文摘
摘要目的:报告真实结果的有效性和安全性cladribine(复合)复发缓和多发性硬化(名RRMS),确定预测的响应。
背景:复合诱导免疫重建的瞬态淋巴细胞耗竭之后,用改进的免疫耐受。真实的复合性能和响应的预测数据是有限的。
设计/方法:Monocentric,观察,实际研究pwm包开始从2018年4月到2021年11月,最低6 month-follow-up。
结果:我们的人口由114 pwm, 71.9%的女性,50%的天真的人,平均年龄33.0岁,平均病程3.0年,平均2.0基线eds,意思是1和2的ARR year-baseline 1.3和1.2。随访中位数:23.8个月。4.4%和30.7%的患者复发和MRI活动前6个月。当考虑数据re-baselined向月6日12复发被报道。84% (CI 76 - 92)和76% (CI 67 - 87)的病人没有MRI活动12和24个月,分别。NEDA-3地位是在月24日达到69%的天真的人,和转换器从低和高效治疗的81%和55%,分别。11个病人转向其他的治疗方法。年轻的年龄(HR 0.94, CI: 0.89 - -0.99,p = 0.011),基线eds分数≥3.0 (HR 4.29, CI: 1.83 - -10.05,p = 0.0008)和钆增强病灶数≥4 (HR 2.24, CI: 0.95 - -5.26,p = 0.065)NEDA-3损失的危险因素。20.0%的pwm称为副作用:皮疹、发热、感染、疲劳。淋巴细胞减少是更有可能在3个月和15。没有4级淋巴球减少症病例观察。
结论:穿着时更有效的放置在治疗早期算法:天真的人或转换器低功效的药物似乎获益更多。钆增强病变基线年龄、残疾和NEDA-3损失的风险因素。复合整体耐受良好。真实的大样本数据和时间后续需要确认我们的发现。
披露:奇亚拉Zanetta没有披露。玛丽亚Assunta罗卡已收到个人薪酬在500 - 4999美元的范围在拜耳的演讲人,生原体,百时美施贵宝公司,Celgene公司,Genzyme,默克公司Serono,诺华,罗氏,Teva。制度的玛丽亚Assunta罗卡收到意大利卫生部的研究支持,加拿大社会和女士基金会Italiana Sclerosi Multipla。亚历山德罗代表我没有披露。Preziosa先生已经收到个人薪酬在500 - 4999美元的范围在百时美施贵宝公司的演讲人。Preziosa先生已经收到个人薪酬在500 - 4999美元的范围在赛诺菲Genzyme的演讲人。Preziosa先生已经收到个人薪酬在500 - 4999美元的范围在诺华的演讲人。Preziosa先生已经收到个人薪酬在500 - 4999美元的范围在罗氏的演讲人。Preziosa先生已经收到个人薪酬在500 - 4999美元的范围在默克的演讲人。Martinelli博士已经收到个人薪酬在500 - 4999美元的范围作为顾问诺生原体,赛诺菲安万特Genzyme TEVA和默克。 Dr. Martinelli has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck . Laura Ferrè, 3233 has received intellectual property interests from a discovery or technology relating to health care. Dr. Moiola has nothing to disclose. Dr. Filippi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Bayer, Biogen Idec, Merck-Serono, Novartis, Roche, Sanofi Genzyme, Takeda, and Teva Pharmaceutical Industries. Dr. Filippi has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Bayer, Biogen Idec, Merck-Serono, Novartis, Roche, Sanofi Genzyme, Takeda, and Teva Pharmaceutical Industries. Dr. Filippi has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Springer Nature. The institution of Dr. Filippi has received research support from Biogen Idec, Merck-Serono, Novartis, Roche, Teva Pharmaceutical Industries, Italian Ministry of Health, Fondazione Italiana Sclerosi Multipla, and ARiSLA (Fondazione Italiana di Ricerca per la SLA).
