RT杂志文章SR电子T1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FD Lippincott Williams & Wilkins SP e1109 OP e1122 DO 10.1212/WNL.0000000000206750首页签证官100年11 A1 Giacomo Boffa A1塞西格诺里A1卢卡Massacesi A1爱丽丝Mariottini A1埃尔韦拉Sbragia A1萨尔瓦多Cottone A1玛丽亚Pia阿马托A1克劳迪奥Gasperini A1卢西亚Moiola A1斯特凡诺Meletti A1安娜玛丽亚Repice A1 Vincenzo布雷西亚猜拳A1朱塞佩归咎于A1弗朗西斯科·帕蒂A1马西莫菲利皮主持A1乔凡娜德卢卡A1 Giacomo逻辑单元A1 Mauro Zaffaroni A1 Patrizia苍井空A1安东内拉·康特·拉斯泰利A1 Riccardo Nistri A1 Umberto Aguglia A1佛朗哥Granella A1 Simonetta Galgani A1路易莎玛丽亚Caniatti A1Alessandra Lugaresi A1 Silvia Romano A1 Pietro Iaffaldano A1 Eleonora Cocco A1 Riccardo Saccardi A1 Emanuele Angelucci A1 Maria Trojano A1 Giovanni Luigi Mancardi A1 Maria Pia Sormani A1 Matilde Inglese A1代表意大利BMT-MS研究组和意大利MS Register YR 2023 UL //www.ez-admanager.com/content/100/11/e1109.abstract首页 AB背景和目标未经控制的证据表明，自体造血干细胞移植(AHSCT)可以有效地用于患有糖尿病的人活动性继发性进行性多发性硬化(SPMS)。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

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