Harvard Medical School, Brigham and Women's Hospital, Partners Multiple Sclerosis Center, 60 Fenwood Road, Boston, MA, 02115, USA.
Harvard Medical School, Massachusetts General Hospital, Boston, MA, USA.
J Neurol. 2018 Nov;265(11):2688-2694. doi: 10.1007/s00415-018-9050-1. Epub 2018 Sep 10.
There are increasingly effective therapies for relapsing forms of multiple sclerosis (MS); however, the options for the progressive patient population are limited. The effect of mycophenolate mofetil (MMF), a disease-modifying agent for several autoimmune diseases, in progressive MS has not been explored effectively. We performed a prospective study to assess the safety and efficacy of MMF in progressive MS patients.
We identified 64 patients enrolled in the comprehensive longitudinal database at the Partners MS Center, who fulfilled our inclusion criteria. They were exposed to MMF for at least 1 year with recorded clinical outcomes. Efficacy was assessed by comparing the absolute relapse rate (ARR), and the mean Expanded Disability Status Scale (EDSS) and timed 25 foot walk (T25FW) test scores before and after MMF treatment.
At the start of MMF, 78% of patients (n = 50) were in the 4-7.5 EDSS range. There was a slight increase in mean EDSS from 5.49 ± 1.65 (n = 48) 1 year before MMF start to 5.85 ± 1.56 (n = 48) 1 year after (p = 0.020). The mean T25FW score increased 1 year before MMF from 12.3 ± 9.6 s (n = 38) to 15.6 ± 12.3 s (n = 38) 1 year after (p = 0.009). The ARR in the 2 years pre-MMF period was 0.30 ± 0.63, which decreased to a 0.09 ± 0.29 (p = 0.022) 2 years post MMF.
MMF did not affect disease progression but did influence relapse rate. We believe that other medication options should be considered before MMF in advanced progressive patients.
多发性硬化(MS)的复发形式有越来越多有效的治疗方法;然而,进展型患者群体的选择有限。吗替麦考酚酯(MMF)作为几种自身免疫性疾病的疾病修饰治疗药物,其在进展型 MS 中的效果尚未得到有效探索。我们进行了一项前瞻性研究,以评估 MMF 在进展型 MS 患者中的安全性和疗效。
我们从合作伙伴 MS 中心的综合纵向数据库中确定了 64 名符合我们纳入标准的患者。他们接受 MMF 治疗至少 1 年,并记录了临床结果。通过比较 MMF 治疗前后的绝对复发率(ARR)、扩展残疾状况量表(EDSS)均值和定时 25 英尺步行(T25FW)测试评分,评估疗效。
在开始使用 MMF 时,78%的患者(n=50)EDSS 处于 4-7.5 范围。在 MMF 开始前 1 年,平均 EDSS 从 5.49±1.65(n=48)略微增加到 5.85±1.56(n=48)(p=0.020)。在 MMF 开始前 1 年,T25FW 评分从 12.3±9.6s(n=38)增加到 15.6±12.3s(n=38)(p=0.009)。在 MMF 前 2 年,ARR 为 0.30±0.63,在 MMF 后 2 年降至 0.09±0.29(p=0.022)。
MMF 并未影响疾病进展,但确实影响了复发率。我们认为,在晚期进展型患者中,应在使用 MMF 之前考虑其他药物选择。