A meta‐analysis comparing first‐line immunosuppressants in neuromyelitis optica

Abstract

Objective As phase III trials have shown interest in innovative but expensive drugs in the treatment of neuromyelitis optica spectrum disorder (NMOSD), data are needed to clarify strategies in the treatment of neuromyelitis optica (NMO). This meta‐analysis compares the efficacy of first‐line strategies using rituximab (RTX), mycophenolate mofetil (MMF), or azathioprine (AZA), which are still widely used. Methods Studies identified by the systematic review of Huang et al. (2019) were selected if they considered at least two first‐line immunosuppressants among RTX, MMF, and AZA. We updated this review. The Medline, Cochrane Central Register of Controlled Trials, Embase, and ClinicalTrials databases were queried between November 2018 and April 2020. To be included, the hazard ratio (HR) [95% CI] for the time to first relapse after first‐line immunosuppression had to be available, calculable, or provided by the authors. Results We gathered data from 919 NMO patients (232 RTX‐, 294 MMF‐, and 393 AZA‐treated patients). The risk of first relapse after first‐line immunosuppression was 1.55 [1.04, 2.31] (p = 0.03) for MMF compared with RTX, 1.42 [0.87, 2.30] (p = 0.16) for AZA compared with RTX, and 0.94 [0.58, 1.54] (p = 0.08) for MMF compared with AZA. Interpretation The findings suggest that RTX is more efficient than MMF as a first‐line therapy. Even if the results of our meta‐analysis cannot conclude that RTX has a better efficacy in delaying the first relapse than AZA, the observed effect difference between both treatments combined with the results of previous studies using as outcome the annualized relapse rate may be in favor of RTX.

Update on the diagnosis and treatment of neuromyelitis optica spectrum disorders (NMOSD) – revised recommendations of the Neuromyelitis Optica Study Group (NEMOS). Part II: Attack therapy and long-term management

“…Rituximab is a chimeric monoclonal antibody that binds to and depletes CD20-positive B-cells. Its clinical effectiveness in NMOSD has been demonstrated in various retrospective and prospective studies, including AQP4-IgG-positive and -negative (adult and pediatric) patients, with a reduction of attack rates by over 80% [ 52 , 53 , 61 , 74 , 135 , 253 ]. A recent randomized, double-blinded, placebo-controlled trial from Japan (RIN-1) confirmed the efficacy of rituximab in AQP4-IgG-positive NMOSD, although the sample size of the trial was small (Table 2 ) ([ 216 ].…”

Treatment access and satisfaction on disease-modifying therapies of neuromyelitis optica spectrum disorder patients in China: a cross-sectional survey

“…AZA was switched-off most often with effectiveness being the main reason for change of therapy. Judging from their respective TSQM scores and literature findings on efficacy and safety, 41 , 42 no definitive conclusion could be drawn yet to explain the dominance of MMF in the study patients. In fact, evidence regarding treatment sequences and switching remains inconclusive.…”

Overlap syndrome of anti-aquaporin-4 positive neuromyelitis optica spectrum disorder and systemic lupus erythematosus: A systematic review of individual patient data

“…35 A meta-analysis assessing the relapse rates showed superiority of RTX over MMF but no difference between AZA and MMF in the NMOSD group; however, no difference was seen between the three of them in the AQP4-NMOSD. 36 Given that MMF is a well-established first-line treatment for SLE with major organ manifestations, it may be better suited for the subset of patients with NMOSD who also have SLE. 37 Although improvements were seen in the majority of reviewed, the short follow-up durations make it difficult to accurately assess neurological outcomes, which require long-term follow-up.…”