The study examines efgartigimod and intravenous immunoglobulin (IVIg) in elderly patients with generalized myasthenia gravis (GMG), focusing on changes in Myasthenia Gravis Activities of Daily Living (MG-ADL) scores, pyridostigmine dosage, and minimal symptom expression (MSE) over an 8-week period. Among 74 enrolled patients, efgartigimod showed greater reduction in MG-ADL scores compared to IVIg at weeks 4 and 8, with no serious adverse events, suggesting its superior efficacy and safety in elderly Chinese patients with acetylcholine receptor antibody-positive (AChR-Ab(+)) GMG.

Myasthenia gravis (MG) specific outcome measures are being used in clinical trials to evaluate therapeutic effectiveness. These validated tools are also becoming a necessity in clinical practice, with payors in the US market often requiring them to be used to monitor disease state. There is considerable variation and subjectivity regarding their use. This study aimed to develop consensus-based recommendations for the standardization of MG specific outcome measures in clinical practice.

A panel of 10 US-based MG specialists developed consensus-based recommendations based on three rounds of formal voting using the UCLA-RAND appropriateness method after surveying myasthenia gravis clinicians and developing a focus group.

Twenty one expert consensus statements based on six themes were developed following clinician survey result review and focus group theme development. Some key recommendations include: the MGFA Clinical Classification assesses disease at that examination and should be updated at intervals of 3-6 months to reflect current clinical status. MGFA PIS represents the overall clinical judgment of the evaluator without the requirement for a defined change in scores on any outcome measure. Patient-reported items, such as MG-ADL and MGC, should be referenced to the previous 1 week to optimize recall. Additional recommendations include scoring outcome measures in the presence of co-morbidity, scoring specific physical exam findings, and clarification regarding the administration of outcome measures.

This method provided expert consensus-based recommendations for the use of MG-specific outcome measures and exam findings to help standardize how they are used in clinical practice.

The therapeutic landscape in generalized myasthenia gravis (gMG) has been continuously evolving in recent years, with over five products approved, each with different efficacy, safety, and administration profiles. With the availability of new targeted treatments, physicians are faced with the challenge of therapeutic decision-making tailored to traditional therapeutic goals, individual preferences, and personal experience, seeking optimal disease control with a positive safety profile. In this context of uncertainty and multiple novel choices, this study aims to provide insights into the preferred treatment choices of neurologists managing gMG and to identify demographic, professional or behavioral factors influencing the decision-making process.

This is a non-interventional, cross-sectional, web-based study involving 150 neurologists treating gMG patients in collaboration with the Spanish Society of Neurology. The primary endpoint will be to assess preferences for different gMG treatment attributes using a conjoint analysis to create hypothetical treatment scenarios. Therapeutic inertia, described as the lack of treatment initiation or intensification when therapeutic goals are not met, will be evaluated as a secondary endpoint through 7 case scenarios simulating real gMG clinical practice situations. Neurologists will also answer a survey composed of demographic, professional, and behavioral characteristics (user resistance behavior, care-related regret, burnout, risk attitude, empathy, work fulfilment, and personality traits) to recognize possible factors influencing decisions.

The study findings will contribute to better understanding of decision-making in gMG under a changing therapeutic landscape with multiple new targeted options, and will identify which factors have a role in affecting those decisions.

The neonatal Fc receptor (FcRn) inhibitors efgartigimod and rozanolixizumab have not long been introduced for treating generalized myasthenia gravis (MG); hence, real-world evidence for their administration in multiple cycles and switching from intravenous to subcutaneous formulation remains insufficient.

We retrospectively assessed 17 consecutive patients with generalized MG and diverse backgrounds who were treated with FcRn inhibitors.

All patients initially received an intravenous efgartigimod formulation. Of 17 patients, 10 (59%) were considered responders, defined as a persistent improvement of at least two points for a minimum of four consecutive weeks in the MG activities of daily living score during the first treatment cycle. Four of the non-responders in the first cycle demonstrated an improvement in fulfilling the criteria for responders in the second cycle. One of these patients, who had thymoma metastatic lesions, experienced a significant worsening of MG symptoms during the first treatment cycle. Five patients switched from intravenous to subcutaneous formulations, which was successful in all patients. The efficacy of the subcutaneous formulations was similar to that of the intravenous formulation, even in patients who switched from efgartigimod to rozanolixizumab. The drugs were well tolerated without any drug-related serious adverse events irrespective of the formulation type.

FcRn inhibitors were effective and safe in patients with generalized MG, but their efficacy may depend on the disease activity during treatment. The transition from the intravenous formulation to more convenient subcutaneous formulations was successful, indicating the likely growth of future demand for subcutaneous formulations.

Myasthenia gravis (MG) clinical trials typically allow rescue therapy during follow-up in the event of marked worsening of MG symptoms. Failure to appropriately address rescue therapy in defining treatment effects and planning statistical analyses may yield biased estimates, increase false positive rates, or decrease statistical power - all of which increase the risk that inaccurate information influences patient care. In alignment with recent International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) guidelines, we review strategies based on the estimand framework that produce rigorously defined treatment effects in MG trials where rescue therapy may be administered. We also discuss the interpretation, clinical relevance, and pitfalls of each strategy in the context of MG trials, suggesting circumstances in which a strategy would or would not be appropriate. Finally, we outline available statistical methods for estimating treatment effects based on each strategy. As primary endpoints and statistical analyses for trials must be defined prior to conducting the study, it is necessary to consider how to address rescue therapy during study planning.

Efgartigimod for intravenous infusion (efgartigimod-IV) is approved in Japan for generalized myasthenia gravis (gMG). Post-marketing surveillance was mandated by regulatory authorities to assess the safety and effectiveness of efgartigimod in patients with gMG.

Patients with gMG who received efgartigimod-IV between May 2022 and September 2023 were registered. The interim analysis data were cutoff in June 2024 and included patients whose institutions agreed to publication.

The safety analysis set consisted of 373 patients: 53.35% (n = 199) anti-acetylcholine receptor antibody positive, 14.21% (n = 53) anti-muscle-specific receptor kinase antibody positive, and 31.64% (n = 118) double-seronegative. Adverse drug reaction and serious adverse drug reaction were reported in 21.45% (80/373) and 4.29% (16/373) of patients, respectively. Although six deaths were reported, none of them were related to efgartigimod. The effectiveness analysis set consisted of 246 patients. After three weeks from the first administration, mean score of MG-Activities of Daily Living decreased from 7.5 to 4.4: -3.1 points improvement (standard deviation: 2.95, p < 0.001). No remarkable differences were observed in the response to efgartigimod between the subgroups of patient baseline characteristics, e.g. autoantibody profiles.

In real-world settings, efgartigimod-IV was well tolerated and effective in patients with gMG.

