Long-term Safety and Efficacy of Efgartigimod in Patients With Generalized Myasthenia Gravis: Interim Results of the ADAPT+ Study

Objective To evaluate the safety and efficacy of efgartigimod in patients with generalized myasthenia gravis (MG) enrolled in the ADAPT+ longterm extension study. Background Treatment 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/Methods Efgartigimod (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. Results Ninety-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. Conclusions This 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.

COVID-19 mortality may be reduced among fully vaccinated solid organ transplant recipients.

BACKGROUND: Solid organ transplant (SOT) recipients are at increased risk for morbidity and mortality from COVID-19 due to their immunosuppressed state and reduced immunogenicity from COVID-19 mRNA vaccines. This investigation examined the association between COVID-19 mRNA vaccination status and mortality among SOT recipients diagnosed with COVID-19. METHODS & FINDINGS: A retrospective, registry-based chart review was conducted investigating COVID-19 mortality among immunosuppressed solid organ transplant (SOT) recipients in a large metropolitan healthcare system in Houston, Texas, USA. Electronic health record data was collected from consecutive SOT recipients who received a diagnostic SARS-CoV-2 test between March 1, 2020, and October 1, 2021. The primary exposure was COVID-19 vaccination status at time of COVID-19 diagnosis. Patients were considered 'fully vaccinated' at fourteen days after completing their vaccine course. COVID-19 mortality within 60 days and intensive care unit admission within 30 days were primary and secondary endpoints, respectively. Among 646 SOT recipients who were diagnosed with COVID-19 at Houston Methodist Hospital between March 2020, and October 2021, 70 (10.8%) expired from COVID-19 within 60 days. Transplanted organs included 63 (9.8%) heart, 355 (55.0%) kidney, 108 (16.7%) liver, 70 (10.8%) lung, and 50 (7.7%) multi-organ. Increasing age was a risk factor for COVID-19 mortality, while vaccination within 180 days of COVID-19 diagnosis was protective in Cox proportional hazard models with hazard ratio 1.04 (95% CI: 1.01-1.06) and 0.31 (0.11-0.90), respectively). These findings were confirmed in the propensity score matched cohort between vaccinated and unvaccinated patients. CONCLUSIONS: This investigation found COVID-19 mortality may be significantly reduced among immunosuppressed SOT recipients within 6 months following vaccination. These findings can inform vaccination policies targeting immunosuppressed populations worldwide.

Hospitalization and survival of solid organ transplant recipients with coronavirus disease 2019: A propensity matched cohort study.

BACKGROUND: Solid organ transplant (SOT) recipients are predicted to have worse COVID-19 outcomes due to their compromised immunity. However, this association remains uncertain because published studies have had small sample sizes and variability in chronic comorbidity adjustment. METHODS: In this retrospective cohort study conducted at a multihospital health system, we compared COVID-19 outcomes and survival up to 60 days following hospital admission in SOT recipients taking baseline immunosuppressants versus hospitalized control patients. RESULTS: The study included 4,562 patients who were hospitalized with COVID-19 (108 SOT recipients and 4,454 controls) from 03/2020 to 08/2020. Mortality at 60 days was higher for SOT recipients (17% SOT vs 10% control; unadjusted odds ratio (OR) = 1.74, 95% confidence interval (CI) 1.04-2.91, P = 0.04). We then conducted a 1:5 propensity matched cohort analysis (100 SOT recipients; 500 controls) using age, sex, race, body mass index, hypertension, diabetes, chronic kidney disease, liver disease, admission month, and area deprivation index. Within 28 days of admission, SOT recipients had fewer hospital-free days (median; 17 SOT vs 21 control; OR = 0.64, 95%CI 0.46-0.90, P = 0.01) but had similar ICU-free days (OR = 1.20, 95%CI 0.72-2.00, P = 0.49) and ventilator-free days (OR = 0.91, 95%CI 0.53-1.57, P = 0.75). There was no statistically significant difference in 28-day mortality (9% SOT vs 12% control; OR = 0.76, 95%CI 0.36-1.57, P = 0.46) or 60-day mortality (16% SOT vs 14% control; OR = 1.15, 95%CI 0.64-2.08, P = 0.64). CONCLUSIONS: Hospitalized SOT recipients appear to need additional days of hospital care but can achieve short-term mortality outcomes from COVID-19 that are similar to non-SOT recipients in a propensity matched cohort study.

