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Clin Exp Neuroimmunol. 2015 Nov 01;6(4):413-418. doi: 10.1111/cen3.12239. Epub 2015 Aug 24.

Bevacizumab is safe in acute relapses of neuromyelitis optica.

Clinical & experimental neuroimmunology

Maureen A Mealy, Kyong Shin, Gareth John, Michael Levy

Affiliations

  1. Department of Neurology, Johns Hopkins University, Baltimore, MD, USA.
  2. Department of Neurology, Mount Sinai Hospital, New York, NY, USA.

PMID: 26834844 PMCID: PMC4729701 DOI: 10.1111/cen3.12239

Abstract

OBJECTIVES: Neuromyelitis optica (NMO) is a relapsing autoimmune disease targeting the spinal cord and optic nerve leading to paralysis and blindness. Current treatment for acute NMO attacks is immunosuppression with high-dose corticosteroids and/or plasmapheresis. Preclinical animal studies suggest that bevacizumab might be beneficial in limiting the extent of inflammation during a NMO relapse by reducing the disruption of the blood-brain barrier.

METHODS: We carried out an open-label phase 1b safety and proof-of-concept trial in 10 participants with NMO immunoglobulin G seropositive NMO, NMO spectrum disease and those at high risk for developing NMO/NMO spectrum disease who presented with an acute attack of transverse myelitis, optic neuritis or brainstem inflammation. In addition to treating with 1 g of daily intravenous methylprednisolone, we infused 10 mg/kg of bevacizumab intravenously on day 1 of treatment. The primary outcome measure was safety and the secondary outcome measure was efficacy.

RESULTS: Of the 10 participants enrolled, five presented with acute transverse myelitis, four with acute optic neuritis and one with a brainstem lesion. Bevacizumab was safe in all 10 participants, with only one serious adverse event within the 90-day follow up that was not attributed to the medication. Three patients recovered to pre-attack neurological function or better, and no patients required escalation to plasmapheresis.

CONCLUSIONS: Bevacizumab is a safe add-on therapy to high-dose corticosteroids for NMO/NMO spectrum disease patients presenting with an acute relapse.

Keywords: aquaporin-4; bevacizumab; neuromyelitis optica; vascular endothelial growth factor

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