Department of Neurology, San Raffaele Scientific Institute, Via Olgettina, 48, 20132, Milan, Italy.
Laboratory of Human Genetics of Neurological Disorders, CNS Inflammatory Unit and INSPE, San Raffaele Scientific Institute, Milan, Italy.
J Neurol. 2018 Apr;265(4):896-905. doi: 10.1007/s00415-018-8791-1. Epub 2018 Feb 12.
Treatment choice in multiple sclerosis (MS) is crucial for optimizing risk-benefit profile.
To assess fingolimod (FTY) effectiveness and identify baseline features associated to disease activity in a large Italian cohort of Relapsing-Remitting (RR) MS patients.
Three-hundred sixty-seven RRMS patients starting FTY treatment at San Raffaele Hospital (Milan-Italy) underwent clinical and MRI evaluations for 2 years. Treatment response was assessed considering the proportion of patients with no evidence of disease activity (NEDA) and recording the time to first relapse. Primary analyses were performed stratifying for Natalizumab (NTZ) treatment in the year before (NO_NTZ vs NTZ group), to account for post-NTZ reactivation.
Almost half of patients were NEDA after 2 years, 53.4% in the NO_NTZ group and 36.2% in the NTZ group. Despite an opposite trend during the first 6-12 months, at 2-year follow-up the two groups were comparable for relapses and number of new/enlarging T2 and Gd-enhancing lesions. Baseline parameters of higher disease activity (ARR, Gd enhancing lesions and age at onset) were associated with increased likelihood of failing NEDA criteria or with shorter time to relapse (p < 0.05).
Our data strengthen FTY effectiveness in everyday clinical practice, even in patients switching from NTZ treatment. Baseline parameters of inflammatory activity are the most important prognostic factors for mid-term disease reactivation also during second-line treatment with FTY, providing hints on how to select therapies towards a more personalized management.
多发性硬化症（MS）的治疗选择对于优化风险效益比至关重要。
评估芬戈莫德（FTY）在意大利大型复发缓解型多发性硬化症（RRMS）患者队列中的疗效，并确定与疾病活动相关的基线特征。
367 名在米兰圣拉斐尔医院开始接受 FTY 治疗的 RRMS 患者在 2 年内接受了临床和 MRI 评估。通过评估无疾病活动证据（NEDA）的患者比例和记录首次复发时间来评估治疗反应。主要分析按纳塔昔单抗（NTZ）治疗在前一年（NO_NTZ 组与 NTZ 组）进行分层，以考虑 NTZ 治疗后的再激活。
近一半的患者在 2 年后达到 NEDA，NO_NTZ 组为 53.4%，NTZ 组为 36.2%。尽管在前 6-12 个月的趋势相反，但在 2 年随访时，两组在复发和新/扩大 T2 和 Gd 增强病变的数量上具有可比性。更高疾病活动度的基线参数（ARR、Gd 增强病变和发病年龄）与更有可能无法达到 NEDA 标准或复发时间更短相关（p<0.05）。
我们的数据加强了 FTY 在日常临床实践中的有效性，即使在从 NTZ 治疗转换的患者中也是如此。炎症活动的基线参数是中期疾病再激活的最重要预后因素，即使在 FTY 的二线治疗中也是如此，为如何选择治疗方法以实现更个性化的管理提供了线索。