Theme 12 - Clinical Management and Support

Power wheelchair prescription, utilization, satisfaction, and cost for patients with amyotrophic lateral sclerosis: preliminary data for evidence-based guidelines. Stage at which riluzole treatment prolongs survival in patients with amyotrophic lateral sclerosis: a retrospective analysis of data from a dose-ranging study. Support needs and interventions for family caregivers of patients with amyotrophic lateral sclerosis (ALS): a narrative review with report of telemedicine experiences at the time of COVID-19 pandemic. [Extracted from the article]

Sources of heterogeneity in studies reporting the venous thrombotic risk in patients admitted with COVID-19: A meta-analysis and explorative meta-regression

Background: Meta-analyses on venous thromboembolism (VTE) risk in admitted patients with COVID-19 have shown substantial heterogeneity among included studies. Aim(s): To explore sources of heterogeneity among studies estimating VTE risk in COVID-19 patients. Method(s): A systematic review using the databases PubMed and Embase was performed searching for studies reporting VTE risk in patients admitted to the ward or intensive care unit (ICU). Analysis was performed with studies from which a incidence proportion could be retrieved. The pooled incidence of VTE and heterogeneity (I2) were estimated in a random-effects model stratified by ICU or ward. Incidence estimates were logit-transformed. The effect of 12 pre-selected clinical and methodological variables were explored in a univariable linear meta-regression model. Because of limited degrees of freedom, a multivariable model was constructed in a stepwise fashion. The outcome measure was regression coefficient with a 95% confidence interval (CI). Result(s): Fifty-three and 26 studies reported on incidence of VTE at the ICU and ward respectively. The pooled incidence at the ICU was 20.3% (95% CI 16.4-24.8%, I2 95.4%) and at the ward was 4.7% (95% CI 3.0-7.3%, I2 96.2%). In studies concerning ICU patients, asian region, VTE-screening, standard error and date of publication were significantly associated with incidence of VTE in the univariable model. (Table Presented) Two multivariable models were fitted (Table 1). In the multivariable model only standard error remained significantly associated. In studies concerning ward patients, average age, VTE-screening, standard error were significantly associated in the univariable model. Two multivariable models were fitted (Table 2). Again, only standard error remained significantly associated. Conclusion(s): Studies with a high standard error yielded a higher VTE incidence in COVID-19, strongly suggesting publication bias. Remaining heterogeneity was not completely explained, but may be due to differences in setting/outcome definition and clinical practice.

Fulminant lung fibrosis in non-resolvable COVID-19 requiring transplantation.

BACKGROUND: Coronavirus Disease 2019 (COVID-19) can lead to the development of acute respiratory distress syndrome (ARDS). In some patients with non-resolvable (NR) COVID-19, lung injury can progress rapidly to the point that lung transplantation is the only viable option for survival. This fatal progression of lung injury involves a rapid fibroproliferative response and takes on average 15 weeks from initial symptom presentation. Little is known about the mechanisms that lead to this fulminant lung fibrosis (FLF) in NR-COVID-19. METHODS: Using a pre-designed unbiased PCR array for fibrotic markers, we analyzed the fibrotic signature in a subset of NR-COVID-19 lungs. We compared the expression profile against control lungs (donor lungs discarded for transplantation), and explanted tissue from patients with idiopathic pulmonary fibrosis (IPF). Subsequently, RT-qPCR, Western blots and immunohistochemistry were conducted to validate and localize selected pro-fibrotic targets. A total of 23 NR-COVID-19 lungs were used for RT-qPCR validation. FINDINGS: We revealed a unique fibrotic gene signature in NR-COVID-19 that is dominated by a hyper-expression of pro-fibrotic genes, including collagens and periostin. Our results also show a significantly increased expression of Collagen Triple Helix Repeat Containing 1(CTHRC1) which co-localized in areas rich in alpha smooth muscle expression, denoting myofibroblasts. We also show a significant increase in cytokeratin (KRT) 5 and 8 expressing cells adjacent to fibroblastic areas and in areas of apparent epithelial bronchiolization. INTERPRETATION: Our studies may provide insights into potential cellular mechanisms that lead to a fulminant presentation of lung fibrosis in NR-COVID-19. FUNDING: National Institute of Health (NIH) Grants R01HL154720, R01DK122796, R01DK109574, R01HL133900, and Department of Defense (DoD) Grant W81XWH2110032 to H.K.E. NIH Grants: R01HL138510 and R01HL157100, DoD Grant W81XWH-19-1-0007, and American Heart Association Grant: 18IPA34170220 to H.K.-Q. American Heart Association: 19CDA34660279, American Lung Association: CA-622265, Parker B. Francis Fellowship, 1UL1TR003167-01 and The Center for Clinical and Translational Sciences, McGovern Medical School to X.Y.

