Impending myasthenic crisis (IMC) is an emergent situation requiring aggressive management to prevent patients from developing myasthenic crisis (MC) in patients with myasthenia gravis (MG). Efgartigimod has been proved to be well tolerated and efficacious in MG patients. The present study aimed to compare the efficacy of efgartigimod and intravenous immunoglobulin (IVIg) in rescuing IMC. IMC patients treated with efgartigimod or IVIg were retrospectively enrolled. The primary outcome was determined as the mean change in MG activities of daily living (MG-ADL) score from baseline to week 1 and 4 after treatment, respectively. Safety was assessed based on medical records during the hospitalization to monitor the adverse events. A total of 9 patients treated with efgartigimod and 10 patients treated with IVIg were enrolled. There were no significant differences in the clinical characteristics at baseline between the two groups (P > 0.05). Compared with the IVIg group, the efgartigimod group had a greater reduction in the MG-ADL score at week 1 (P = 0.035) and week 4 (P = 0.005). One patient in the efgartigimod group had an upper respiratory infection. These findings suggest that efgartigimod is a treatment option for IMC in addition to IVIg and plasma exchange.

This study aims to determine the serum and cerebrospinal fluid (CSF) levels of neurofilament light chain (NFL) and phosphorylated neurofilament heavy chain (pNFH) in amyotrophic lateral sclerosis (ALS) patients, and to explore their feasibility as valid biomarkers for quantifying disease progression and predicting individual prognosis. 52 patients with ALS and 30 controls with noninflammatory neurological diseases were included. NFL and pNFH levels in serum and CSF were measured by enzyme‐linked immunosorbent assay. Our findings showed that serum and CSF levels of NFL and pNFH in ALS patients were significantly increased. These values were negatively correlated with disease duration (except CSF NFL with disease duration) and ALSFRS‐r score, and positively correlated with disease progression rate (DPR) and upper motor neuron (UMN) score, but did not correlate with bilateral median and ulnar nerve compound muscle action potential (cMAP) amplitudes (except a weak correlation between CSF NFL and cMAP amplitudes). The optimal cut‐off values with high sensitivity and specificity were obtained in ROC curve analysis to discriminate ALS from controls. Kaplan‐Meier survival curves illustrated that survival was significantly shorter for patients with higher neurofilament levels at diagnosis. The Cox proportional hazards regressions confirmed that NFL and pNFH were significant predictors of survival. Overall, NFL and pNFH in serum and CSF can be used as reliable biomarkers in ALS.

Objectives: The aims of this study were to investigate the differences of fasciculations detected by muscle ultrasonography (MUS) among patients with amyotrophic lateral sclerosis (ALS), patients with ALS mimics and healthy controls, and to propose a simplified MUS fasciculation score for the diagnosis of ALS. Methods: We included 16 patients with ALS (ALS group), 10 patients with ALS mimics (disease-control group), and 10 healthy adults (healthy control group). Subjects underwent MUS in 11 muscles, including the tongue, and bilateral upper trapezius, biceps brachii, abductor pollicis brevis, rectus femoris, and tibialis anterior. Results: The number of muscles with fasciculations per person was more in the ALS group (6.44 ± 2.56) than in the disease-control group (1.20 ± 1.87, P = 0.001) and healthy control group (0.50 ± 1.08, P < 0.001). Fasciculations in 3 of 11 muscles could predict the ALS diagnosis with high sensitivity (88.2%) and specificity (94.7%). Conclusions: Fasciculations detected by MUS can be a simple and useful diagnostic tool for ALS.