Although fluctuating muscular weakness is the hallmark of myasthenia gravis (MG), research into it, especially during the course of the day, remains limited. Understanding the dynamics of myasthenic symptoms is essential for diagnosis and anticipatory treatment. Therefore, in this study, we used orthoptic and other established quantitative and subjective methods to measure ocular myasthenia gravis (OMG) symptoms throughout the day, during the course of 2 months, and in response to treatment.

The goal of the study was to determine the change of gaze deviation and ptosis during the course of the day, over 2 months, and 1 hour after pyridostigmine intake. Each subject participated in 3 sessions during the day, 2 follow-up sessions, and 1 measurement before and after pyridostigmine. Measurements included the Quantitative Myasthenia Gravis (QMG) score, palpebral fissure height (PFH) photography, the conventional Hess screen test, and a video Hess screen test using video-oculography. The Myasthenia Gravis Activities of Daily Living score (MG-ADL) was obtained on each assessment day. Sum scores were calculated for the gaze deviations of the inner and outer fields of the conventional and the video Hess screen tests.

Twelve patients were recruited, including 11 patients with ocular and 1 patient with generalized MG (mean age 65.7 years, SD 16.9 years; 11 males). The mean sum scores of both the conventional and the video Hess screen test showed a worsening in the evening, reaching significance in the outer field of the Hess screen test (mean increase 13.4°, SD 15.3°, P = 0.02). Similarly, ptosis also worsened during the day, with a significant decrease in PFH in the evening (mean decrease 0.53 mm, SD 0.55 mm, P = 0.04). Although ptosis improved significantly after pyridostigmine intake (mean increase 0.96 mm, SD 1.05, P = 0.03), no significant changes were observed in the sum deviations of the Hess screen tests (P = 0.6). Both ptosis and the sum scores generally improved over the 2-month period, even in some patients without any therapeutic adjustments. Correspondingly, the mean QMG and MG-ADL scores decreased.

This prospective cohort study provides insight into the dynamics of OMG, which is crucial for the optimization of diagnostic and therapeutic approaches. Our orthoptic measurements demonstrated the worsening of OMG symptoms after daily activity and a better response of ptosis to pyridostigmine than diplopia. The complexity of this fluctuating disease leads to strong interindividual variability, which requires an individual approach to improve the quality of life of patients with MG.

Myasthenia gravis (MG) is an autoimmune disorder primarily caused by autoantibodies against the acetylcholine receptor (AChR). Approximately 15% of MG patients, categorized as seronegative (snMG), lack detectable antibodies. Due to the snMG status, there may be a diagnostic delay. Moreover, there are limited data on treatment response in comparison to AChR-Ab+ patients. This study examines the burden of disease, treatment response, and quality of life of snMG patients in comparison to AChR-ab+ MG patients and healthy controls.

A questionnaire-based survey was conducted collecting sociodemographic and clinical data including antibody status, therapy, treatment response, and self-rated disease severity along with standardized assessments such as MG-ADL (activities of daily living) and the Short Form Health (SF-36, generic Health-Related Quality of Life, HRQoL). HRQoL was evaluated through matched-pairs analyses. Participants from a general health survey served as the control group. Negative binomial regression was applied to evaluate the impact of antibody status on MG-ADL.

Compared to AChR-ab+ patients, snMG patients (n = 237) were younger at symptom onset [median age 42 (IQR 30.5/53) vs. 51 (31/64) years, p < 0.001] and had longer diagnostic delays. Complete stable remission was less frequent in snMG patients (15.9% vs. 27.8%, p < 0.001), and they reported higher disease severity (52.8% medium, 9.5% severe vs. 41.9% medium, 8.5% severe, p = 0.005). snMG patients had higher MG-ADL scores [median 5 (IQR 2/9) vs. 3 (1/6), p < 0.001] and more employment restrictions (64.4% vs. 49.3%, p < 0.001). Furthermore, compared to healthy controls, snMG patients showed worse outcomes in all domains of the SF-36.

The burden of disease in snMG patients is higher compared to AChR-ab+ MG due to delay in diagnosis, worse treatment response, and sociodemographic factors. These findings highlight the challenges patients and treating physicians face in snMG. There is a high need for earlier diagnosis, improved diagnostic tools, and inclusion of snMG patients in clinical trials to address their unique therapeutic challenges.

clinicaltrials.gov, identifier NCT03979521. Registered 7 June 2019 (retrospectively registered).

Autoimmune generalized myasthenia gravis is a disease that manifests with fluctuating muscle weakness. Inebilizumab is a monoclonal antibody that depletes CD19+ B cells, which are central to disease pathogenesis.

In this phase 3, double-blind, randomized, placebo-controlled trial, we enrolled participants with myasthenia gravis who had anti-acetylcholine receptor antibodies or anti-muscle-specific kinase antibodies. Participants were randomly assigned, in a 1:1 ratio, to receive intravenous inebilizumab (300 mg administered on days 1 and 15 for all, and additionally on day 183 for participants who were acetylcholine receptor antibody-positive) or matching placebo for 52 weeks (in participants who were acetylcholine receptor antibody-positive) or 26 weeks (in those who were muscle-specific kinase antibody-positive). Glucocorticoid therapy was tapered, starting at week 4, to a target of 5 mg per day by week 24. The primary end point was the change from baseline in the score on the Myasthenia Gravis Activities of Daily Living scale (MG-ADL; scores range from 0 to 24, with higher scores indicating greater disease activity) at week 26 in the combined acetylcholine receptor antibody-positive and muscle-specific kinase antibody-positive trial populations. A key secondary end point was the change from baseline in the score on the Quantitative Myasthenia Gravis scale (QMG; scores range from 0 to 39, with higher scores indicating greater disease activity) at week 26 in the combined population. Safety was assessed.

A total of 238 participants underwent randomization (119 per group). Participants who received inebilizumab had a greater reduction in the MG-ADL score than those who received placebo (least-squares mean change, -4.2 vs. -2.2; adjusted difference, -1.9; 95% confidence interval [CI], -2.9 to -1.0; P<0.001) at week 26. Participants who received inebilizumab had a greater reduction in the QMG score than those who received placebo (least-squares mean change, -4.8 vs. -2.3; adjusted difference, -2.5; 95% CI, -3.8 to -1.2; P<0.001). The most common adverse events with inebilizumab were headache, cough, nasopharyngitis, infusion-related reactions, and urinary tract infections. Inebilizumab was not associated with a higher incidence of serious adverse events.

In participants with acetylcholine receptor antibody-positive or muscle-specific kinase antibody-positive generalized myasthenia gravis, inebilizumab improved function and reduced disease severity. (Funded by Amgen; MINT ClinicalTrial.gov number, NCT04524273.).

