Departments of Neurology and Biomedicine, University Hospital Basel, Switzerland.
Mult Scler. 2014 Feb;20(2):243-52. doi: 10.1177/1352458513507817. Epub 2013 Oct 22.
Oral BG-12 (dimethyl fumarate), approved for the treatment of the relapsing forms of MS, has demonstrated clinical efficacy with an acceptable safety profile in the Phase III "Determination of the Efficacy and Safety of Oral Fumarate in Relapsing-Remitting Multiple Sclerosis (RRMS)" (DEFINE) and "Comparator and an Oral Fumarate in RRMS" (CONFIRM) studies.
To evaluate the health-related quality of life (HRQoL) impairment that is associated with RRMS and to assess the effects of BG-12 on HRQoL in the DEFINE study.
Patients with RRMS were randomized to BG-12 240 mg twice (BID) or three times (TID) daily, or placebo, for 2 years. HRQoL was assessed by the Short Form-36 (SF-36), global assessment of well-being visual analog scale and the EuroQol-5D.
In the 1237 patients from DEFINE, HRQoL impairment was greatest in patients who had higher disability scores and in those who had experienced relapse. Change in SF-36 physical component summary scores during 2 years' treatment significantly favored BG-12 over placebo (both doses: p < 0.001). We saw similar benefits in other measures of functioning and general well-being as early as Week 24. These benefits were maintained during the study.
Our results add to evidence for a negative impact of RRMS on HRQoL and they demonstrate the benefits of BG-12 on HRQoL measures, which coupled with significant clinical efficacy, further support its use as a new treatment for RRMS.
已批准用于治疗复发型多发性硬化症（MS）的口服 BG-12（富马酸二甲酯）在 III 期“确定口服富马酸在复发缓解型多发性硬化症（RRMS）中的疗效和安全性（DEFINE）和“比较剂和 RRMS 中的口服富马酸（CONFIRM）”研究中显示出临床疗效和可接受的安全性。
评估与 RRMS 相关的健康相关生活质量（HRQoL）受损情况，并评估 BG-12 在DEFINE 研究中对 HRQoL 的影响。
RRMS 患者随机接受 BG-12 240mg 每日两次（BID）或每日三次（TID），或安慰剂，治疗 2 年。通过短表 36（SF-36）、整体幸福感视觉模拟量表和欧洲五维健康量表评估 HRQoL。
在DEFINE 中的 1237 名患者中，HRQoL 受损在残疾评分较高的患者和经历过复发的患者中最为严重。在 2 年治疗期间，SF-36 生理成分综合评分的变化明显优于安慰剂（两种剂量：p < 0.001）。早在第 24 周，我们就看到了在其他功能和一般健康测量中类似的益处。这些益处在研究期间得到了维持。
我们的结果增加了 RRMS 对 HRQoL 有负面影响的证据，并证明了 BG-12 对 HRQoL 测量的益处，这些益处与显著的临床疗效相结合，进一步支持将其作为 RRMS 的新治疗方法。
