TY - T1的自体造血干细胞移植在活跃的多发性硬化症JF -神经学乔-神经病学SP - e890 LP - e901做- 10.1212 / WNL。首页0000000000012449六世- 97 - 9盟(Richard s . Nicholas盟Mariottini爱丽丝-伊利亚·e·罗纳盟盟Eli Silber AU -奥马尔·马利克盟维多利亚Singh-Curry AU -本特纳AU -安东尼奥Scalfari AU -奥尔加Ciccarelli AU -玛丽亚·p·索尔AU -爱德华多Olavarria盟Varun用AU -伊恩·加布里埃尔盟Majid a伤势严重非盟-保罗Muraro盟代表伦敦小组自体造血干细胞移植对多发性硬化症Y1 - 2021/08/31 UR - //www.ez-admanager.com/content/97/9/e890.abstract N2 -多发性硬化症患者客观检查结果(pwm)治疗自体造血干细胞移植(AHSCT)在现实世界的环境。首页方法这是一个回顾性队列研究pwm AHSCT 2处理中心在伦敦,英国,2012年到2019年之间连续≥6个月的随访或死亡。主要结果生存自由的多发性硬化症(MS)复发,MRI新病灶,规模扩大残疾状况恶化(eds)得分。不良事件率也检查了。结果中包括120 pwm;有52%的进步(一级或二级)和48%女士复发缓和女士在基线,eds得分中值为6.0;90%的可评价的案例显示,MRI AHSCT前12个月的活动。平均随访AHSCT后21个月(范围6 - 85个月)。女士在2年复发存活率为93%和87%,AHSCT后4年。没有新的MRI病灶被发现在90%的参与者在2年和4年85%。eds得分无进展生存(PFS)是75%在2年和4年65%。 Epstein-Barr virus reactivation and monoclonal paraproteinemia were associated with worse PFS. There were 3 transplantation-related deaths within 100 days (2.5%), all after fluid overload and cardiac or respiratory failure.Conclusions Efficacy outcomes of AHSCT in this real-world cohort are similar to those reported in more stringently selected clinical trial populations, although the risks may be higher.Classification of Evidence This study is rated Class IV because of the uncontrolled, open-label design.AHSCT=autologous hematopoietic stem cell transplantation; BEAM=BiCNU (carmustine), etoposide, Ara-C (cytarabine), melphalan; DMT=disease-modifying treatment; EBMT=European Bone Marrow Transplantation Society; EDSS=Expanded Disability Status Scale; G-CSF=granulocyte colony-stimulating factor; HH=Hammersmith Hospital; KCH=Kings College Hospital; KM=Kaplan-Meier; MDT=multidisciplinary team; MS=multiple sclerosis; NEDA=no evidence of disease activity; PBSC=peripheral blood stem cell; PPMS=primary progressive MS; PwMS=people with MS; rATG=rabbit anti-thymocyte globulin; RCT=randomized controlled trial; RRMS=relapsing-remitting MS; SPMS=secondary progressive MS; TRM=treatment-related mortality ER -