The generic and easy-to-use questionnaire EQ-5D-5L measures health status in five dimensions and is translated into one utility score, EQ-Utility. The study examined if the EQ-5D-5L could be a useful tool for identifying difficult-to-treat patients with myasthenia gravis who needed new therapies. The patients were informed about the study by phone and invited for a test visit. Close to the test visit, the patients completed online questionnaires. Four hundred twenty-five patients are registered with an MG diagnosis in our clinic. Of these, 80 could not be contacted, 39 were excluded, and 85 did not want to participate. Two hundred twenty-one patients participated in the tests, and of these, 178 completed the EQ-5D-5L and were included. The mean EQ-Utility (SD) was 0.85 ± 0.19. Women reported lower EQ-Utility than men (0.79 versus 0.92, p < .0001). The health-related quality of life decreased with increasing disease severity (p < .0001). The symptoms that impacted the EQ-Utility were swallowing (β = 0.065, p = .007), brushing hair/teeth (β = 0.089, p < .0001), rising from a chair (β = 0.069, p < .001), and ptosis (β = 0.045, p = .038). Factors influencing the EQ-Utility were MG-ADL score and depression. The EQ-5D-5L could detect patients with unmet needs and can be used routinely in the clinic.

The adoption of telemedicine is generally considered as advantageous for patients and physicians, but there is limited rigorous assessment of examination strengths and limitations. We set out to perform a quantitative assessment of the limitations of a standardized examination of subjects with myasthenia gravis (MG) during video-taped telemedicine sessions.

We utilized a video bank containing recordings from 51 MG patients who completed two telemedicine-based examinations with neuromuscular experts; each recording included the MG core examination (MG-CE) and the MG activities of daily living (MG-ADL). We then applied artificial intelligence (AI) algorithms from computer vision and speech analysis to natural language processing to generate and assess the reproducibility and inter-rater reliability of the MG-CE and MG-ADL.

We successfully developed a technology to assess video examinations. While overall MG-CE scores were consistent across examiners, individual metrics showed significant variability, with up to a 25% variation in scoring within the MG-CE's range. Additionally, there was wide variability in adherence to MG-ADL instructions. These variations were attributed to differences in examiner instructions, video recording limitations, and patient disease severity.

We were able to develop a system of digital analysis of neuromuscular examinations in order to assess variability in individual scoring measures of the MG-ADL and MG-CE. Our approach enabled post hoc quantitative analysis of neuromuscular examinations. Further refinement of this technology could enhance examiner training and reduce variability in clinical trial outcome measures.

Limited data are available on the relationship between myasthenia gravis (MG) severity and MG exacerbations and healthcare resource utilization (HCRU) following exacerbations. The objective of this study was to assess patient characteristics, exacerbation risk in relation to the MG Activities of Daily Living (MG-ADL) score, and HCRU following exacerbation.

This was a retrospective, cross-sectional, observational study of the patient-reported Myasthenia Gravis Foundation of America Global MG Patient Registry (MGFAPR). Participants were based in the USA, aged ≥ 18 years, had a self-reported MG diagnosis and complete MG-ADL data, and were enrolled between July 1, 2013 and September 30, 2022. Patient demographics, disease characteristics, and HCRU were stratified by MG-ADL score. Negative binomial regression was used to assess the association between MG-ADL score and exacerbation. HCRU for those who had one exacerbation was calculated.

In total, 3416 patients (2092 [61.2%] females) were eligible; mean (standard deviation) age at diagnosis was 49.4 (17.4) years. Compared with patients in the groups with lower MG-ADL scores (≤ 7), more patients in the higher MG-ADL groups (> 7) were female, younger at the time of MG diagnosis, Black, unemployed, uninsured, had a greater comorbidity burden, and had a shorter disease duration. A positive association between the number of exacerbations and MG-ADL score was observed at enrollment. For each additional point on the MG-ADL score, the rate of exacerbations increased by 13% (incidence rate ratio: 1.13; 95% confidence interval: 1.11-1.15; p < 0.001). At enrollment, 49.6% (n = 386/778) of patients who had one exacerbation had HCRU.

We found socio-demographic disparities in disease severity, a higher comorbidity burden, and an increased MG exacerbation risk with higher MG-ADL scores, with a significant impact of MG exacerbation on HCRU. These results highlight the relationship of MG-ADL score to clinical outcomes and the need for treatment optimization and personalized approaches to MG management, especially in socio-demographic groups with an increased risk of exacerbations.

Ravulizumab, an anti-complement C5 monoclonal antibody, was efficacious with acceptable safety in the randomized controlled period (RCP) and interim open-label extension (OLE) periods of the CHAMPION MG phase 3 trial in adults with anti-acetylcholine receptor antibody-positive (AChR-Ab+) generalized myasthenia gravis (gMG). Here, we report final results from the OLE.

Patients who completed the 26-week RCP could enter the OLE and receive ravulizumab for up to 4 years. Efficacy and safety were assessed throughout the OLE.

Among all ravulizumab-treated patients (n = 169; median [range] ravulizumab treatment, 759.0 [14.0, 1265.0] days), 161 entered the OLE (ravulizumab-ravulizumab: n = 78; placebo-ravulizumab: n = 83). Sustained improvements were observed in Myasthenia Gravis Activities of Daily Living (MG-ADL) total scores (ravulizumab-ravulizumab, least squares mean [95% CI] change from RCP baseline at week 164: -4.0 [-5.3, -2.8]; p < 0.0001; placebo-ravulizumab, change from OLE baseline after 138 weeks of treatment: -2.1 [-3.3, -0.9]; p = 0.0005). One hundred and forty-one out of 160 (88.1%) patients achieved a ≥ 2-point improvement in MG-ADL total score, and 59/141 (41.8%) achieved a score of 0 or 1; once achieved, 32/59 (54.2%) sustained this status for > 50% of their remaining time in the study. Similar improvements were observed in Quantitative Myasthenia Gravis and Myasthenia Gravis Quality of Life-15 revised scores, and Neurological Quality of Life Fatigue subscale scores. Clinical deterioration event rates were reduced in the OLE versus placebo in the RCP. Corticosteroid usage was reduced in the OLE. Ravulizumab was well tolerated; no meningococcal infections were reported.

Ravulizumab demonstrated clinically meaningful and durable efficacy and safety in adults with AChR-Ab+ gMG.

Efgartigimod, a first-in-class neonatal Fc receptor antagonist, is approved for generalized myasthenia gravis (gMG). Its safety and efficacy across MG subtypes remain unclear.

This single-center real-world study (September 2023-July 2024) analyzed patients from an MG registry study in China. The primary efficacy outcome is the mean MG-ADL score changes from baseline at weeks 4, 8, and 12, analyzed via generalized estimating equations. Safety was assessed by adverse events.

Among 77 patients (mean age 56.1 ± 15.2 years; 59.7% male), 76 completed at least one treatment cycle (20 completed 2 cycles; 1 completed 3 cycles). After efgartigimod treatment, MG-ADL scores decreased significantly by week 4 (mean difference -6.4, 95% CI -7.2 to -5.6, p < 0.001), sustaining through week 12 (-6.9, -7.8 to -6.1, p < 0.001). After the second cycle, MG-ADL scores at week 12 trended lower than the first cycle (mean difference: -0.8, 95% CI: -2.0 to -0.5, p = 0.061). Efficacy was consistent across MGFA classes and thymoma status. In refractory patients, efgartigimod reduced MG-ADL scores (p < 0.001). Adverse events occurred in 3.9% (3/77).

