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

• Published in: Neurology Vol. 82; no. 15; p. 1302
• Main Authors: Araki, Manabu, Matsuoka, Takako, Miyamoto, Katsuichi, Kusunoki, Susumu, Okamoto, Tomoko, Murata, Miho, Miyake, Sachiko, Aranami, Toshimasa, Yamamura, Takashi
• Format: Journal Article
• Language: English
• Published: United States 15.04.2014
• Subjects: 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
• ISSN: 1526-632X
• DOI: 10.1212/WNL.0000000000000317

To evaluate the safety and efficacy of a humanized anti-interleukin-6 receptor antibody, tocilizumab (TCZ), in patients with neuromyelitis optica (NMO). 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. 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. Interleukin-6 receptor blockade is a promising therapeutic option for NMO. This study provides Class IV evidence that in patients with NMO, TCZ reduces relapse rate, neuropathic pain, and fatigue.