Real-World experience with efgartigimod in patients with myasthenia gravis.

Recommendations for the treatment of myasthenia gravis (MG) have been difficult to develop because of limited evidence from large randomized controlled trials. New drugs and treatment approaches have recently been shown to be effective in phase 3 studies in seropositive generalized (g) MG. One such drug is efgartigimod, a human-Fc-fragment of IgG1, with a high affinity for the endosomal FcRn. We conducted a multicenter study to evaluate the real-world clinical and safety effects of efgartigimod in 22 gMG patients. We evaluated the strategies for the timing of re-treatment with it. The participants received a total of 59 efgartigimod -treatment cycles. The median number of cycles was 2 (range 1-6). Twenty patients (86.3%) improved by at least 2 MG-ADL points after the first treatment cycle. The median MG-ADL score at baseline was 6.5 (range: 3-17) and 2.5 (range: 0-9) post-treatment (p < 0.001). A consistent improvement of at least 2 points in the MG-ADL score after each cycle occurs in 18 patients. The effect duration of the treatment was usually between 4 and 12 weeks. Two major clinical patterns of treatment response were found. Treatment with efgartigimod was also associated with significant reductions of prednisone doses Overall, the treatment was safe and associated with only minor adverse events. The single fatality was apparently due tosevere respiratory failure. We found that efgartigimod is clinically effective, may be used as a steroid sparing agent and is generally safe for gMG patients. We recommend a personalized preventive treatment approach until clinical stabilization, followed by discontinuation and periodic evaluations.

Impact of high-resolution intracranial vessel wall magnetic resonance imaging on diagnosis in patients with embolic stroke of unknown source.

BACKGROUND: The mechanism responsible for stroke in patients with embolic stroke of unknown source (ESUS) often remains unknown despite extensive investigations. We aimed to test whether high-resolution intracranial vessel wall MR imaging (icVWI) can add to the diagnostic yield in these patients. PATIENTS AND METHODS: Patients with ESUS were prospectively included into an ongoing registry. Patients that underwent icVWI as part of their diagnostic workup were compared to those that did not have an icVWI. Patients with icVWI positive for intracranial vulnerable plaques were than compared to those without evidence of plaque vulnerability on VWI. RESULTS: A total of 179 patients with ESUS were included and 48 of them (27%) underwent icVWI. Patients that had an icVWI scan were significantly younger, had lower rates of ischemic heart disease and prior disability as well as significantly lower stroke severity. On regression analysis the only factor that remained associated with not obtaining an icVWI scan was increasing age (Odds ratio [OR] 0.97/year, 95% confidence intervals [CI] 0.95-0.97). Among patients that had an icVWI scan 28 (58%) had evidence of plaque enhancement on VWI in the same distribution of the stroke and the remaining 20 studies were negative. The relative proportion of stroke presumed to be secondary to intracranial non-stenotic atheromatous disease increased from 15% in patients without icVWI scans to 58% among patients with icVWI scans (p = 0.001). On regression analysis the only factor that was associated with vulnerable plaques on icVWI was smoking (OR 11.05 95% CI 1.88-65.17). CONCLUSIONS: icVWI can add significant information relevant to stroke pathogenesis and treatment in patients with ESUS and a negative initial exhaustive diagnostic workup.

An uncommon presentation of paraneoplastic limbic encephalitis.

Paraneoplastic limbic encephalitis (PLE) is a rare disease with established diagnostic criteria. We describe a case of an uncommon presentation of PLE in a female who presented with a one- year duration of short-term memory loss and mild behavioral changes who was eventually diagnosed with PLE associated with breast cancer. Our case demonstrates atypical presentation of PLE, with chronic presentation and an uncharacteristic mild neurological symptoms. This case aims to highlight the importance of a diagnostic work up of autoimmune encephalitis in selected cases that does not present with common diagnostic criteria.