Efgartigimod safely improved MG-ADL scores and reduced steroid use across MG subtypes, with sustained efficacy through multiple treatment cycles. These findings support its potential when conventional therapies fail.

Myasthenia gravis (MG) is a neuromuscular junction disorder clinically characterized by fluctuating muscle weakness, in which some patients with respiratory muscle weakness are at risk of progressing to myasthenia gravis crisis and respiratory failure, requiring treatment with rapid antibody clearance. Currently widely used intravenous immunoglobulin and plasma exchange therapy remains ineffective in some patients and is limited by multiple contraindications. Efgartigimod is a newly approved FcRn antagonist for the treatment of myasthenia gravis, which rapidly cleans IgG antibodies in the body, but there is still a lack of guidance on the use of efgartigimod in patients with renal insufficiency. Here, we report a case of MG patient with end-stage renal disease undergoing maintenance hemodialysis who successfully navigated a myasthenic crisis and achieved significant clinical remission through efgartigimod therapy. Moreover, sustained efgartigimod maintenance therapy enabled achievement of clinical minimum state. This case demonstrates the therapeutic potential of efgartigimod in MG patients with concomitant renal impairment and provides clinical evidence supporting its application in this special population.

The neonatal Fc receptor (FcRn) inhibitor Efgartigimod (EFG) has been approved for treatment of generalized Myasthenia Gravis (gMG) with anti-AChR antibodies. Information on the effect of EFG in non-AChR MG is limited. We investigated the efficacy of EFG in non-AChR gMG along a clinical follow-up of 2 years.

We treated 13 patients with gMG without anti-AChR antibodies: 5 MuSK+, 2 LRP4 + and 6 triple-negative (confirmed by live CBA). EFG was administered according to the GENERATIVE protocol (consisting of a Fixed period of 2 treatment cycles of 4 infusions at weekly intervals, followed by a Flexible period during which EFG was given in case of initial worsening) starting from November 2021. Outcomes were evaluated by means of the MG-ADL, QMG, MGC and MGQoL15r scales.

The mean follow-up was 21 ± 5.3 months. Meaningful improvement was observed with the clinical scores adopted. The number of cycles/year was 3.92 ± 0.9. The interval between cycles was 10.1 ± 3.6 weeks. MG-ADL improvement of at least 5 points was recorded in 58% of cycles. 46% of patients required hospitalization during the two years before treament with EFG and 70% plasmaexchange/IVIG; during EFG none of the patients was hospitalized or required immunomodulation. No major side effects or infusion related reactions occurred.

EFG was effective in non-AChR gMG and modified significantly the course of the disease. Our experience strengthens the role of FcRn inhibition as a new therapeutic tool for MG without anti-AChR antibodies.

Myasthenia gravis (MG) is an autoimmune disorder affecting neuromuscular junctions, leading to fluctuating muscle weakness. While many patients respond well to standard immunosuppression, a substantial subgroup faces ongoing disease activity. Emerging treatments such as complement factor C5 inhibition (C5IT) and neonatal Fc receptor (FcRn) antagonism hold promise for these patients. However, the current landscape is hindered by a paucity of comparative data that is crucial for treatment decisions.

This study aims to compare the effectiveness and safety of C5IT and FcRn antagonists in a real-world setting.

A retrospective analysis of 153 MG patients from 8 German specialised MG centres receiving either C5IT (26 eculizumab, 80 ravulizumab) or efgartigimod (47 patients) was conducted. Propensity score matching (PSM) was employed to compare changes in MG-specific outcome parameters within the first 6 months after treatment initiation, along with safety profiles and concomitant MG therapy.

Both treatment strategies led to rapid clinical improvements and substantial reductions in prednisolone doses. However, insufficient response was noted in 20%-49.1% of patients based on Quantitative MG and MG Activities of Daily Living (MG-ADL) scores. We did not identify any new safety concerns. After PSM, 40 patients remained in each group. In both cohorts, reductions in MG-ADL as prespecified primary study endpoint were comparable. Moreover, analyses of secondary outcome parameters demonstrated similar results for C5IT versus FcRn.

In contrast to current meta-analyses and indirect comparisons of clinical trial data, our real-world study demonstrates comparable efficacy and safety of C5IT and FcRn antagonism in MG.

Patients with myasthenia gravis (MG) often experience impairment of respiratory function. Some case reports have explored the diaphragmatic compound muscle action potential (DCMAP) amplitudes in patients with MG using single-stimulus phrenic nerve conduction studies. Herein, we aimed to investigate whether the DCMAP amplitude correlated with the clinical severity of MG and whether this improved with treatment.

We retrospectively evaluated patients with MG who underwent right phrenic nerve conduction studies. We evaluated the patients' clinical characteristics and the clinical severity of MG, as assessed by compound muscle action potential (CMAP) amplitudes, and compared the CMAP amplitudes before and after treatment in patients with generalized MG (gMG).

This study included 76 patients with MG (30 with ocular MG [OMG] and 46 with gMG). The median DCMAP amplitudes were significantly higher in OMG than in gMG (364 vs. 212 μV, p < 0.01). Receiver operating characteristic analysis showed that DCMAP amplitude < 256 μV distinguished gMG from OMG with 71.7% sensitivity and 93.3% specificity. The DCMAP amplitudes were moderately correlated with the MG activities of daily living score (p < 0.01, r = -0.52) and quantitative myasthenia gravis score (p < 0.01, r = -0.60). Median DCMAP amplitudes significantly increased in patients with gMG after treatment (from 184 to 336 μV, p < 0.01).

DCMAP amplitudes may correlate with the clinical severity of MG, differentiate gMG from OMG, and reflect the treatment response. We propose that DCMAP measurement be included in the evaluation of patients with MG.

We herein report two patients with anti-muscle-specific kinase (MuSK) antibody-positive myasthenia gravis who experienced rapid deterioration of weakness, particularly respiratory muscle weakness, necessitating non-invasive positive pressure ventilation (NIPPV) and were treated with efgartigimod. After treatment initiation, a rapid reduction in IgG levels and recovery from clinical symptoms were observed. NIPPV was no longer required two to three weeks after the first infusion of efgartigimod. These findings suggest that the reduction of IgG levels using efgartigimod is a good treatment option in patients with myasthenia gravis positive for anti-MuSK antibodies, even during the acute phase of the disease.

Myasthenia gravis (MG) is rare, chronic autoimmune disorder of the neuromuscular junction that requires specialized care and often lifelong treatment, facing challenges due to its rarity and the limited availability of specialists. Telemedical solutions in specialized centers hold considerable promise in bridging this gap by increasing access to this care to a broader patient population in a timely manner. However, there is no research regarding interventional remote care solutions in the field of MG to date.

