Predictors of azathioprine and mycophenolate mofetil response in patients with neuromyelitis optica spectrum disorder: A cohort study.

BACKGROUND: Relapse rates of 47 % have been reported in patients with neuromyelitis optica (NMOSD) using Azathioprine (AZA) and mycophenolate mofetil (MMF). Prediction of non-responders could help determine which patients are most likely to benefit from newer monoclonal antibody treatments from the outset. OBJECTIVES: To identify predictors of AZA and MMF treatment response in NMOSD. METHODS: Multicenter cohort study of NMOSD patients from Brazil and the United Kingdom, treated with AZA and MMF. An unsatisfactory response was defined as one severe or two non-severe attacks in a year. Cox regression was used to identify predictive factors of unsatisfactory response to AZA and MMF. RESULTS: 103 NMOSD patients, mean age 38 years, 83% female, and 65% of Black ethnic group were included. An unsatisfactory IS response was observed in 42% of patients over 2.5 years (IQR 1.0-8.8) years. A severe preceding attack was more common in non-responders (31.1% x 76.7%, p = <0.001). In multivariable analysis, severe attack (RR 3.13; 95 % CI 1.37-7.18, p = 0.007) or higher annualized relapse rate (RR 4.84; 95 % CI 2.01-11.65, p = < 0.001) predicted an unsatisfactory response. Interestingly, Black NMOSD patients had a lower risk of poor response (RR 0.39, 95 % CI 0.17-0.85, p = 0.019). CONCLUSION: Severe attack and a higher annualized relapse rate before AZA or MMF initiation were associated with an unsatisfactory IS response. In patients with these characteristics, treatment with higher-efficacy drugs should be considered from the outset.

Clinical and prognostic aspects of patients with the Neuromyelitis Optica Spectrum Disorder (NMOSD) from a cohort in Northeast Brazil.

INTRODUCTION: Neuromyelitis optica spectrum disorders (NMOSD) is a rare inflammatory and demyelinating disease of the central nervous system (CNS) more frequent in women and Afro-descendants. No previous epidemiological or prognostic study has been conducted in the region of the state of Bahia, Brazilian Northeast. OBJECTIVE: To evaluate clinical and prognostic aspects in patients with NMOSD from a cohort in northeastern Brazil. MATERIAL AND METHODS: A single-center retrospective study was conducted with consecutive patients diagnosed with NMOSD. Clinical and epidemiological characteristics were described. The degree of disability was expressed by the Expanded Disability Status Scale (EDSS). Worsening disability were analyzed through negative binomial regression adjusted for disease duration. RESULTS: Ninety-one patients were included, 72 (79.1%) female and 67 (73.6%) afro descendants. Mean age at onset was 36 (± 14) years and 73.3% were anti-aquaporin-4 antibody positive. Isolated transverse myelitis (32.9%) and isolated optic neuritis (22.4%) were the most frequent initial clinical syndromes. After multivariate analysis, optic neuritis (RR = 0.45; 95% CI = 0.23 - 0.88; p = 0.020) and dyslipidemia (RR = 0.40; 95% CI = 0.20 - 0.83; p = 0.014) were associated with slower disease progression. Area postrema involvement (RR = 6.70; 95% CI = 3.31 - 13.54; p < 0.001) and age at onset (RR = 1.03; 95% CI = 1.01 - 1.05; p = 0.003) were associated with faster disease progression. CONCLUSIONS: In the first clinical and prognostic study in northeastern Brazil, we identified area postrema involvement, age at onset, optic neuritis at fist syndrome and dyslipidemia as the main prognostic factors associated with disease progression.