Quality Of Life In Myasthenia Gravis Research Articles

Efgartigimod and Ravulizumab for Treating Acetylcholine Receptor Auto-antibody-Positive (AChR-Ab+) Generalized Myasthenia Gravis: Indirect Treatment Comparison.

Efgartigimod and ravulizumab, both approved for treating acetylcholine receptor auto-antibody-positive (AChR-Ab+) generalized myasthenia gravis (gMG), have not been directly compared. This paper assessed comparative effects of efgartigimod vs. ravulizumab for treating adults with AChR-Ab+gMG using indirect treatment comparison methods. The matching-adjusted indirect comparison used data from two randomized trials of adult men and women. The ADAPT (efgartigimod vs. placebo; individual patient data available) population was reweighted to match the CHAMPION (ravulizumab vs. placebo; index study; aggregate data available) population. The relative effect of efgartigimod versus placebo was estimated in this reweighted population and compared with the observed ravulizumab versus placebo effect to estimate the efgartigimod versus ravulizumab effect. The outcomes were Myasthenia Gravis Activities of Daily Living (MG-ADL), QuantitativeMyasthenia Gravis (QMG), and Myasthenia Gravis Quality of Life 15-item-revised scale (MG-QoL15r) assessed as cumulative effect (area under the curve; AUC) over 26 weeks (primary) and change from baseline at 4 weeks and time of best response (week 4 for efgartigimod; week 26 for ravulizumab). For MG-QoL15r, efgartigimod had a statistically significant improvement compared with ravulizumab over 26 weeks [mean difference (95% confidence interval): -52.6 (-103.0, -2.3)], at week 4 [-4.0 (-6.6, -1.4)], and at time of best response [-3.9 (-6.5, -1.3)]. Efgartigimod had a statistically significant improvement over ravulizumab in MG-ADL at week 4 [-1.9 (-3.3, -0.5)] and at time of best response [-1.4 (-2.8, 0.0)] and in QMG at week 4 [-3.2 (-5.2, -1.2)] and at time of best response [-3.0 (-5.0, -1.0)]. For AUC over 26 weeks, improvements were not significantly different between efgartigimod and ravulizumab for MG-ADL [-8.7 (-36.1, 18.8)] and QMG [-13.7 (-50.3, 22.9)]. Efgartigimod may provide a faster and greater improvement over 26 weeks in quality of life than ravulizumab in adults with AChR-Ab+gMG. Efgartigimod showed faster improvements in MG-ADL and QMG than ravulizumab.

Translation and validation of the Myasthenia Gravis Activities of Daily Living Questionnaire: Latvian version.

Our aim was to translate, adapt and validate the Myasthenia Gravis Activities of Daily Living scale into the Latvian language and to evaluate this instrument (MG-ADL-L) in terms of construct validity and reliability. We enrolled patients with a confirmed MG diagnosis, who could speak Latvian fluently. We performed translation and adaptation according to the cross-cultural adaptation guidelines for self-reported measures. The patients were evaluated by a physician according to the Myasthenia Gravis Foundation of America classification (MGFA) and using the Myasthenia Gravis Composite Score (MGCS). Patients were asked to complete the MG-ADL-L and the 15-item Myasthenia Gravis Quality of Life (MGQOL15) Internal consistency was evaluated based on Cronbach's α, reproducibility-Cohen's weighted kappa and construct validity-Spearman's correlation between the MG-ADL-L and the MGQOL15 and MGCS. We used the Kruskal-Wallis H test to compare the MG-ADL-L score distribution between the MGFA groups. 38 enrolled patients in the study. There was an acceptable internal consistency (Cronbach's α = 0.76) and moderate to very good agreement between the test and retest scores (Cohen's weighted kappa = 0.54 and 0.81). The MG-ADL-L showed a moderate positive correlation with the MGQOL15 (r = 0.5, p = 0.001) and the MGCS (r = 0.62, p < 0.001). There was a significant difference in MG-ADL-L scores between the MGFA groups (p = 0.007). The MG-ADL-L is a valid and reliable self-reported scale to assess and evaluate symptom severity and the impact of the disease on the lives of patients with MG.

