@article {Boffae1109作者= {Giacomo Boffa塞西格诺里和卢卡Mariottini Massacesi和爱丽丝埃尔韦拉Sbragia萨尔瓦多Cottone玛丽亚Pia阿马托和克劳迪奥Gasperini和露西亚Moiola斯特凡诺Meletti和安娜玛丽亚Repice Vincenzo布雷西亚猜拳朱塞佩•生活和弗朗西斯科·帕蒂·马西莫菲利皮主持和乔凡娜De Luca Giacomo逻辑单元和毛罗·Zaffaroni Patrizia苍井空和安东内拉·康特·拉斯泰利Riccardo Nistri Umberto Aguglia和佛朗哥Granella Simonetta Galgani和路易莎玛丽亚Caniatti和亚历山德拉Lugaresi西尔维亚·罗马诺和Pietro Iaffaldano爱Cocco和里卡多。Saccardi Emanuele Angelucci和玛丽亚Trojano Giovanni Luigi Mancardi玛丽亚Pia索尔和马蒂尔德Inglese代表意大利BMT-MS研究集团和意大利女士注册},title ={造血干细胞移植患者积极继发型多发性硬化},体积={100}={11},页面= {e1109——e1122} = {2023}, doi = {10.1212 / WNL。出版商0000000000206750}= {Wolters Kluwer健康,公司代表美国神经病学学会},文摘={背景和目标不受控制的证据表明,自体造血干细胞移植(AHSCT)可以有效地活跃继半岛投注体育官网发型多发性硬化患者(spm)。在这项研究中,我们比较AHSCT的效果与其他抗炎疾病修饰治疗(儿童)在活跃的spm长期残疾恶化。方法收集的数据从意大利骨髓移植研究小组和意大利多发性硬化症登记。患者被认为是合格的,如果治疗后开始spm的诊断。残疾恶化是评估患者6个月的累积比例确认残疾进展(CDP)根据扩大残疾状态量表(eds)评分。关键二次端点eds时间治疗后开始和残疾的流行趋势改善随着时间的推移。时间第一CDP比例风险Cox回归模型的评估手段。线性混合模型和时间{\ texttimes}治疗组相互作用被用来评估纵向eds时间趋势。流行的改进估计使用修改后的kaplan meier估计和比较各组引导曲线下的面积。结果七十九AHSCT-treated病人和1975例患者治疗与其他儿童(β干扰素,硫唑嘌呤,glatiramer-acetate米托蒽醌,fingolimod, natalizumab,甲氨蝶呤,teriflunomide、环磷酰胺、富马酸二甲酯,和阿仑单抗)使用倾向得分匹配来减少治疗选择偏见和重叠的加权方法。时间第一CDP大大延长移植患者(危险比[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.ebmtp.com/content/100/11/e1109}, eprint = {//www.ebmtp.com/content/100/11/e1109.full.pdf}, journal = {Neurology} }