@article {Boffae1109,作者= {Giacomo Boffa和Alessio Signori和Luca Massacesi和Alice Mariottini和Elvira Sbragia和Salvatore Cottone和Maria Pia Amato和Claudio Gasperini和Lucia Moiola和Stefano Meletti和Anna Maria Repice和Vincenzo Brescia Morra和Giuseppe Salemi和Francesco Patti和Massimo Filippi和Giovanna De Luca和Giacomo Lus和Mauro Zaffaroni和Patrizia Sola和Antonella Conte和Riccardo nstri和Umberto Aguglia和Franco Granella和Simonetta Galgani和LuisaMaria Caniatti, Alessandra Lugaresi, Silvia Romano, Pietro Iaffaldano, Eleonora Cocco, Riccardo Saccardi, Emanuele Angelucci, Maria Trojano, Giovanni Luigi Mancardi, Maria Pia Sormani和Matilde Inglese，代表意大利BMT-MS研究小组和意大利MS登记册}，标题={活动性继发性进行性多发性硬化患者的造血干细胞移植}，卷={100}，数={11}，页= {e1109- e1122}，年份= {2023}，doi = {10.1212/WNL。0000000000206750}，出版商= {Wolters Kluwer Health, Inc.代表美国神经病学学会}，摘要={背景和目的未经控制的证据表明，自体造血干细胞移植(AHSCT首页)对活动性继发性进行性多发性硬化症(SPMS)患者有效。在本研究中，我们比较了AHSCT与其他抗炎疾病修饰疗法(DMTs)对活跃SPMS患者长期残疾恶化的影响。方法:我们从意大利骨髓移植研究组和意大利多发性硬化症登记处收集数据。如果诊断为SPMS后开始治疗，则认为患者符合条件。根据扩展残疾状态量表(EDSS)评分，通过6个月确认残疾进展(CDP)患者的累积比例来评估残疾恶化。关键的次要终点是治疗开始后的EDSS时间趋势和随着时间的推移残疾改善的患病率。通过比例危险Cox回归模型评估首次CDP的时间。 A linear mixed model with a time {\texttimes} treatment group interaction was used to assess the longitudinal EDSS time trends. Prevalence of improvement was estimated using a modified Kaplan-Meier estimator and compared between groups by bootstrapping the area under the curve.Results Seventy-nine AHSCT-treated patients and 1975 patients treated with other DMTs (beta interferons, azathioprine, glatiramer-acetate, mitoxantrone, fingolimod, natalizumab, methotrexate, teriflunomide, cyclophosphamide, dimethyl fumarate, and alemtuzumab) were matched to reduce treatment selection bias using propensity score and overlap weighting approaches. Time to first CDP was significantly longer in transplanted patients (hazard ratio [HR] = 0.50; 95\% CI = 0.31{\textendash}0.81; p = 0.005), with 61.7\% of transplanted patients free from CPD at 5 years. Accordingly, EDSS time trend over 10 years was higher in patients treated with other DMTs than in AHSCT-treated patients (+0.157 EDSS points per year compared with -0.013 EDSS points per year; interaction p \< 0.001). Patients who underwent AHSCT were more likely to experience a sustained disability improvement: 34.7\% of patients maintained an improvement (a lower EDSS than baseline) 3 years after transplant vs 4.6\% of patients treated by other DMTs (p \< 0.001).Discussion The use of AHSCT in people with active SPMS is associated with a slowing of disability progression and a higher likelihood of disability improvement compared with standard immunotherapy.Classification of Evidence This study provides Class III evidence that autologous hematopoietic stem cell transplants prolonged the time to CDP compared with other DMTs.AHSCT=autologous hematopoietic stem cell transplantation; ARR=annualized relapse rate; ATG=antithymocyte globulin; AUC=area under the curve; CDP=confirmed disability progression; DMT=disease-modifying therapy; EDSS=Expanded Disability Status Scale; HR=hazard ratio; IQR=interquartile range; MSMs=marginal structural models; OW=overlap weighting; PS=propensity score; SMDs=standardized mean differences; SPMS=secondary progressive multiple sclerosis}, issn = {0028-3878}, URL = {//www.ez-admanager.com/content/100/11/e1109}, eprint = {//www.ez-admanager.com/content/100/11/e1109.full.pdf}, journal = {Neurology} }