ObjectiveTo evaluate the safety and efficacy of efgartigimod in patients with generalized myasthenia gravis (MG) enrolled in the ADAPT+ long-term extension study.BackgroundTreatment with efgartigimod, a human IgG1 antibody Fc-fragment that blocks neonatal Fc receptor, resulted in clinically meaningful improvement (CMI) in MG-specific outcome measures in the ADAPT phase 3 clinical trial. All patients who completed ADAPT were eligible to enroll in its ongoing open-label, 3-year extension study, ADAPT+.Design/MethodsEfgartigimod (10 mg/kg IV) was administered in cycles of once-weekly infusions for 4 weeks, with subsequent cycles initiated based on clinical evaluation. Efficacy was assessed during each cycle utilizing Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scales.ResultsNinety-one percent of ADAPT patients (151/167) entered ADAPT+. As of February 2021, 106 AChR-Ab+ and 33 AChR-Ab– patients had received at least 1 dose of open-label efgartigimod (including 66 ADAPT placebo [PBO] patients). The mean (SD) study duration was 363 (114) days, resulting in 138 patient-years of observation. Similar incidence rates per patient year (IR/PY) of serious adverse events were seen in ADAPT (efgartigimod: 0.11; placebo: 0.29) compared to ADAPT+ (0.25). Five deaths (acute myocardial infarction, COVID-19 pneumonia/septic shock, bacterial pneumonia/MG crisis, malignant lung neoplasm, and unknown [multiple cardiovascular risk factors identified on autopsy]) occurred; none were considered related to efgartigimod by the investigator. AEs were predominantly mild or moderate. CMI was observed in AChR-Ab+ patients during each cycle (up to 10 cycles) at magnitudes comparable to improvements observed at week 3 of cycle 1 (mean[SE] improvements: MG-ADL, –5.1[0.34]; QMG, –4.7[0.41]). Clinical improvements mirrored maximal reductions in total IgG and AChR-Abs across all cycles.ConclusionsThis analysis suggests the efficacy of long-term treatment with efgartigimod was consistent across multiple cycles. No new safety signals were identified, despite being conducted before vaccine availability during the COVID-19 pandemic.

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The US Army Corps of Engineers (USACE), St. Louis District (MVS), manages multiple lock and dam structures on the Mississippi River. One of these, Melvin Price Locks and Dam (MPLD), was the subject of at least 12 allision events from downbound (southbound) vessels between January and November 2018 according to US Coast Guard (USCG) records, an unusually high number for this location. In an effort to understand how vessel operations change under varying river conditions, historical river gauge data and historical vessel position data for both upbound (northbound) and downbound (southbound) traffic were examined together to describe general approach paths for vessels at different water levels. Historic tracks for vessels involved in allision events are not included in this work because of ongoing investigations at the time of publication.

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ObjectiveTo evaluate real-world utilization patterns of intravenous immunoglobulin (IVIg) among patients with generalized myasthenia gravis (gMG) over 3 years post-IVIg initiation.BackgroundgMG is a rare autoimmune neuromuscular disorder with no known cure. Although IVIg is the most commonly used add-on therapy after standard of care treatments in gMG, it is currently unclear whether it is more commonly used as a “one-off” treatment to manage exacerbations, or as maintenance therapy aimed at reducing or replacing steroid use.Design/MethodsPatients with gMG who initiated IVIg treatment were identified from a US claims database (Symphony Health, an ICON plc Company, Integrated Dataverse [IDV]®, January 1, 2014-December 31, 2019). The frequency of subsequent IVIg treatment and associated cost during the first 12 months post-IVIg initiation were analyzed. Usage patterns of concomitant gMG treatments during the year preceding and 3 years post-IVIg initiation were compared.ResultsAmong 1225 patients with gMG who initiated IVIg treatment, 706 patients (57.6%) received 1 to 5 IVIg treatment courses (intermittent IVIg users), and 519 patients (42.4%) received = 6 IVIg treatment courses (chronic IVIg users) within the subsequent year. Mean annual medical cost per patient was nearly 2.5-fold higher for chronic vs intermittent IVIg users ($161,478 vs $64,888, p < 0.001). Usage frequency of other immunotherapy treatments did not decrease over the follow-up period, even for patients who continued annual chronic IVIg for 3 consecutive years post-initiation.ConclusionsNearly half of patients with gMG received chronic and multiple IVIg treatment courses within the first year once initiating IVIg treatment, indicating higher usage than expected. For all IVIg initiators, usage frequency of other immunotherapy treatments did not decrease over 3 years despite IVIg initiation.