This study aimed to assess the feasibility and usability among patients with MG and specialists of a telemedicine platform, tailored to patients with MG and designed to facilitate remote monitoring, treated in a specialized center.

The telemedicine platform consisted of an app for patients and a web-based portal for physicians. Over a period of 3 months, 30 patients continuously monitored their vital parameters through external devices, including a digital spirometer and a wearable (activity tracker). Furthermore, patients completed 7 different patient-reported outcome measures (PROMs) through the app at predefined intervals. Specialists could review this monitoring data and adjust therapy as necessary. In addition, communication between patients and physicians was facilitated through a chat module. Feasibility was evaluated by total adherence rates for completing PROMs within the app, alongside the collection of spirometry and wearable data. Furthermore, user satisfaction was assessed among both patients with MG and physicians at the end of study.

Total adherence rates ranged from 74.3% (1830/2464) to 97.9% (327/334) across all data types, with the highest adherence observed for PROMs (1139/1179, 96.6%), followed by spirometry (293/334, 87.7%) and wearables (1830/2261, 80.9%). Notably, patients wore the wearable longer than required by protocol and conducted a higher number of spirometry measurements during the study than required per protocol (median 20 h/d [IQR 15-24] vs 14 h/d and median 49 [IQR 15-59] measurements vs 11 measurements, respectively). Technical issues and discomfort with wearables were factors affecting lower adherence in some patients. The System Usability Scale yielded a median score of 85 indicating "excellent usability." In addition, results from a more detailed user evaluation questionnaire showed high levels of user satisfaction among both patients and health care professionals across diverse categories, including their experience of the care program, communication, and evaluation of the program.

Remote monitoring of patients with MG through the telemedical platform demonstrated good feasibility and acceptability, as evidenced by above-average adherence rates and user satisfaction for both patients and physicians. The majority of patients wanted to continue using the app. These findings highlight the potential for user-friendly digital tools to enhance care for patients with MG, although addressing technical challenges and ensuring patient comfort with wearables are essential for optimal implementation. Further research involving larger cohorts and longer study duration is warranted to validate these findings.

Myasthenia gravis (MG) is a rare autoimmune disease characterised by exertion-induced muscle weakness that can lead to potentially life-threatening myasthenic crises. Detectable antibodies are directed against specific postsynaptic structures of the neuromuscular junction. MG is a chronic condition that can be improved through therapies, but to date, not cured. Standard treatment has been unchanged for decades and includes symptomatic treatment with acetylcholine-esterase inhibitors and disease-modifying treatment with steroids, steroid-sparing immunosuppressants and thymectomy. Overall, a relevant proportion of patients does not achieve a satisfactory clinical improvement under standard treatment. Additionally, long-term therapy with steroids can cause significant side effects and latency to clinical improvement with standard steroid-sparing immunosuppressants and after thymectomy can take months to years. In recent years, treatment of MG has changed fundamentally due to improved evidence from phase 3 trials and the regulatory approval of complement inhibitors and FcRn inhibitors as add-on treatment options. This provides new optimism for substantially more patients reaching minimal manifestation status and has led to a shift in treatment strategy with more targeted therapies being employed early in the course of the disease, especially in patients with high disease activity. In this focussed review, we provide an overview of the diagnosis, classification and standard treatment of MG, followed by data from randomised controlled trials on the modern drugs already available for therapy and those still in the final stages of clinical development. In the second part, we provide an overview of real-world data for already approved therapies and outline how the availability of new biologicals is changing both clinical decision-making and patient journey.

Myasthenic crisis is a life-threatening exacerbation of myasthenia gravis leading to respiratory failure, while zilucoplan is a C5 complement inhibitor that prevents complement-mediated destruction of the neuromuscular junction, thereby helping to restore muscle function and respiration. This case report describes an 85-year-old woman with late-onset myasthenia gravis who developed myasthenic crisis triggered by aspiration while eating. Initial treatment with Intravenous immunoglobulin and intravenous methylprednisolone showed limited efficacy, making weaning from mechanical ventilation challenging. Due to her advanced age, diabetes, and heart failure, plasmapheresis was considered high-risk. Zilucoplan, a C5 inhibitor administered subcutaneously, was introduced as an adjunctive therapy on Day 11, leading to rapid 50% hemolytic complement activity level reduction and significant muscle strength improvement. Extubation was achieved within four days, avoiding tracheostomy. Add-on therapy of zilucoplan provided a safe and accessible treatment option for myasthenic crisis. Previous reports have demonstrated the benefits of other C5 inhibitors and neonatal Fc receptor inhibitors in refractory myasthenic crisis. This case supports the early addition of zilucoplan with conventional rescue therapies for myasthenic crisis may have enabled earlier extubation and the avoidance of tracheostomy. Further studies are needed to determine the universal applicability of add-on therapies in myasthenic crisis, considering comorbidities and complications.

Myasthenia gravis (MG) is a neuromuscular disorder resulting in exertion-dependent muscle fatigability. Affecting all age groups MG is a chronic disease with unpredictable fluctuations and a range of symptom expression which can require costly immunosuppressive therapy and intensive care critically influencing economic burden. Here we aim to elucidate both the personal and societal economic burden of MG. Data were used from two sources: (1) a cross sectional study of MG patients registered in the German Myasthenia Society (DMG) collected by a survey in 2019 (n = 1660), and (2) from a dataset provided by a German health insurance company covering the period from 01/01/2014 to 31/12/2019 (n = 750) and categorized to similar subgroups for comparison. Individual and societal economic burden was highest in patients with intensified medical treatment. Absence days from work per patient year in the first year after index in claims data were 67.1 days (95% CI 66.1-68.2) (referring to n = 260 non-retired patients). Of 686 patients with early onset (< 50 years) MG 260 (41.2%) reported loss of income due to MG in DMG data. Average inpatient costs from claims data in patients with intensified treatment (38,669€; 95% CI 38,627-38,712€) were particularly high compared with standard treatment (2980€; 95% CI 2975-2986€) and to no MG-related treatment (887€; 95% CI 877-886€). MG represents a major economic burden for the individual and the health care system. The costs are positively associated with the severity of disease. The economic burden is particularly high for working patients. UCB Pharma provided financial support for the subanalysis, but was not involved in the design of the study.Trial registration: clinicaltrials.gov, NCT03979521, submitted: June 7, 2019, first patient enrolled: May 1, 2019, https://clinicaltrials.gov/ct2/show/NCT03979521 .

Efgartigimod is an approved biologic for generalized myasthenia gravis (gMG), which is an autoimmune disease and can potentially be life-threatening. However, the therapeutic response to efgartigimod among the acetylcholine receptor gMG (AChR-gMG) subtypes remains inconclusive.

To explore the patterns and predictors for the therapeutic response to efgartigimod among AChR-gMG subtypes.

