Abstract：
OBJECTIVE: The aim was to determine the value of autologous haematopoietic stem cell transplantation (aHSCT) as a therapeutic intervention for progressive multiple sclerosis (PMS) based on a systematic review of the current literature.
METHODS: All studies from the databases PubMed and Google Scholar published in English before February 2024 which provided individual data for PMS patients were systematically reviewed. PICO was defined as population (P), primary progressive MS and secondary progressive MS patients; intervention (I), treatment with aHSCT; comparison (C), none, disease-modifying therapy treated/relapsing-remitting MS cohorts if available; outcome (O), transplant-related mortality, progression-free survival (PFS) and no evidence of disease activity.
RESULTS: A total of 15 studies met the criteria including 665 patients with PMS (74 primary progressive MS, 591 secondary progressive MS) and 801 patients with relapsing-remitting MS as controls. PFS data were available for 647 patients. PMS patients showed more severe disability at baseline than relapsing-remitting MS patients. The average transplant-related mortality for PMS in 10 studies was 1.9%, with 10 deaths in 528 patients. PFS ranged from 0% to 78% in PMS groups 5 years after treatment initiation, demonstrating a high variability. No evidence of disease activity scores at 5 years ranged from 0% to 75%.
CONCLUSIONS: Based on the available data, aHSCT does not halt progression in people with PMS. However, there appears to be evidence of improved outcome in selected patients. Due to the heterogeneity of the available data, more comprehensive clinical trials assessing the efficacy of aHSCT across different patient groups are urgently needed to reduce variability and improve patient stratification.
METHODS: All studies from the databases PubMed and Google Scholar published in English before February 2024 which provided individual data for PMS patients were systematically reviewed. PICO was defined as population (P), primary progressive MS and secondary progressive MS patients; intervention (I), treatment with aHSCT; comparison (C), none, disease-modifying therapy treated/relapsing-remitting MS cohorts if available; outcome (O), transplant-related mortality, progression-free survival (PFS) and no evidence of disease activity.
RESULTS: A total of 15 studies met the criteria including 665 patients with PMS (74 primary progressive MS, 591 secondary progressive MS) and 801 patients with relapsing-remitting MS as controls. PFS data were available for 647 patients. PMS patients showed more severe disability at baseline than relapsing-remitting MS patients. The average transplant-related mortality for PMS in 10 studies was 1.9%, with 10 deaths in 528 patients. PFS ranged from 0% to 78% in PMS groups 5 years after treatment initiation, demonstrating a high variability. No evidence of disease activity scores at 5 years ranged from 0% to 75%.
CONCLUSIONS: Based on the available data, aHSCT does not halt progression in people with PMS. However, there appears to be evidence of improved outcome in selected patients. Due to the heterogeneity of the available data, more comprehensive clinical trials assessing the efficacy of aHSCT across different patient groups are urgently needed to reduce variability and improve patient stratification.
摘要：
目的：目的是在对现有文献进行系统综述的基础上，确定自体造血干细胞移植（aHSCT）作为进行性多发性硬化（PMS）治疗干预措施的价值。
方法：系统回顾了2024年2月之前以英文发表的PubMed和GoogleScholar数据库中提供PMS患者个体数据的所有研究。PICO被定义为人口（P），原发性进展性MS和继发性进展性MS患者；干预(I)，用aHSCT治疗；比较(C)，无,疾病改善治疗/复发缓解型MS队列（如果可用）；结果（O），移植相关死亡率，无进展生存期（PFS），没有疾病活动的证据。
结果：共有15项研究符合标准，包括665名PMS患者（74名原发性进展型MS，591例继发性进行性MS）和801例复发缓解型MS患者作为对照。有647例患者的PFS数据。PMS患者在基线时表现出比复发缓解型MS患者更严重的残疾。在10项研究中，PMS的平均移植相关死亡率为1.9％，528例患者中有10例死亡。治疗开始后5年，PMS组的PFS范围为0％至78％，表现出高度的可变性。没有证据表明5年时的疾病活动性评分范围为0%至75%。
结论：根据现有数据，aHSCT不停止进步与PMS的人。然而,在选定的患者中似乎有改善预后的证据.由于现有数据的异质性，迫切需要更全面的临床试验来评估aHSCT在不同患者组中的疗效,以减少变异性并改善患者分层.
方法：系统回顾了2024年2月之前以英文发表的PubMed和GoogleScholar数据库中提供PMS患者个体数据的所有研究。PICO被定义为人口（P），原发性进展性MS和继发性进展性MS患者；干预(I)，用aHSCT治疗；比较(C)，无,疾病改善治疗/复发缓解型MS队列（如果可用）；结果（O），移植相关死亡率，无进展生存期（PFS），没有疾病活动的证据。
结果：共有15项研究符合标准，包括665名PMS患者（74名原发性进展型MS，591例继发性进行性MS）和801例复发缓解型MS患者作为对照。有647例患者的PFS数据。PMS患者在基线时表现出比复发缓解型MS患者更严重的残疾。在10项研究中，PMS的平均移植相关死亡率为1.9％，528例患者中有10例死亡。治疗开始后5年，PMS组的PFS范围为0％至78％，表现出高度的可变性。没有证据表明5年时的疾病活动性评分范围为0%至75%。
结论：根据现有数据，aHSCT不停止进步与PMS的人。然而,在选定的患者中似乎有改善预后的证据.由于现有数据的异质性，迫切需要更全面的临床试验来评估aHSCT在不同患者组中的疗效,以减少变异性并改善患者分层.