The correlation between functional capacity and quality of life in patients with myasthenia gravis

Objective. The objective of this study was to analyze the correlation between functional capacity with quality of life in patients diagnosed with myasthenia gravis (MG). Method. This cross-sectional study included 23 patients with MG between July and August 2021 (7 men,16 women; mean age: 46.78 ± 6.24 years; range: 33-56 years). Using a 2-minute walking test (2MWT) for 2-minute walking distance (2MWD), maximum oxygen uptake (VO2Max), and metabolic equivalents (METs), the functional capacity was determined. The quality of life can be evaluated by the Myasthenia Gravis Quality of Life 15 (MG-QL15). Result. Functional capacity was not significantly correlated with quality of life: (2MWD and MG-QoL15 r=-0.002, p=0.992), (VO2Max and MG-QoL15 r=0.190, p=0.386), and (METs and MG-QoL15 r=0.188, p=0.391). Multiple linear regression analysis showed that 2MWD, VO2Max, and METs of functional capacity have no significant effect on MG-QoL15 of quality of life but 2MWD, METs, and VO2Max had a stronger relationship strength with MG-QoL15 domain ranging from VO2Max, MET, and 2MWD. Quality of life and functional capacity of myasthenia gravis patients showed no regression relationship partially of current age, age at onset, length of medication, severity of disease using myasthenia gravis composite (MGC) score, and body mass index (BMI). Conclusion. In MG patients, no significant correlation was observed between functional capacity and quality of life, and further research is required to determine the factors that influence functional capacity and quality of life.

Plasma C18:0-ceramide is a novel potential biomarker for disease severity in myasthenia gravis.

Myasthenia gravis (MG) is an antibody-mediated autoimmune disorder characterized by fluctuation of fatigue and weakness of muscle. Due to the heterogeneity of the course of MG, available biomarkers for prognostic prediction are urgently needed. Ceramide (Cer) was reported to participate in immune regulation and many autoimmune diseases, but its effects on MG remain undefined. This study aimed to investigate the ceramides expression levels in MG patients and their potential as novel biomarkers of disease severity. Levels of plasma ceramides were determined by ultra performance liquid chromatography-tandem mass spectrometry (UPLC-MS/MS). Severity of disease was assessed by quantitative MG scores (QMGs), MG-specific activities of daily living scale (MG-ADLs) and 15-item MG quality of Life (MG-QOL15). The concentrations of serum interleukin-1β (IL-1β), IL-6, IL-17A, and IL-21 were determined by enzyme-linked immunosorbent assay (ELISA), and the proportions of circulating memory B cells and plasmablasts were detected by flow-cytometry assay. Four plasma ceramides levels we studied were detected higher in MG patients. And three of them (C16:0-Cer, C18:0-Cer, and C24:0-Cer) were positively associated with QMGs. In addition, receiver operating characteristic (ROC) analysis suggested that plasma ceramides have a good ability of differentiating MG from HCs. Importantly, only C18:0-Cer was shown to be positively associated with the concentration of serum IL and circulating memory B cells, and the decrease in plasma C18:0-Cer paralleled the clinical improvement of patients with MG. All together, our data suggest that ceramides may play an important role in the immunopathological mechanism of MG, and C18:0-Cer has the potential to be a novel biomarker for disease severity in MG.

Reduced quality of life in myasthenia gravis patients: A study on 185 patients from China.