This prospective, observational study included AChR-gMG patients treated with efgartigimod at 15 centers in China with a follow-up for at least 20 weeks.

The primary outcome was the proportion of minimal symptom expression (MSE) responders, denoted by a Myasthenia Gravis Activities of Daily Living (MG-ADL) score of 0 or 1 within 4 weeks and maintained for ⩾4 weeks. AChR antibody-positive MG (AChR-MG) subtypes were classified into early onset myasthenia gravis (EOMG), late-onset myasthenia gravis (LOMG), and thymoma-associated myasthenia gravis (TAMG). The predictive factors for MSE responders were identified by univariate and multivariate logistic regression analysis.

One hundred sixteen patients were included with a median follow-up duration of 238 days (172.5-306.3). There were 50 (43.1%) patients with EOMG, 28 (24.1%) with LOMG and 38 (32.8%) with TAMG. After efgartigimod initiation, 35 (30.2%) patients were MSE responders, and the proportion of MSE responders was highest in the LOMG group (42.9%). The MG-ADL score reduction in the LOMG group was more significant than in the EOMG group by weeks 16 and 20 (both p = 0.022). Response patterns to efgartigimod among the AChR-MG subtypes differed as measured by the proportion of improved patients and MSE. LOMG presented sustained symptom control, while EOMG and TAMG showed more fluctuations. Eight TAMG patients (21.1%) switched to another biologic (p = 0.005). Baseline MG-ADL was an independent predictor for therapeutic response to efgartigimod (p < 0.001).

Our findings revealed patterns of treatment responses among AChR-gMG subtypes, with LOMG patients potentially presenting a more sustained response. These findings likely provide preliminary data for precision therapy in MG in the era of biologics.

NCT04535843.

Inflammatory and immune mechanisms are considered in restless legs syndrome (RLS) pathophysiology with several autoimmune diseases associated with RLS. There is a paucity of studies examining RLS prevalence in myasthenia gravis (MG), an autoimmune neuromuscular disease. This study investigated RLS prevalence and association with patient-reported measures in a large registry of participants with MG using a validated RLS diagnostic questionnaire.

The Myasthenia Gravis Foundation of America MG Patient Registry is used on a semiannual basis to survey participants with MG. Patients aged ≥ 18 years, living in the United States, and answering "yes" to physician diagnosed MG were invited by email to enroll in an RLS-customized web-based survey. Collection of data included demographics, disease variables, patient-reported measures with a simple depression scale, MG-quality of life-15 revised, MG-activities of daily living instruments, and 13-item short-form Cambridge-Hopkins diagnostic questionnaire for RLS. Multivariable logistic regression models explored the association between RLS and MG variables of interest.

A total of 630 eligible participants with MG (age: 62.8 ± 13.2; 54.9% female; 91.6% White) completed the survey. The overall prevalence of RLS was 14.8%. The prevalence of clinically significant RLS was 8.4%. The odds of having RLS were increased with higher (worse) MG-activities of daily living, MG-quality of life-15 revised, and depression scores. History of "thymic tumor with thymectomy" and "continuous positive airway pressure therapy" were also independent predictors of RLS.

RLS is common in patients with MG and is associated with worse functional status, quality of life, and depression. The thymus could play a key role in an autoimmune process associating MG with RLS.

AlGaeed M, McPherson T, Lee I, et al. Prevalence of restless legs and association with patient-reported outcome measures in myasthenia gravis. J Clin Sleep Med. 2025;21(2):269-276.

China has a large number of myasthenia gravis (MG) patients, creating an urgent need for rapid and tolerable treatment options. As the first-approved Fc receptor antagonist, efgartigimod has bright prospects for treating MG. However, real-world evidence on its application within the Chinese MG population are limited.

This study aims to evaluate the rapid efficacy and safety of efgartigimod in Chinese MG population.

This single-center prospective study enrolled Chinese MG patients aged 18 and older who were treated with efgartigimod, classified as Myasthenia Gravis Foundation of America I-IV, with a baseline Myasthenia Gravis Activities of Daily Living (MG-ADL) score of at least 4.

Patients received efgartigimod at a dose of 10 mg/kg infused once weekly for 4 weeks. During the treatment, the corticosteroids dosage could be adjusted as appropriate or the non-steroidal immunosuppressive therapies (NSISTs) added. Prior to each infusion, patients' MG-ADL scores, IgG levels, and routine laboratory tests were evaluated, while also recording the prednisone tapering and any adverse events occurring during the treatment.

Twenty five Chinese MG patients were enrolled between November 2023 and June 2024, including 3 with ocular MG (OMG) and 22 with generalized MG (GMG). During the 8-week follow-up, in GMG patients, whether positive for acetylcholine receptor (AChR) or muscle-specific tyrosine kinase (MuSK) antibodies, the overall efficacy was significant. Within one treatment cycle, 18 (82%) patients showed a reduction of at least 2 points in MG-ADL scores and sustained for at least 4 weeks, and 6 (27%) attained minimal symptom expression (MSE) and sustained for at least 4 weeks. Only 1 patient experienced exacerbation. Among OMG patients, 1 achieved MSE within the treatment cycle, while 2 showed minor improvements. Patients who added tacrolimus concurrently with efgartigimod did not achieve better improvement in MG-ADL scores compared to others. The average reduction in prednisone dosage was 27.4%. Only one patient experienced transient vomiting and diarrhea, with no serious adverse reactions reported.

This study confirmed the short-term efficacy and safety of efgartigimod in Chinese MG patients. However, in clinical practice, careful consideration is needed regarding its application in OMG and whether to add NSISTs regimen during the treatment. Efgartigimod could potentially serve as an alternative to long-term corticosteroids therapy.

Myasthenia Gravis (MG) is a common neuromuscular junction disorder that is primarily mediated by anti-acetylcholine receptor antibodies (AChR-Ab). However, using AChR-Ab titers to predict MG severity and improvement remains controversial. This study aims to explore the relationship between AChR-Ab titers and AChR-Ab rate of change (RR-AChR-Ab, %) and MG scores.

We used a prospective study approach, and included 62 patients with generalized MG (GMG) who were positive for AChR-Ab. We measured AChR-Ab titers, MGFA-QMGS, and MG-ADL scores at baseline (before treatment) and at 3 and 6 months into treatment. Pearson and Spearman correlation analyses were used to study the relationships between changes in AChR-Ab titers, rates of change, and MG scores.

(1) At baseline, there was no correlation between AChR-Ab titers and age, duration of illness, gender, MGFA classification, or presence of thymic abnormalities. (2) The trend of decreasing AChR-Ab titers matched the trend of reduced QMGS and ADL scores. (3) Six months into treatment,there was a correlation between AChR-Ab titer changes and changes in ADL scores. (4) Three months into treatment, RR-AChRAb showed a correlation with the rate of change in ADL at the same time point.

