RT期刊文章SR电子T1造血干细胞移植患者积极乔继发型多发性硬化摩根富林明神经病学神经病学FD Lippincott Williams &威尔金斯SP e1109 OP e1122 10.1212 / WNL。半岛投注体育官网100签证官0000000000206750 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De Luca 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 A1亚历山德拉Lugaresi A1西尔维亚Romano A1 Pietro Iaffaldano A1爱Cocco A1 Riccardo Saccardi A1 Emanuele Angelucci A1玛丽亚Trojano A1 Giovanni Luigi Mancardi A1玛丽亚Pia索尔A1马蒂尔德Inglese A1代表意大利BMT-MS研究集团和意大利女士登记年2023 UL //www.ebmtp.com/content/100/11/e1109.abstract AB背景和目标不受控制的证据表明,自体造血干细胞移植(AHSCT)可以有效地活跃继发型多发性硬化患者(spm)。半岛投注体育官网在这项研究中,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×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。 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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