To explore the quality of life (QOL) in patients with myasthenia gravis (MG) and factors associated with QOL. This observational study included patients with MG diagnosed at the First Affiliated Hospital of Fujian Medical University between January 2020 and March 2022. The QOL of patients was evaluated with the 15-item Myasthenia Gravis Quality of Life (MG-QOL15). Current MG severity was evaluated with MGFA grade, MG-ADL score, MGC score, and MGFA Postintervention Status. The data about gender, age of onset, subgroup, antibodies, age, duration, education, employment state, marital status, skeletal muscle affected, thymic histology, and current treatment methods of the patient were collected. A total of 185 patients [72 males (38.9%), aged 45.2 years (14-77)] with MG were enrolled. Age at onset was 38.3 ± 17.9 years, and disease duration was 87.9 months (0-672). The median MG-QOL15 score was 12.5 (0-58). The item "have trouble using my eyes" was the highest scoring item in both ocular and generalized patients with MG. The MG-QOL15 score was significantly different among patients with OMG (9.2 ± 9.4, n = 63), GMG (9.0 ± 8.8, n = 22), and BMG (15.4 ± 14.2, n = 100) (P = 0.018). Patients with BMG had higher MG-QOL15 scores than OMG (P = 0.001) and GMG (P = 0.009), but there was no significant difference between OMG and GMG (P = 0.467). The MG-QOL15 score was significantly lower in patients who had undergone thymectomy (9.7 ± 9.8, n = 58) compared to those who had not (13.8 ± 13.4, n = 127, P = 0.022). MG-QOL15 score was significantly lower in patients who underwent thymectomy compared to those who did not (9.7 ± 9.8, n = 58 vs. 13.8 ± 13.4, n = 127, P = 0.022). MG-QOL15 score was different among MGFA grades (Remission: 5.2 ± 5.4, n = 41; I: 11.3 ± 10, n = 61; II: 11.6 ± 11.1, n = 40; III: 18.1 ± 12.1, n = 29; and IVa: 30.1 ± 20, n = 14, P < 0.001). There was no significant difference between patients in MGFA grade I and II (P = 0.896), and there was no significant difference between patients in MGFA grade III and IVa (P = 0.052). MG-ADL (P < 0.001) and MGC (P < 0.001) were positively correlated with MG-QOL15. Men had higher MG-QOL15 than women (P = 0.094), and LOMG had higher MG-QOL15 than EOMG (P = 0.072). Multivariate linear regression identified that higher MG-ADL (P < 0.001), higher MGC (P = 0.02), and poor employment status (P = 0.045) were independently associated with higher MG-QOL15. Having trouble using the eyes accounted for the highest score in MG-QOL15, eye symptoms affect QOL more than limb weakness in MG. Daily life activity, disease severity, and employment status were associated with patients' QOL. Adequate treatment should be applied to improve QOL, while mild symptoms can be accepted. Men and patients over the age of 50 years of onset may need more attention.

Clinical Profile and Quality of Life in Myasthenia Gravis Using MGQOL15 R(Hindi): An Indian Perspective.

Myasthenia Gravis (MG) is a chronic fluctuating illness, due to the dysfunction of neuromuscular junction which is autoimmune in nature. The disease severely affects the Quality Of Life (QOL). The primary objective of our study was to assess the QOL in patients with MG using Short Form 36 (SF 36) and MGQOL 15 R (Hindi translated). The secondary objective was to assess the correlation of age, sex, illness duration, clinical characteristics, severity, and treatment with the QOL in MG patients. A cross sectional study of 55 MG patients was done to analyse and evaluate the clinical status using Hybrid Myasthenia Gravis Foundation of America (HMGFA), Myasthenia gravis composite score (MGCS) and The Myasthenia Gravis Activities of Daily Living (MG - ADL). QOL was assessed by SF 36 and Hindi version of Myasthenia Gravis Quality of Life 15 - Revised (MG-QOL15R) score. 78.2% patients had generalized MG. The mean MGC and MG-ADL scores were 5.27 and 3.29 (95% CI: 2.24 -4.34) respectively. The mean MGQOL15R score was 6.52 ± 7.7 and the score correlated with the symptoms. The SF 36 scores were the best and the worst in the bodily pain (93.72 ± 13.52) and general health subset (61.81 ± 39.64) respectively. Except for steroid dose, there was no significant correlation between SF36 and other factors. QOL in MG was found to be affected due to the disease. The MGQOL 15 R scores correlated with the clinical features, remission or active status, steroid use and thymectomy. No Significant association was observed between MG QOL scores and various lab parameters and repetitive nerve stimulation (RNS) test results. Higher dose of steroid was associated with poor QOL, while thymectomy was associated with better QOL scores. MGQOL15R (Hindi) is a quick and simple tool to assess the QOL in MG patients.

