2019年9月5日，美国加州大学洛杉矶分校Bruce A C Cree课题组在《柳叶刀》发表论文，宣布他们的最新研究发现Inebilizumab可用于治疗视神经脊髓炎谱系障碍（NMOSD）。

这项多中心、双盲、随机、安慰剂对照、临床2/3期的研究在25个国家的99个医疗机构进行。2015年1月6日至2018年9月24日，研究组共招募了230名参与者，年龄均超过18岁，被诊断为NMOSD，扩展的残疾状态量表得分不高于8.0，一年内至少发作一次且需要抢救，或两年内至少发作两次且需要抢救。参与者按照3：1随机分组，其中174名接受Inebilizumab治疗，56名接受安慰剂治疗。

Inebilizumab组中有21名（12%）患者NMOSD发作，对照组有22名（39%），风险比为0.272。Inebilizumab组中有125名（72%）患者发生不良反应，安慰剂组中有41名（73%）。Inebilizumab组中有8名（5%）发生严重不良事件，安慰剂组中有5名（9%）。

综上，与安慰剂相比，Inebilizumab显著降低了NMOSD发作的风险，具有潜在应用前景。

据悉，NMOSD尚未有标准的治疗方法，这是一种罕见的，复发性，自身免疫性的中枢神经系统炎症性疾病，可导致失明和瘫痪。

附：英文原文

Title: Inebilizumab for the treatment of neuromyelitis optica spectrum disorder (N-MOmentum): a double-blind, randomised placebo-controlled phase 2/3 trial

Author: Bruce A C Cree, Jeffrey L Bennett, Ho Jin Kim, Brian G Weinshenker, Sean J Pittock, Dean M Wingerchuk, Kazuo Fujihara, Friedemann Paul, Gary R Cutter, Romain Marignier, Ari J Green, Orhan Aktas, Hans-Peter Hartung, Fred D Lublin, Jorn Drappa, Gerard Barron, Soraya Madani, John N Ratchford, Dewei She, Daniel Cimbora, Eliezer Katz

Issue&Volume: 5 September 2019

Abstract: 

Background

No approved therapies exist for neuromyelitis optica spectrum disorder (NMOSD), a rare, relapsing, autoimmune, inflammatory disease of the CNS that causes blindness and paralysis. We aimed to assess the efficacy and safety of inebilizumab, an anti-CD19, B cell-depleting antibody, in reducing the risk of attacks and disability in NMOSD.

Methods

We did a multicentre, double-blind, randomised placebo-controlled phase 2/3 study at 99 outpatient specialty clinics or hospitals in 25 countries. Eligible participants were adults (≥18 years old) with a diagnosis of NMOSD, an Expanded Disability Status Scale score of 8·0 or less, and a history of at least one attack requiring rescue therapy in the year before screening or at least two attacks requiring rescue therapy in the 2 years before screening. Participants were randomly allocated (3:1) to 300 mg intravenous inebilizumab or placebo with a central interactive voice response system or interactive web response system and permuted block randomisation. Inebilizumab or placebo was administered on days 1 and 15. Participants, investigators, and all clinical staff were masked to the treatments, and inebilizumab and placebo were indistinguishable in appearance. The primary endpoint was time to onset of an NMOSD attack, as determined by the adjudication committee. Efficacy endpoints were assessed in all randomly allocated patients who received at least one dose of study intervention, and safety endpoints were assessed in the as-treated population. The study is registered with ClinicalTrials.gov, number NCT02200770.

Findings

Between Jan 6, 2015, and Sept 24, 2018, 230 participants were randomly assigned to treatment and dosed, with 174 participants receiving inebilizumab and 56 receiving placebo. The randomised controlled period was stopped before complete enrolment, as recommended by the independent data-monitoring committee, because of a clear demonstration of efficacy. 21 (12%) of 174 participants receiving inebilizumab had an attack versus 22 (39%) of 56 participants receiving placebo (hazard ratio 0·272 [95% CI 0·150–0·496]; p<0·0001). Adverse events occurred in 125 (72%) of 174 participants receiving inebilizumab and 41 (73%) of 56 participants receiving placebo. Serious adverse events occurred in eight (5%) of 174 participants receiving inebilizumab and five (9%) of 56 participants receiving placebo.

Interpretation

Compared with placebo, inebilizumab reduced the risk of an NMOSD attack. Inebilizumab has potential application as an evidence-based treatment for patients with NMOSD. 

DOI: 10.1016/S0140-6736(19)31817-3

Source: https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(19)31817-3/fulltext