We found the trend of decreased AChR-Ab titers after standardized treatment that was consistent with reductions in QMGS and ADL scores. Additionally, the rate of change in AChR-Ab titers at 3 months and the change in AChR-Ab titers at 6 months into treatment did reflect improvements in activities of daily living for MG patients.

Generalized myasthenia gravis (gMG) is characterized by fluctuating muscle weakness. Exacerbation frequency, adverse events (AEs) related to immunosuppressant therapy and healthcare resource utilization (HCRU) are not well understood. Our study aimed to describe long-term clinical outcomes, drug-related AEs and estimated HCRU in gMG patients.

This was a retrospective cohort analysis of clinical data from patients with gMG followed-up over eight consecutive years in a Spanish referral unit. Myasthenia Gravis Foundation of America (MGFA) clinical classification, MGFA post-interventional status (MGFA-PIS), Myasthenia Gravis Activities of Daily Living (MG-ADL) score, exacerbations, MG crises, therapies, AEs reported, specialist consultations and emergency room visits were studied biannually. An estimation of HRCU was made based on these data.

Some 220 patients newly diagnosed with gMG were included. Ninety percent were seropositive (84.5% anti-acetylcholine receptor [AChR], 5.9% anti-muscle-specific kinase [MuSK]). Baseline mean MG-ADL score was 5.04 points (SD 3.17), improving to 0.7 points (SD 1.40) after 8 years. Exacerbations were more frequent in years 1-2 (30.1%) but still occurred in years 7-8 (20.2%). Myasthenic crisis frequency remained 1% in years 7-8. Eighty-nine percent achieved MGFA-PIS minimal manifestations or better at 8 years. Fifty-one percent of patients reported at least one AE during the study period, leading to drug withdrawal in approximately 20% of cases. HCRU decreased between years 1-2 to years 7-8 with an estimated cost of MG from 8074.19 € per patient/year to 1679.46 €, respectively.

There is a group of MG patients that suffers from persistent symptoms and exacerbations (11%-20%) or MG crises, and drug AEs, which may increase disease burden and impact on the healthcare system.

Fluctuating symptoms and fatigue are common issues in myasthenia gravis (MG), but it is unclear if these symptoms are related to physical activity or sleep patterns. This study sought to determine the day-to-day relationship between patient-reported symptoms and physical activity and sleep over 12 weeks.

Sixteen participants with generalized MG wore a wrist-mounted accelerometer continuously for the study duration and reported their symptoms and fatigue each evening. Cumulative link mixed models were used to analyze whether clinical and demographic characteristics, physical activity, and sleep were related to symptom severity and fatigue over the study period. Three types of models were constructed: a cohort model, a model in which data was scaled to each participant, and individual models.

The cohort model indicated that higher disease severity, female sex, more comorbidities, less physical activity, more inactive time, and lower quantity of sleep were significantly associated with increased symptom severity and fatigue (p < .05). However, in the within-participant scaled model, there were almost no significant associations with physical activity or sleep. In the individual models, some participants showed similar results to the cohort model, but others showed no associations or the opposite response in some variables.

While physical activity and sleep were associated with self-reported symptoms and fatigue within this population, this was not necessarily applicable to individuals. This demonstrates the importance of an individualized analysis for determining how physical activity and sleep may impact outcomes in MG, with implications for clinical and self-management.

Biologics that target pathogenic antibodies (Abs) and their effector functions such as the complement inhibitor ravulizumab and the neonatal Fc receptor agonist efgartigimod have recently been approved for the treatment of acetylcholine receptor (AChR)-Ab-positive myasthenia gravis (MG), but comparative studies are lacking.

In a prospective, exploratory real-world study, we assessed clinical efficacy, safety, and biological effects of ravulizumab and efgartigimod treatment initiation. Myasthenia Gravis-Activities of Daily Living and Quantitative Myasthenia Gravis scores were used as clinical endpoints. Ab effector functions were determined by AChR-Ab-dependent complement activation and phagocytosis assays and systemic complement activation profiling.

We observed similar moderate short-term efficacy of ravulizumab and efgartigimod in achieving clinical improvement. Ravulizumab reduced systemic terminal complement activation, but neither treatment showed significant effects on complement pathways proximal to C5 or functional capacities of AChR-Abs. Both treatment modalities were well tolerated with no serious adverse events reported.

Clinical benefits obtained with ravulizumab and efgartigimod can be remarkably heterogeneous in daily clinical practice. Neither treatment relevantly changed effector functions of pathogenic AChR-Abs, supporting the concept that durable disease control in MG requires continuous administration of both fast-acting agents.

This study provides Class III evidence that in AChR-Ab-positive patients with generalized MG, ravulizumab and efgartigimod provide comparable modest improvement in MG functional scales.

Myasthenia gravis (MG) is characterized by fluctuating muscle weakness due to immune-mediated damage to acetylcholine receptors. Viral infections can exacerbate symptoms of muscle weakness, and the clinical status of patients with MG may influence the outcomes of such infections. Here, we identified factors of symptom exacerbation, severe SARS-CoV-2 infection, and pneumonia in patients with MG who are infected with SARS-CoV-2.

The clinical characteristics and outcomes of 341 MG patients infected with SARS-CoV-2 across multiple regions in China were determined.

The median age of the patients was 49 years (range: 35-60 years) and the median disease duration was 4 years (range: 2-8 years). Among the patients, 67 (49.0%) were male and 174 (51.0%) were female. Multivariate analysis indicated that thymectomy [OR, 1.654 (95% CI, 1.036-2.643); p = 0.035], severe SARS-CoV-2 infection [OR, 4.275 (95% CI, 2.206-8.286); p < 0.001], and pyridostigmine bromide [OR, 1.955 (95% CI, 1.192-3.206); p = 0.008] were associated with exacerbation of MG symptoms in patients infected with SARS-CoV-2. Age was significantly associated with severe SARS-CoV-2 infection [OR, 1.023 (95% CI, 1.001-1.046); p = 0.008], while patients with cardiac/vascular comorbidities exhibited an increased likelihood of severe SARS-CoV-2 infection [OR, 3.276 (95% CI, 1.027-10.449); p = 0.045]. Likewise, steroid treatment [OR, 6.140 (95% CI, 2.335-16.140); p < 0.001] was associated with a significantly increased likelihood of severe SARS-CoV-2 infection compared with symptomatic treatment. Additionally, gender [OR, 0.323 (95% CI, 0.120-0.868); p = 0.025] and SARS-CoV-2 severity [OR, 6.067 (95% CI, 1.953-18.850); p = 0.002] were associated with the occurrence of pneumonia.

We identified factors that were associated with the exacerbation of MG symptoms in patients infected with SARS-CoV-2, including thymectomy, severe SARS-CoV-2 infection, and the use of pyridostigmine bromide. Due to the retrospective nature of the study, these findings should be interpreted as associations rather than predictive factors. However, the results confirm the established relationships between severe SARS-CoV-2 infection and age, cardiovascular comorbidities, and the use of steroid treatment, suggesting that these factors should be considered when managing MG patients during SARS-CoV-2 infection.