Characteristics of Repetitive Nerve Stimulation (Rns) and Correlation with Severity and Quality of Life of Myasthenia Gravis Patients

Background: Myasthenia gravis is a rare autoimmune disease due to neuromuscular junction (NMJ) disorders. The diagnosis of myasthenia gravis is based on cardinal clinical symptoms, including fluctuating weakness, worsening with activity, and improving with rest. In addition to clinical signs and antibody tests, other tests that are also crucial for diagnosis are electrodiagnostic examination (EDX) such as repetitive nerve stimulation (RNS) and single fibre electromyography (SFEMG). Recent studies have focused on assessing patients' severity and quality of life, essential for determining the prognosis and subsequent treatment plans. Objective: To evaluate the characteristic of RNS and its correlation with rigor and quality of life of myasthenia gravis patients. Methods: Inclusion criteria: patients diagnosed with myasthenia gravis, aged 18-65 years, willing to participate in the study. Exclusion criteria include a history of neuropathy, motor neuron disease, or myopathy and dropping out of the study. Patient demographic data were collected. The results of the RNS examination were divided into two categories, normal and abnormal. MG severity was assessed by the Myasthenia gravis foundation of America (MGFA) classification, and the patient's quality of life was assessed by Myasthenia Gravis Quality of Life 15 (MG-QOL 15). Results: Of the 24 samples, 69.1% of RNS were positive. The most sensitive muscle for assessing RNS is the anconeus muscle, followed by the trapezius and nasal. There was no significant relationship between RNS features and severity and the quality of life of myasthenic Gravis patients. Conclusion: Onconeus muscle is the best location for assessing RNS. Although the number of patients with normal RNS had lower severity and better quality of life, there was no statistically significant relationship. Further studies with larger samples and longer follow-ups are needed.

VP.38 A double-blinded, randomized, placebo-controlled phase II study of FcRn antagonist batoclimab in Chinese generalized myasthenia gravis patients

A randomized, double-blind, placebo-controlled, parallel phase II study was conducted for generalized myasthenia gravis (gMG) in Chinese population to evaluate the efficacy and safety of batoclimab as a novel anti-FcRn therapy. Eligible patients were randomized in a 1:1:1 ratio to placebo, batoclimab 340 mg, 680 mg groups, respectively. In double-blinded period, patients received batoclimab or placebo on days 1, 8, 15, 22, 29, and 36. In the open-label period, all patients received batoclimab 340 mgon days 50, 64, and 78. In follow-up period, patients were examined on days 92, 106, and 120. The primary endpoint was myasthenia gravis activities of daily living (MG-ADL) score change on day 43 from baseline. Secondary endpoints included assessment on Quantitative Myasthenia Gravis (QMG), Myasthenia Gravis Composite (MGC), Myasthenia Gravis Quality of Life (MG-QoL), pharmacokinetics, pharmacodynamics and safety events. Totally 30 subjects were randomized, with 9,10 and 11 in the placebo, batoclimab 340mg and 680mg groups respectively.MG-ADL score changes from baseline to day 43 were -2.2±0.9, -4.7±0.6, and -4.4±1.0 in the placebo, batoclimab 340 mg, and 680 mg groups, respectively. Similar changes were observed in QMG, MGC, and MG-QoL scores. The proportion of patients with clinically significant improvement on day 43 was higher in batoclimab groups. In open-label period, symptom recurrence and IgG levels rise again except the former placebo group. On day 120 the placebo group had more improvement compared to batoclimab groups, with IgG levels reaching a plateau. Treatment-emergent adverse events (TEAEs) were comparable among the three groups. No death or TEAEs leading to study discontinuation was observed. The batoclimab groups experienced mild-to-moderate albumin reductions and total cholesterol elevation, all returned to baseline in follow-up without clinical interference. In conclusion, batoclimab is effective and safe in Chinese patients with gMG.

