文摘
摘要目的:获得真实的比较是很重要的数据有效性口腔疾病修饰治疗用于高度活跃的人(儿童)复发多发性硬化症(HA-RMS)。
背景:主要目标是比较复发率之间HA-RMS病人cladribine平板电脑(CladT)或fingolimod单一疗法治疗。二级目标报道这里包括额外的复发的结果比较,治疗开关,停止。
设计/方法:一个多中心回顾性图表回顾研究是在英国和德国进行评估CladT合格HA-RMS病人与fingolimod的前12个月的治疗。non-inferiority保证金1.2设置调整后折合成年率的复发率(ARR) CladT fingolimod比。治疗逆概率权重应用于创建平衡治疗组。Physician-confirmed复发,扩大残疾状态量表(eds)得分,和磁共振成像(MRI)的数据也被检查。
结果:1095名患者包括:610年启动CladT和485启动fingolimod。在治疗开始后的12个月,1例(0.2%)病人发起CladT又转战另一个DMT,而10 (2.1%)fingolimod又转战另一个DMT和7例(1.5%)患者中断治疗。加勒比海盗是0.10(95%可信区间[CI]: 0.07 - -0.14) CladT为0.14(95%置信区间:0.10—-0.20)fingolimod。调整ARR比率为0.68(95%置信区间:0.42—-1.11)数值有利于CladT和95%置信区间上限non-inferiority保证金。第一次复发时间和比例的患者无复发组之间是相似的。
结论:前12个月的治疗,CladT证明可比复发活动有效性fingolimod HA-RMS患者。治疗开关/中止fingolimod组更常见。未来的研究应该调查和治疗效果持久性第一12个月的治疗。
披露:Brownlee博士已经收到个人薪酬在500 - 4999美元的范围为罗氏公司担任顾问。Brownlee博士已经收到个人薪酬在500 - 4999美元的范围作为顾问生原体。Brownlee博士已经收到个人薪酬在500 - 4999美元的范围为Mylan担任顾问。Brownlee博士已经收到个人薪酬在500 - 4999美元的范围为默克公司担任顾问。Brownlee博士已经收到个人薪酬在500 - 4999美元的范围为Celgene公司担任顾问。Brownlee博士已经收到个人薪酬在500 - 4999美元的范围为赛诺菲担任顾问。Brownlee博士已经收到个人薪酬在500 - 4999美元的范围为诺华公司担任顾问。Brownlee博士已经收到个人薪酬在500 - 4999美元的范围作为一个编辑,副主编,圣人或编辑顾问委员会成员。Haghikia博士已经收到个人薪酬在500 - 4999美元的范围与生原体作为一个演讲者。Haghikia博士已经收到个人薪酬在500 - 4999美元的范围与赛诺菲安万特担任议长。 Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving as a speaker with Novartis. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving as a speaker with Merck Serono. Dr. Haghikia has received personal compensation in the range of $500-$4,999 for serving as a speaker with Celgene. Brooke Hayward, SM, MBA has received personal compensation for serving as an employee of EMD Serono, Inc., Rockland, MA USA, an affiliate of Merck KGaA, Darmstadt, Germany. Ms. Waser has received personal compensation for serving as an employee of ICON plc.. Mrs. Kayaniyil has received personal compensation for serving as an employee of ICON plc.. Mrs. Kayaniyil has received personal compensation for serving as an employee of AstraZeneca. Mr. Khan has nothing to disclose. Mrs. Duncan has received personal compensation for serving as an employee of ICON. Ms. Millar has received personal compensation for serving as an employee of ICON plc. Gerard Harty has received personal compensation for serving as an employee of Merck KGaA Darmstadt.
