospitalization cost was lower than that in the control group (all P<0.05). Conclusions  Plerixafor combined with G-CSF mobilization regimen is safe and effective in ASCT for AL amyloidosis. It can reduce the frequency of collection, improve the quality of collection, effectively promote hematopoietic reconstitution, shorten the length of hospital stay, and reduce the cost of hospitalization.