Effectiveness of Eculizumab Treatment for Generalized Myasthenia Gravis in Us Clinical Practice: GMG Registry Data

INTRODUCTION: Clinical trial data have demonstrated that eculizumab improves clinical outcomes in individuals with refractory generalized myasthenia gravis (gMG). Data from clinical practice on treatment patterns and effectiveness of eculizumab in gMG are being collected by the Alexion-sponsored gMG Registry. OBJECTIVE: To describe treatment outcomes and safety (serious adverse events [SAEs]) in current gMG Registry participants during eculizumab therapy in clinical practice in the USA. METHODS: Starting in December 2019, adults with gMG who had ever received eculizumab enrolled in the gMG Registry (NCT04202341). After obtaining consent, demographic data, myasthenia gravis activities of daily living (MG-ADL) total score and Myasthenia Gravis Foundation of America (MGFA) classification were collected from medical records at two time points: In the 6 months before eculizumab initiation and at first gMG Registry assessment after eculizumab treatment initiation (at Registry enrollment). SAEs in patients receiving eculizumab during Registry participation were recorded. RESULTS: As of November 29, 2021, in total, 111 adults with gMG had enrolled in the gMG Registry (male, 52.3%;mean [range] age at MG diagnosis, 56.1 [16.0-92.0] years). The mean (range) time from eculizumab initiation to gMG Registry enrollment was 2.0 (0.0-6.7) years. Mean (standard deviation) MG-ADL total score decreased from 8.3 (3.6) before eculizumab initiation to 3.1 (3.6) after eculizumab treatment. MGFA classification improved with eculizumab treatment: Class I, 0.0% of patients before eculizumab initiation versus 28.9% after eculizumab treatment;class II, 36.8% versus 55.3%;class III, 52.6% versus 15.8%;class IV, 10.5% versus 0.0%. The median MGFA class was III before eculizumab initiation and II after eculizumab treatment. One SAE (invasive pulmonary aspergillosis) considered by the investigator to be related to eculizumab was reported in a patient who died, and two serious infections considered unrelated to eculizumab were reported (COVID-19/pneumonia and urinary tract infection);there were no meningococcal infections. Two patients died of causes considered to be unrelated to eculizumab (lung adenocarcinoma and acute congestive heart failure/myocardial infarction). CONCLUSION: These data from the gMG Registry provide evidence of the effectiveness of eculizumab for the treatment of gMG in clinical practice across the USA, demonstrating a benefit/risk profile consistent with that observed in clinical trials.

mTOR inhibitors, mycophenolates, and other immunosuppression regimens on antibody response to SARS-CoV-2 mRNA vaccines in solid organ transplant recipients.

A recent study concluded that SARS-CoV-2 mRNA vaccine responses were improved among transplant patients taking mTOR inhibitors (mTORi). This could have profound implications for vaccine strategies in transplant patients; however, limitations in the study design raise concerns about the conclusions. To address this issue more robustly, in a large cohort with appropriate adjustment for confounders, we conducted various regression- and machine learning-based analyses to compare antibody responses by immunosuppressive agents in a national cohort (n = 1037). MMF was associated with significantly lower odds of positive antibody response (aOR = 0.09 0.130.18 ). Consistent with the recent mTORi study, the odds tended to be higher with mTORi (aOR = 1.00 1.452.13 ); however, importantly, this seemingly protective tendency disappeared (aOR = 0.47 0.731.12 ) after adjusting for MMF. We repeated this comparison by combinations of immunosuppression agents. Compared to MMF + tacrolimus, MMF-free regimens were associated with higher odds of positive antibody response (aOR = 2.39 4.267.92 for mTORi+tacrolimus; 2.34 5.5415.32 for mTORi-only; and 6.78 10.2515.93 for tacrolimus-only), whereas MMF-including regimens were not, regardless of mTORi use (aOR = 0.81 1.542.98 for MMF + mTORi; and 0.81 1.512.87 for MMF-only). We repeated these analyses in an independent cohort (n = 512) and found similar results. Our study demonstrates that the recently reported findings were confounded by MMF, and that mTORi is not independently associated with improved vaccine responses.

A pilot randomized controlled trial of de novo belatacept-based immunosuppression following anti-thymocyte globulin induction in lung transplantation.