Myasthenia gravis (MG) is a rare, chronic neurological disorder leading to fluctuating muscle weakness and potentially life-threatening crises. Patients often require life-long specialized treatment, but timely interventions are frequently hindered by the limited availability of specialists. Telemedical solutions at specialized centers enabling patient-physician interaction hold promise in bridging this gap, but are not yet available for MG. We developed 'MyaLink,' a remote monitoring platform tailored for MG, and outline the study design assessing the platform and clinical outcomes regarding telemedical intervention. Additionally, we present study results on care-related aspects in MG prior to telemedical intervention to identify challenges in the current care provision process.

The platform comprises a patient app and a physician portal, enabling systematic symptom monitoring using data from patient-reported outcome measures (PROMs), coupled devices and a communication module. The randomized controlled study included 45 study participants (SP) over a 12-weeks period, including a group receiving standard care (15 MG patients) and a group with additional telemedical treatment (30 MG patients) including assessment of PROMs, wearable data collection and telemedical check-ups. Questions regarding care-related aspects were assessed at baseline visit.

Many SP (N = 33, 73.3%) communicate with the physician managing their MG via email. 73.3% (N = 33) of SP identify areas for improvement in their MG care including symptom monitoring (N = 23, 69.7%), specialist appointment availability (N = 22, 66.7%), medication (N = 22, 66.7%) and specialist accessibility (N = 20, 60.6%). Additionally, 73.3% (N = 33) reported that the effort required to manage their MG was high.

Our results emphasize the high demand of affected MG patients for continuous telemedicine services. MyaLink can provide such a service through personalized support based on the exchange of health data. Telemedicine solutions such as MyaLink promise to improve myasthenia care by providing accessible, patient-centred care that enables early detection of worsening symptoms and non-response to treatment.

The study was registered under DRKS00029907 on August 19, 2022.

To capture patient perceptions about living with myasthenia gravis (MG) with respect to aspirations and ways to improve treatment.

Online patient survey.

Patients recruited by MG patient associations or at MG reference treatment centres.

Adults with physician-diagnosed generalised MG, living in France for ≥12 months, who had received ≥1 MG treatment were eligible. 291 patients opened the questionnaire, 255 were eligible and 246 completed the survey (age range 41-67 years; 187 women and 59 men).

Primary: free-text response to the question 'Living with your disease, what would you like to do that you currently find difficult or impossible to do?' Secondary: free-text response to the question 'What improvements do you think could be made to treatments for MG?' Themes from replies to these questions were analysed using grounded theory and cluster analysis.

For the disease aspirations question, 617 citations were provided by 238 participants, which were grouped into 45 dimensions and six high-level domains (physical activity, activities of daily living, psychological burden, social activities, work/school and other). The most frequently cited dimensions were sport (82 citations), greater mobility (56 citations), being less tired (46 citations) and greater endurance (37 citations). Younger age, female gender, recent diagnosis and poorer quality of life were associated with citing more themes. For the treatment amelioration question, 263 citations were provided by 195 participants, which were grouped into 60 dimensions and three high-level domains (medication characteristics, safety and care paradigm). The most cited treatment-related dimensions were fewer side effects (40 citations), fewer daily medication intakes (21 citations) and fewer digestive side effects (20 citations).

These findings could help healthcare professionals to understand and better address patients' aspirations about living with MG, notably concerning the importance of physical activity, and their expectations for improved treatments.

This study used network meta-analysis (NMA) to inform and compare the number needed to treat (NNT), number needed to harm (NNH), and cost per improved outcome (CPIO) associated with more recently approved treatments for anti-acetylcholine receptor antibody-positive (anti-AChR Ab+) generalized myasthenia gravis (gMG).

Clinical trials of neonatal Fc receptor (FcRn) inhibitors, efgartigimod intravenous (IV) and rozanolixizumab, and complement inhibitors, ravulizumab and zilucoplan, versus placebo (with background conventional treatment) were included in the primary NMA to compare efficacy and safety outcomes. The outputs from the NMAs were used to estimate NNT and NNH of each treatment versus placebo. CPIO (2024 USD) was estimated for a ≥ 3- or ≥ 5-point reduction from baseline in Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores. Sensitivity analyses were performed adding efgartigimod PH20 subcutaneous (SC) and eculizumab to the NMA.

Efgartigimod IV had the lowest NNT versus placebo for achieving a ≥ 3- and ≥ 5-point reduction in QMG, as well as a ≥ 5-point reduction in MG-ADL, whereas rozanolixizumab had the lowest NNT for a ≥ 3-point reduction in MG-ADL. The NNH versus placebo was similar across comparator treatments. Efgartigimod IV had the lowest CPIO among all treatments for all assessed efficacy outcomes. Sensitivity analyses yielded results consistent with primary analysis and indicated that efgartigimod PH20 SC had comparable NNT and CPIO values to efgartigimod IV, whereas eculizumab had comparable NNT and higher CPIO values compared to other complement inhibitors.

FcRn inhibitors and complement inhibitors assessed in this study all demonstrated clinical benefit in terms of NNT as well as an acceptable safety profile in terms of NNH. Within the limitations of this meta-analysis, efgartigimod was associated with a favorable benefit-risk profile as well as a better economic value compared to ravulizumab, rozanolixizumab, and zilucoplan as treatments for anti-AChR Ab+ gMG.

Myasthenia gravis (MG) is a rare, autoimmune, neurological disorder. Most MG patients have autoantibodies against acetylcholine receptors (AChRs). Some have autoantibodies against muscle-specific tyrosine kinase (MuSK) or lipoprotein-receptor-related protein 4 (LRP4), and some are seronegative. Standard of care, which includes anti-cholinesterase drugs, thymectomy, corticosteroids (CS), and off-label use of non-steroidal immunosuppressive drugs (NSISTs), is bounded by potential side effects and limited efficacy in refractory generalized MG (gMG) patients. This highlights the need for new therapeutic approaches for MG. Eculizumab, a monoclonal antibody that inhibits the complement system, has been recently approved in Italy for refractory gMG. A panel of 11 experts met to discuss unmet therapeutic needs in the acute and chronic phases of the disease, as well as the standard of care for refractory patients. Survival was emphasized as an acute phase outcome. In the chronic phase, persistent remission and early recognition of exacerbations to prevent myasthenic crisis and respiratory failure were considered crucial. Refractory patients require treatments with fast onset of action, improved tolerability, and the ability to slow disease progression and increase life expectancy. The Panel agreed that eculizumab would presumably meet the therapeutic needs of many refractory gMG patients. The panel concluded that the unmet needs of current standard of care treatments for gMG are significant. Evaluating new therapeutic options accurately is essential to find the best balance between efficacy and tolerability for each patient. Collecting real-world data on novel molecules in routine clinical practice is necessary to address unmet needs.