Abstract
Background: To investigate the remedial efficacy and safety of intravenous cyclophosphamide (CP) in the acute phase in patients with neuromyelitis optica spectrum disorder-related optic neuritis (NMOSD-ON) who are refractory to intravenous methylprednisolone (MP) treatment.Design: This study was a single-center, retrospective, observational case-control cohort study.Methods: Thirty-six patients who had acute NMOSD-ON attacks and were refractory to MP treatment were included. Patents were divided into two groups: the remedial CP group, and the MP group. The best-corrected visual acuity (BCVA), mean deviation (MD) of the visual field (VF), visual evoked potential amplitude (VEP-A), visual evoked potential latency (VEP-T), and average thickness of the retinal nerve fiber layer (RNFL) at onset, 1 month (m), 3 m, and 6 m after the attack were analyzed. Routine blood test results, liver and kidney function, routine urinalysis results and general condition were analyzed for safety issues at each follow-up. Fisher's exact test, the Mann-Whitney U test, the Kruskal-Wallis test and the Wilcoxon rank-sum test were used for statistical analysis.Results: The remedial CP group showed significant improvement over 6 m with regard to BCVA and MD (P < 0.05),whereas MP group only showed significant improvement in MD (P < 0.05). Regarding remedial CP intervention time window, the CP ≤ 30 days group showed significant improvement over 6 m with regard to BCVA (P = 0.002), MD (P = 0.003), and VEP-A (P = 0.036), while those CP > 30 days group did not. Both two subgroups showed significantly RNFL thickness reduction, however, BCVA, MD, VEP-A, VEP-T, and RNFL thickness showed no significant differences between the two subgroups at any follow-up point (P > 0.05).Conclusion: CP within 30 days of attack onset is safe and might have a beneficial degree of therapeutic efficacy for acute-phase treatment of NMOSD-ON that is refractory to MP treatment alone.
Highlights
Neuromyelitis optica spectrum disorders (NMOSD), which include the neuromyelitis optica (NMO), previously known as Devic’s syndrome, are defined as a group of inflammatory disorders of the central nervous system(CNS) characterized by episodes of immune-mediated demyelination and axonal damage preferentially affecting the optic nerves and spinal cord (1); these disorders are mainly associated with aquaporin-4 [AQP4– immunoglobulin G (IgG)] seropositivity
In 91 NMOSD-ON patients admitted at Zhongshan Ophthalmic Center, Sun Yat-sen University, between June 2013 and March 2020 following the clinical onset of an acute attack, the following clinical information was retrospectively reviewed: serum AQP4 antibody status, ophthalmology and general medical history, comprehensive ophthalmic examination data, immunological examination and craniocerebral magnetic resonance imaging (MRI) examination, treatment regimen, and effects of treatment at each follow-up
MP nonresponders were defined as (1) having visual acuity improvement < 2 lines of the Snellen chart with a baseline visual acuity ≥ 0.1 (9); (2) having improvement < 0.1 with a baseline visual acuity of ∼0.02; (3) if the baseline visual acuity was worse than CF, such as no light perception (NLP), light perception (LP) or hand motion (HM), improvement from NLP to LP, from LP to HM, or from HM to CF was considered a response to MP treatment
Summary
Neuromyelitis optica spectrum disorders (NMOSD), which include the neuromyelitis optica (NMO), previously known as Devic’s syndrome, are defined as a group of inflammatory disorders of the central nervous system(CNS) characterized by episodes of immune-mediated demyelination and axonal damage preferentially affecting the optic nerves and spinal cord (1); these disorders are mainly associated with aquaporin-4 [AQP4– immunoglobulin G (IgG)] seropositivity.The visual outcome of NMOSD-ON is often devastating, with one or two acute attacks potentially leading to irreversible blindness. The available clinical choices include intravenous methylprednisolone (MP), plasma exchange (PE) or immunoadsorption (IA), cyclophosphamide (CP), intravenous immunoglobulin (IVIg), rituximab and eculizumab (2). Among these treatments, intravenous CP has the advantages of speed, low cost, and obtained materials. Intravenous CP has the advantages of speed, low cost, and obtained materials It has been widely used for many years to treat a variety of autoimmune diseases, such as neuropsychiatric systemic lupus erythematosus (NPSLE) (3) and Sjögren’s syndrome (SS) (4). To investigate the remedial efficacy and safety of intravenous cyclophosphamide (CP) in the acute phase in patients with neuromyelitis optica spectrum disorder-related optic neuritis (NMOSD-ON) who are refractory to intravenous methylprednisolone (MP) treatment
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