Unitat de Neuroimmunologia Clínica, Multiple Sclerosis Centre of Catalonia (CEM-Cat), Barcelona, Spain.
Mult Scler. 2009 Oct;15(10):1195-205. doi: 10.1177/1352458509106937. Epub 2009 Sep 29.
Inflammation and neurodegeneration may have differential impacts on disease evolution in the different forms of multiple sclerosis. However, a beneficial effect of immunomodulatory drugs should not be ruled out in primary progressive multiple sclerosis. Our aim is to investigate the safety and efficacy of interferon beta-1b in primary progressive multiple sclerosis. We conducted a double-blind, stratified, randomized, parallel group, phase II pilot study where patients with primary progressive multiple sclerosis or 'transitional' forms of multiple sclerosis received interferon beta-1b at doses of 8 MIU or placebo for 24 months. The main objective of the study was to investigate the safety and tolerability of interferon beta-1b. The primary efficacy variable was the time to neurological deterioration (Expanded Disability Status Scale) confirmed at 3 months. Seventy-three patients were included and three dropped out the study. More patients in the treatment arm had at least one related adverse event (94.4% versus 45.9%; p < 0.001); no other significant differences in safety endpoints were observed. Time to neurological deterioration was not different between trial arms (log-rank test, p = 0.3135). Statistically significant differences favoring treatment were observed for the Multiple Sclerosis Functional Composite score at several timepoints, T1 and T2 lesion volume changes at 12 and 24 months, mean number of active lesions and proportion of patients with active lesions at 24 months. We conclude that interferon beta-1b is safe and well tolerated in patients with primary progressive multiple sclerosis and transitional multiple sclerosis. Positive effects of interferon beta on secondary clinical and magnetic resonance imaging outcomes were observed, but a beneficial effect on Expanded Disability Status Scale progression was not demonstrated.
炎症和神经退行性变可能对多发性硬化的不同形式的疾病进展产生不同的影响。然而，免疫调节药物的有益作用不应排除在原发性进展性多发性硬化症中。我们的目的是研究干扰素β-1b 在原发性进展性多发性硬化症中的安全性和疗效。我们进行了一项双盲、分层、随机、平行组、二期试验研究，其中原发性进展性多发性硬化症或“过渡型”多发性硬化症患者接受 8 MIU 干扰素β-1b 或安慰剂治疗 24 个月。该研究的主要目的是研究干扰素β-1b 的安全性和耐受性。主要疗效变量是在 3 个月时确认的神经功能恶化（扩展残疾状况量表）时间。共纳入 73 例患者，3 例退出研究。治疗组中更多的患者至少有一次相关不良事件（94.4%比 45.9%；p < 0.001）；未观察到其他安全性终点的显著差异。试验组之间神经功能恶化的时间无差异（对数秩检验，p = 0.3135）。在多个时间点、12 个月和 24 个月时的 T1 和 T2 病变体积变化、活跃病变的平均数量和活跃病变患者的比例上，治疗组具有统计学意义的优势。我们得出结论，干扰素β-1b 在原发性进展性多发性硬化症和过渡性多发性硬化症患者中安全且耐受良好。观察到干扰素β对继发临床和磁共振成像结果的积极影响，但对扩展残疾状况量表进展没有显示出有益作用。