Efficacy and Safety of Rituximab for New-Onset Generalized Myasthenia Gravis

Rituximab is a third-line option for refractory generalized myasthenia gravis (MG) based on empirical evidence, but its effect in new-onset disease is unknown. To investigate the efficacy and safety of rituximab compared with placebo as an add-on to standard of care for MG. This randomized, double-blind, placebo-controlled study took place throughout 48 weeks at 7 regional clinics in Sweden. Key inclusion criteria were age older than 18 years, onset of generalized symptoms within 12 months or less, and a Quantitative Myasthenia Gravis (QMG) score of 6 or more. Patients were screened from October 20, 2016, to March 2, 2020. Key exclusion criteria included pure ocular MG, suspected thymoma, previous thymectomy, and prior noncorticosteroid immunosuppressants or high doses of corticosteroids. Participants were randomized 1:1 without stratification to a single intravenous infusion of 500 mg of rituximab or matching placebo. Minimal disease manifestations at 16 weeks defined as a QMG score of 4 or less with prednisolone, 10 mg or less daily, and no rescue treatment. Of 87 potentially eligible patients, 25 were randomized to rituximab (mean [SD] age, 67.4 [13.4] years; 7 [28%] female) and 22 to placebo (mean [SD] age, 58 [18.6] years; 7 [32%] female). Compared with placebo, a greater proportion with rituximab met the primary end point; 71% (17 of 24) in the rituximab group vs 29% (6 of 21) in the placebo group (Fisher exact test P = .007; probability ratio, 2.48 [95% CI, 1.20-5.11]). Secondary end points, comparing changes in Myasthenia Gravis Activities of Daily Living and Myasthenia Gravis Quality of Life at 16 weeks with QMG at 24 weeks did not differ between groups with censoring for rescue treatment (per-protocol analysis) but were in favor of active treatment when rescue treatment was taken into account by worst rank imputation (post hoc analysis). Rescue treatments were also more frequent in the placebo arm (rituximab: 1 [4%]; placebo, 8 [36%]). One patient in the placebo arm had a myocardial infarction with cardiac arrest and 1 patient in the active arm experienced a fatal cardiac event. A single dose of 500 mg of rituximab was associated with greater probability of minimal MG manifestations and reduced need of rescue medications compared with placebo. Further studies are needed to address long-term benefit-risk balance with this treatment. ClinicalTrials.gov Identifier: NCT02950155.

Effect of tacrolimus on soluble costimulatory molecules in patients with refractory myasthenia gravis

ObjectivesTo investigate the expression and possible role of soluble costimulatory molecules in the treatment of refractory myasthenia gravis. MethodsThirty-two patients with refractory myasthenia gravis were enrolled into this study and given tacrolimus 3 mg/day. At the beginning of treatment and 12 months follow-up period, clinical data were collected and recorded. The clinical classification of myasthenia gravis Foundation (MGFA) was performed. The MGFA-quantitative myasthenia gravis score (MGFA-QMGS), manual muscle test (MMT), MG activity of daily living (MG-ADL) and the activity of daily living (MG-ADL), the 15-item myasthenia gravis quality of life (MG QOL-15) and the dose change of prednisone were used to evaluate the efficacy. The expression levels of soluble costimulatory molecules and their ligands (sPD-1/sPD-L1, sICOS/sICOSL, sCD40/sCD40L), soluble CD25 and IL-2 in serum were determined by enzyme-linked immunosorbent assay (ELISA). ResultsWe observed that oral administration of 3 mg tacrolimus daily for 1 year can significantly improve the clinical symptoms of patients with refractory myasthenia gravis, which is characterized by a significant reduction in clinical scores, such as QMG, MMT, ADL, MGQOL-15, and a reduction daily oral prednisolone (PSL) dose (P < 0.0001).We also found that the levels of plasma sPD-1, sCD40, IL-2 in refractory MG patients increased significantly, and those decreased significantly 12 months after tacrolimus treatment (P < 0.05). The level of sCD25 was negatively correlated with clinical severity scores (P < 0.05). After tacrolimus treatment, the level of sPD-L1 increased although there was no significant difference. ConclusionTacrolimus could relieve the symptoms of refractory MG and significantly decrease the levels of plasma sPD-1, sICOSL, sCD40, sCD25 and IL-2. Soluble costimulatory molecules might be potential biomarkers for MG and tacrolimus treatment.