The development of donor-specific antibodies (DSA) after lung transplantation is common and results in adverse outcomes. In kidney transplantation, Belatacept has been associated with a lower incidence of DSA, but experience with Belatacept in lung transplantation is limited. We conducted a two-center pilot randomized controlled trial of de novo immunosuppression with Belatacept after lung transplantation to assess the feasibility of conducting a pivotal trial. Twenty-seven participants were randomized to Control (Tacrolimus, Mycophenolate Mofetil, and prednisone, n = 14) or Belatacept-based immunosuppression (Tacrolimus, Belatacept, and prednisone until day 89 followed by Belatacept, Mycophenolate Mofetil, and prednisone, n = 13). All participants were treated with rabbit anti-thymocyte globulin for induction immunosuppression. We permanently stopped randomization and treatment with Belatacept after three participants in the Belatacept arm died compared to none in the Control arm. Subsequently, two additional participants in the Belatacept arm died for a total of five deaths compared to none in the Control arm (log rank p = .016). We did not detect a significant difference in DSA development, acute cellular rejection, or infection between the two groups. We conclude that the investigational regimen used in this study is associated with increased mortality after lung transplantation.

Predicting a Positive Antibody Response After 2 SARS-CoV-2 mRNA Vaccines in Transplant Recipients: A Machine Learning Approach With External Validation.

BACKGROUND: Solid organ transplant recipients (SOTRs) are less likely to mount an antibody response to SARS-CoV-2 mRNA vaccines. Understanding risk factors for impaired vaccine response can guide strategies for antibody testing and additional vaccine dose recommendations. METHODS: Using a nationwide observational cohort of 1031 SOTRs, we created a machine learning model to explore, identify, rank, and quantify the association of 19 clinical factors with antibody responses to 2 doses of SARS-CoV-2 mRNA vaccines. External validation of the model was performed using a cohort of 512 SOTRs at Houston Methodist Hospital. RESULTS: Mycophenolate mofetil use, a shorter time since transplant, and older age were the strongest predictors of a negative antibody response, collectively contributing to 76% of the model's prediction performance. Other clinical factors, including transplanted organ, vaccine type (mRNA-1273 versus BNT162b2), sex, race, and other immunosuppressants, showed comparatively weaker associations with an antibody response. This model showed moderate prediction performance, with an area under the receiver operating characteristic curve of 0.79 in our cohort and 0.67 in the external validation cohort. An online calculator based on our prediction model is available at http://transplantmodels.com/covidvaccine/ . CONCLUSIONS: Our machine learning model helps understand which transplant patients need closer follow-up and additional doses of vaccine to achieve protective immunity. The online calculator based on this model can be incorporated into transplant providers' practice to facilitate patient-centric, precision risk stratification and inform vaccination strategies among SOTRs.

Spontaneous intracranial hemorrhage presenting in a patient with vitamin K deficiency and COVID-19: illustrative case

BACKGROUND Coronavirus disease 2019 (COVID-19) is known to cause more severe symptoms in the adult population, but pediatric patients may experience severe neurological symptoms, including encephalopathy, seizures, and meningeal signs. COVID-19 has also been implicated in both ischemic and hemorrhagic cerebrovascular events. This virus inhibits angiotensin-converting enzyme 2, decreasing angiotensin (1–7), decreasing vagal tone, disrupting blood pressure autoregulation, and contributing to a systemic vascular inflammatory response, all of which may further increase the risk of intracranial hemorrhage. However, there has only been one reported case of intracranial hemorrhage developing in a pediatric patient with COVID-19. OBSERVATIONS The authors discuss the first case of a pediatric patient with COVID-19 presenting with intracranial hemorrhage. This patient presented with lethargy and a bulging fontanelle and was found to have extensive intracranial hemorrhage with hydrocephalus. Laboratory tests were consistent with hyponatremia and vitamin K deficiency. Despite emergency ventriculostomy placement, the patient died of his disease. LESSONS This case demonstrates an association between COVID-19 and intracranial hemorrhage, and the authors have described several different mechanisms by which the virus may potentiate this process. This role of COVID-19 may be particularly important in patients who are already at a higher risk of intracranial hemorrhage, such as those with vitamin K deficiency.

