TY - JOUR T1 -活动性继发性进行性多发性硬化患者的造血干细胞移植JF -神经学JO -神经学SP - e1109 LP - e1122 DO - 10.1212/WNL.0000000000206750半岛投注体育官网六世- 100 - 11盟Giacomo Boffa AU -塞西格诺里盟卢卡Massacesi AU -爱丽丝Mariottini AU -埃尔韦拉Sbragia AU -萨尔瓦多Cottone盟-玛丽亚Pia阿马托盟克劳迪奥Gasperini AU -卢西亚Moiola盟斯特凡诺Meletti AU -安娜玛丽亚Repice盟Vincenzo布雷西亚猜拳AU -朱塞佩•归咎于盟弗朗西斯科·帕蒂AU -马西莫菲利皮主持盟乔凡娜De Luca盟Giacomo Patrizia苍井空逻辑单元非盟-毛罗·Zaffaroni盟盟-安东内拉·康特·拉斯泰利盟Riccardo Nistri AU - Umberto Aguglia AU -弗朗哥Granella AU - Simonetta Galgani AU -路易莎玛丽亚Caniatti盟亚历山德拉Lugaresi AU -西尔维亚·罗马诺盟Pietro Iaffaldano AU -爱Cocco AU - Riccardo Saccardi盟Emanuele Angelucci AU -玛丽亚Trojano盟Giovanni Luigi Mancardi AU -玛丽亚Pia索尔盟马蒂尔德Inglese盟代表意大利BMT-MS研究集团和意大利女士登记Y1 - 2023/03/14 UR - //www.ebmtp.com/content/100/11/e1109.abstract N2 -不受控制的证据显示背景和目标半岛投注体育官网自体造血干细胞移植(AHSCT)对活动性继发性进展性多发性硬化症(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。采用时间与治疗组相互作用的线性混合模型来评估EDSS的纵向时间趋势。 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 = 0.31–0.81; 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 ER -