Efficacy of the anti-IL-6 receptor antibody tocilizumab in neuromyelitis optica: a pilot study

Neurology. 2014 Apr 15;82(15):1302-6. doi: 10.1212/WNL.0000000000000317. Epub 2014 Mar 14.


Objective: To evaluate the safety and efficacy of a humanized anti-interleukin-6 receptor antibody, tocilizumab (TCZ), in patients with neuromyelitis optica (NMO).

Methods: Seven patients with anti-aquaporin-4 antibody (AQP4-Ab)-positive NMO or NMO spectrum disorders were recruited on the basis of their limited responsiveness to their current treatment. They were given a monthly injection of TCZ (8 mg/kg) with their current therapy for a year. We evaluated the annualized relapse rate, the Expanded Disability Status Scale score, and numerical rating scales for neurogenic pain and fatigue. Serum levels of anti-AQP4-Ab were measured with AQP4-transfected cells.

Results: Six females and one male with NMO were enrolled. After a year of TCZ treatment, the annualized relapse rate decreased from 2.9 ± 1.1 to 0.4 ± 0.8 (p < 0.005). The Expanded Disability Status Scale score, neuropathic pain, and general fatigue also declined significantly. The ameliorating effects on intractable pain exceeded expectations.

Conclusion: Interleukin-6 receptor blockade is a promising therapeutic option for NMO.

Classification of evidence: This study provides Class IV evidence that in patients with NMO, TCZ reduces relapse rate, neuropathic pain, and fatigue.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Antibodies, Monoclonal, Humanized / adverse effects
  • Antibodies, Monoclonal, Humanized / therapeutic use*
  • Fatigue / drug therapy
  • Female
  • Humans
  • Male
  • Middle Aged
  • Neuralgia / drug therapy
  • Neuromyelitis Optica / drug therapy*
  • Pilot Projects
  • Receptors, Interleukin-6 / antagonists & inhibitors*
  • Secondary Prevention
  • Treatment Outcome


  • Antibodies, Monoclonal, Humanized
  • Receptors, Interleukin-6
  • tocilizumab