Real-world experience of eculizumab treatment for generalized myasthenia gravis: initial data from a registry of patients with generalized myasthenia gravis in the USA

Objective: To describe the baseline characteristics and interim treatment outcomes of registry participants with generalized myasthenia gravis (gMG) treated with eculizumab in real-world clinical practice in the USA. Background: Clinical trials have shown that eculizumab improves clinical outcomes in individuals with refractory gMG. The Alexion-sponsored gMG Registry collects real-world data on treatment patterns and effectiveness of eculizumab in gMG. Design/Methods: Adults with gMG who had ever received eculizumab enrolled in the Alexion-sponsored gMG Registry (NCT04202341) from December 2019. After consent, demographic data, Myasthenia Gravis (MG) activities of daily living (ADL) total scores, MG Foundation of America (MGFA) classification and serious adverse events (SAEs) were collected from medical records at two time points: before eculizumab initiation (within prior 6 months) and at Registry enrollment (first Registry assessment during eculizumab treatment). Results: As of February 28, 2021, 59 adults with gMG had enrolled in the Registry (male, 54.2%;mean [range] age at MG diagnosis, 58.3 [16.0-84.0] years). Mean (range) time from eculizumab initiation to Registry enrollment was 697.1 (7.0-2435.0) days. Mean (standard deviation) MG-ADL total score decreased with eculizumab, from 8.3 (3.71) before eculizumab to 3.1 (3.43) at first Registry assessment during treatment (n=38). MGFA classification improved with eculizumab (n=32): before eculizumab, MG was class I (0.0% of patients), II (40.6%), III (53.1%) or IV (6.3%);at first Registry assessment during treatment, MG was class I (25.0%), II (62.5%), III (12.5%) or IV (0.0%). SAEs (lung adenocarcinoma, respiratory failure, atrial fibrillation, heart failure, colostomy closure, COVID-19, death) were reported in six patients. There were two deaths (unrelated to eculizumab). No meningococcal infections were reported. Conclusions: Initial analyses of Registry data showed that MG-ADL total score and MGFA classification improved with eculizumab, and that eculizumab was generally well tolerated. These findings provide additional evidence of the effectiveness of eculizumab for treating gMG in real-world clinical practice.

Long-term Safety, Tolerability, and Efficacy of Efgartigimod in Patients With Generalized MyastheniaGravis: Interim Results of the ADAPT+ Study

Objective: Evaluate the safety, tolerability, and efficacy of efgartigimod in patients with generalized myasthenia gravis (MG) enrolled in the ADAPT+ long-term extension study. Background: Treatment 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 study. All patients who completed ADAPT were eligible to enroll in its ongoing open-label, 3-year extension study, ADAPT+. Design/Methods: Efgartigimod (EFG), 10 mg/kg, was administered intravenously in cycles of once-weekly infusions for 4 weeks, with subsequent cycles initiated based on predefined criteria. Efficacy was assessed during each cycle utilizing Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scales. Results: Ninety-one percent of ADAPT patients (151/167) entered ADAPT+. As of February 2021, 106 AChR-Ab+ and 33 AChR-Ab- 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 rates of the most common adverse events (AEs) were seen in the EFG-EFG and PBO-EFG arms: headache (15.1%/30.3%), nasopharyngitis (8.2%/13.6%), and diarrhea (6.8%/10.6%). 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. Similar results were observed in AChR-Ab- patients. Conclusions: This analysis suggests long-term treatment with efgartigimod was well-tolerated and efficacious, regardless of antibody status. Despite being conducted during the COVID-19 pandemic, before vaccine availability, no new safety signals were identified.

A prospective natural history study and biorepository for patients with myasthenia gravis (EXPLORE-MG2)

Objective: To define clinical features, disease course, and collect biospecimens in patients with myasthenia gravis (MG), including understudied subgroups such as seronegative, muscle-specific kinase antibody (MuSK), and LRP4 antibody positive MG. Background: The MG Rare Disease Clinical Research Network (MGNet) was funded by the National Institutes of Health (NIH) to gain better understanding of the clinical course of MG and develop improved approaches to diagnosis and treatment. As part of this initiative a multicenter prospective natural history study and biorepository was developed: Exploring Outcomes and Characteristics of Myasthenia Gravis 2 (EXPLORE-MG2). Design/Methods: EXPLORE-MG2 is a web-based observational registry that incorporates NIH recommended common data elements for MG. Key eligibility criteria include: ≥18 years old;diagnosed with MG within 2-years of study enrollment based on clinical presentation and seropositivity for MG associated autoantibodies, and/or abnormal neurophysiology test or positive edrophonium test. Biospecimen collection focuses on immunosuppressive naïve patients and rare MG subgroups. Participants will be followed for at least 2-years with study visits occurring approximately every 6 months in the context of usual clinical care. The study has been open to enrollment since January 2021 with 6 sites currently activated/participating. Results: A total of 62 patients have been enrolled and 152 biospecimens were collected as of 10/1/2021. The mean age was 57 years (range 20-84);47% were female, 66% were acetylcholine receptor antibody-positive, 16% were MuSK MG, and 18% were seronegative. Enrollment of new participants and follow-up of existing participants are ongoing to reach our current goal of 400 enrolled participants. We will present updated enrollment data and demographics at the meeting. Conclusions: The EXPLORE-MG2 study is active after a COVID-19 pandemic related delay. Samples and clinical data will be available to researchers for current and future investigation. Data from EXPLORE-MG2 will improve clinical trial readiness for future studies and facilitate development of treatment-responsive biomarkers.

