治疗MOG-IgG-associated脱髓鞘与利妥昔单抗:跨国研究的98名患者(S13.003)

摘要目的:评估的有效性利妥昔单抗在预防复发的MOG-IgG-associated脱髓鞘。

背景:MOG-IgG-associated髓鞘脱失(MOG-D)越来越认可,诊断测试是可用的,作为一种独特的非ms与中枢神经系统脱髓鞘疾病有许多共同特征aquaporin-4-IgG-associated neuromyelitis视谱系障碍(AQP4-NMOSD)。在回顾系列中,利妥昔单抗在AQP4-NMOSD可以减少复发80%。美罗华的功效MOG-D是未知的。

设计/方法:回顾性研究的Rituximab-treated MOG-D病人(n = 112)从25中心在欧洲(16),美国(4)、阿根廷(1)、巴西(1)、印度(1),韩国(1)和日本(1)。

结果:排除病例诊断的不确定性(1),治疗不足(3)(10)和不完整的数据,分析了98例(59%女性;在疾病发病平均年龄为26.1岁)。疾病持续时间之前治疗中位数为19.5个月,在利妥昔单抗的平均时间为12.2个月。81名患者(83%)复发疾病之前,利妥昔单抗治疗。58例患者(59%)接受利妥昔单抗作为第一线治疗。

复发患者的疾病开始美罗华(n = 81)之前有复发率降低42%利妥昔单抗(95% ci = 21 - 57%, p = 0.001,泊松回归)。平均年复发率(至少6个月的随访期间患者和post-Rituximab, n = 51)降至0.9(范围0.1 - -6.0),0.0(范围0.0 - -4.0)(p = 0.013,老Wilcoxon测试)。生存分析预测,63% (95% ci = 49 - 73%)和35% (95% ci = 19 - 49%) Rituximab-treated患者分别将在一到三年不复发。中位数时间第一次复发利妥昔单抗(n = 39)是5.0个月(范围0.9 - -23.7个月)。有些复发发生尽管证实b细胞耗竭。17岁的患者只有一个事件开始前利妥昔单抗5至少有一个与利妥昔单抗治疗复发。

结论:美罗华治疗MOG-D显著降低复发率。在AQP4-NMOSD相似的研究相比,好处似乎不太明显,需要进一步研究。

披露:Whittam博士没有披露。Cobo-Calvo博士没有披露。洛佩斯博士没有披露。Pardo博士没有披露。博士多德没有披露。布兰德博士没有披露。Berek博士没有披露。伯格尔博士已经收到个人赔偿咨询、担任科学顾问委员会说,与参加会议或其他活动赞助和接收谢礼(讲座、咨询委员会磋商)从Almirall生原体,Celgene公司,Genzyme,默克公司诺华Octapharma,罗氏公司,赛诺菲基督复临论者/ TEVA。伯格尔博士已经收到了从他的机构已经收到了金融支持的研究不受限制的研究经费(生原体,诺华,罗氏公司,赛诺菲-安万特/ TEVA)在多发性硬化症和参与临床试验由Alexion,拜耳,生原体,默克公司诺华,Octapharma,。Gombolay博士没有披露。 Dr. Oliveira has nothing to disclose. Dr. Callegaro has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biogen, Merck, Roche, Genzyme. Dr. Kaneko has nothing to disclose. Dr. Misu has nothing to disclose. Dr. Brochet has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Novartis, Biogen-idec, Genzyme, Medday. Dr. Brochet has received research support from Teva, Biogen, Medday, Novartis, Roche, Actelion, Genzyme, Merck, Bayer. Dr. Audoin has nothing to disclose. Dr. Mathey has nothing to disclose. Dr. Laplaud has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Novartis, Biogen, Roche, Merck. Dr. Laplaud has received research support from Novartis, Biogen, Roche, Merck. Dr. Thouvenot has nothing to disclose. Dr. Cohen has nothing to disclose. Dr. Tourbah has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biogen, Novartis, MedDay, Merck serono, Roche, Sanofi-Genzyme, Teva. Dr. Maillart has nothing to disclose. Dr. Ciron has nothing to disclose. Dr. Deschamps has nothing to disclose. Dr. Biotti has nothing to disclose. Dr. Matiello has nothing to disclose. Dr. Palace has nothing to disclose. Dr. Lim has nothing to disclose. Dr. Fujihara has nothing to disclose. Dr. Nakashima has nothing to disclose. Dr. Bennett has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Consulting / advisory board: Genentech, MedImmune, EMD Serono, Teva Neuroscience, Genzyme. Dr. Bennett has received royalty, license fees, or contractual rights payments from Royalty payments for intellectual property on Aquaporumab. Dr. Bennett has received research support from EMD Serono. Dr. Pandit has nothing to disclose. Dr. Chitnis has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Bayer, Biogen, Novartis, Sanofi-Genzyme, Roche-Genentech. Dr. Weinshenker has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Novartis, Alexion, MedImmune, Caladrius Biosciences, Brainstorm Therapeutics. Dr. Wildemann has nothing to disclose. Dr. Sato has nothing to disclose. Dr. Kim has nothing to disclose. Dr. Kim has nothing to disclose. Dr. Reindl has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Speaker honoraria and compensation of travel costs from Chugai. Dr. Reindl has received research support from The Neurological Research Laboratory (Markus Reindl, Medical University of Innsbruck and Tirol Kliniken) receives payments for antibody assays (AQP4- and anti-neuronal antibodies) and for AQP4- and MOG-antibody validation experiments organized by Euroimmu. Dr. Levy has nothing to disclose. Dr. Jarius has nothing to disclose. Dr. Tenembaum has nothing to disclose. Dr. Paul has nothing to disclose. Dr. Pittock has nothing to disclose. Dr. Marignier has nothing to disclose. Dr. Jacob has received personal compensation for consulting, serving on a scientific advisory board, speaking, or other activities with Biogen, Chugai, Sanofi-Genzyme and Terumo-BCT. Dr. Jacob has received research support from Biogen Adec, Alexion Pharmaceuticals.