Factors associated with utilization of telemedicine during the COVID-19 pandemic among patients with myasthenia gravis: A retrospective regional cohort study

Objective: 1. Describe telemedicine utilization (TMU) during the COVID-19 pandemic among neurology outpatients with myasthenia gravis (MG);2. Analyze characteristics of patients completing virtual video visits (VVV) compared to audio visits only (AVO). Background: TMU data by patients with MG are limited. Effects of demographic characteristics on MG patients' TMU and choice between VVV and AVO are unknown. Design/Methods: We performed a retrospective cohort study using Mass General Brigham's (MGB) data warehouse. Inclusion criteria were: 1) adult patients with autoimmune MG (ICD10 code = G70.∗);2) ≥ 1 outpatient neurology encounter for MG among 4 MGB hospitals April 1, 2020 - March 31, 2021. Chart review excluded congenital myasthenic syndromes, LambertEaton myasthenic syndrome and patients without a confirmed MG diagnosis. Primary outcome was any TMU. Secondary outcome was proportion of telemedicine users achieving ≥ 1 VVV. EMR portal status was a proxy for digital literacy and access. Analyses included descriptive statistics, Chi Square test, and multivariable logistic regression. Results: 459 MG patients completed 1083 neurology encounters: 451 (41.6%) in-person, 481 (44.4%) VVV and 151 (13.9%) AVO. 336/459 (73.2%) achieved telemedicine utilization (TMU). Baseline patient characteristics (age, sex, race/ethnicity, language, state of residence, insurance, distance to neurologist, portal access) were not associated with TMU. Patients with a MGB PCP were less likely to participate in telemedicine (OR 0.60 [0.36-0.99]). Of 336 patients with TMU, 259 (77.1%) had ≥ 1 VVV, while 77 (22.9%) had AVO. The AVO group had a smaller proportion of white patients (76.6% vs.84.3%, p=0.031). In adjusted analysis, no characteristics remained associated with VVV achievement. Conclusions: During 1 year of COVID-19, this MG cohort had high utilization rates of TM (73.2%) and VVV (77.1%). Older age, non-English language and portal inactivity were not associated with lower rates of TMU and VVV. Future studies should assess regional variation and outcomes of TMU in MG.

An Evaluation of the Quality of COVID-19 Internet Resources for Cancer Patients.

Cancer patients may face difficulty evaluating web-based COVID-19 resources in context with their cancer diagnosis. The purpose of this study is to systematically evaluate educational resources available for cancer patients seeking online information on COVID-19 and cancer. The term "COVID-19 and Cancer" was searched in Google and metasearch engines Yippy and Dogpile. After applying inclusion and exclusion criteria, the results from the 3 lists were systematically combined for a final ranked list. This list was analyzed using a validated structured rating tool with respect to accountability, interactivity, organization, readability, and content coverage and accuracy. Three hundred ninety-eight websites were identified, and 37 websites were included for analysis. Only 43% of sites disclosed authorship, 24% cited sources, and 32% were updated within 3 months of the search date. Fifty-four percent of websites had high school readability (8.0-12.0), 43% were at university level or above, and no websites demonstrated the recommended reading level for health information for the public (< 6.0). Topics most discussed were special considerations for cancer patients during COVID-19 (84%) and COVID-19 risk factors (73%). Topics least covered were COVID-19 incidence/prevalence (5%) and prognosis (8%). There is some COVID-19 information for cancer patients available online, but quality is variable. Healthcare professionals may direct cancer patients to the most reliable COVID-19 and cancer websites shown in this study and results may be helpful when designing future online health information resources.