Effectiveness of modified Buzhong Yiqi decoction in treating myasthenia gravis: study protocol for a series of N-of-1 trials

BackgroundMyasthenia gravis (MG) is an acquired autoimmune disease with high heterogeneity. The disease is chronic, relapsing repeatedly and progressive with acute exacerbation occasionally. Although the treatment of MG has developed, it is still unsatisfactory and has some unexpected side effects. Traditional Chinese medicine (TCM) has shown great potential in MG treatment, including relief of muscle weakness syndrome, improvement of patient’s quality of life, and reduction of side effects of western medicine. The purpose of this study is to evaluate the effectiveness of modified Buzhong Yiqi decoction (MBYD) as an add-on therapy for MG through a small series of N-of-1 trials.MethodsSingle-centre, randomized, double-blind, 3 crossover N-of-1 trials will be conducted to enroll patients with MG diagnosed as spleen-stomach deficiency syndrome or spleen-kidney deficiency syndrome in TCM. Each N-of-1 trial has 3 cycles of two 4-week periods containing the MBYD period and placebo period. The wash-out interval of 1 week is prior to switching each period. Primary outcome: quantitative myasthenia gravis (QMG). Secondary outcomes: the following scales: myasthenia gravis composite (MGC), myasthenia gravis activities of daily living profile (MG-ADL), myasthenia gravis quality of life (MG-QOL); the level of CD4+FoxP3+Treg cells and cytokines (IL-4, IL-17A, INF-γ, TGF-β) in the peripheral blood; the alterations of the composition of gut microbiota; reduction of the side effects of western medicine.DiscussionUsed by WinBUGS software, we will conduct a hierarchical Bayesian statistical method to analyze the efficacy of MBYD in treating MG in individuals and populations. Some confounding variables such as TCM syndrome type and potential carryover effect of TCM will be introduced into the hierarchical Bayesian statistical method to improve the sensitivity and applicability of the trials, and the use of prior available information within the analysis may improve the sensitivity of the results of a series of N-of-1 trials, from both the individual and population level to study the efficacy of TCM syndrome differentiation. We assumed that this study would reveal that MBYD is effective for MG and provide robust evidence of the efficacy of TCM to treat MG.Trial registrationChinese Clinical Trial Register, ID: ChiCTR2000040477, registration on 29 November 2020.

Studying the relationship between clinical features and mental health among late-onset myasthenia gravis patients.

BACKGROUNDMental disorders are common comorbidities among individuals with neurological diseases, and the prevalence of depressive and anxiety-related symptoms in newly referred patients at neurology outpatient clinics is high. There have been few studies on the mental health of patients with late-onset myasthenia gravis (MG).AIMTo examine the relationship between clinical features and the mental health symptoms within late-onset MG patients.METHODSA total of 105 patients diagnosed with MG were recruited consecutively from a neuromuscular outpatient clinic between December 2020 and February 2021. Patients were classified into two groups: early-onset MG (age at onset < 50 years, n = 63) and late-onset MG (age at onset ≥ 50 years, n = 42). Social demographic data and information about marital status, education level, clinical symptoms, serum antibody levels, and therapies used were collected for all participants. Participants were also evaluated using the Myasthenia Gravis Composite scale, the Myasthenia Gravis Activities of Daily Living scale, the Myasthenia Gravis Quality of Life 15 (MG-QOL-15) questionnaire, the 17-item version of the Hamilton Depression Rating Scale (HAM-D) and the Hamilton Anxiety Rating Scale (HAM-A). The relationship between clinical features and mental health in late-onset MG patients was examined using multivariate logistic regression analyses. RESULTSLate-onset MG patients were more prone to dyspnea, had higher levels of serum anti-acetylcholine receptor antibodies, and higher total scores on the MG-QOL-15, HAM-D, and HAM-A questionnaires, than early-onset MG patients had (P < 0.05). Among those with late-onset MG, female patients had higher total HAM-D and HAM-A scores than male patients had (P < 0.05). High scores on the QOL-15 questionnaire were associated with higher incidences of anxiety and depression, and the association was found to be independent after adjusting for confounding risk factors. In the late-onset subgroup, the areas under the receiver operating characteristic curves for the MG-QOL-15 score-based diagnostic accuracy for anxiety and depression state were 0.816 (P = 0.001) and 0.983 (P < 0.001), respectively.CONCLUSIONHigher MG-QOL-15 scores were a risk factor for anxiety and depression in late-onset MG, and women with late-onset MG were more likely to have anxiety and depression than men were.

Validation of myasthenia gravis activity of daily living questionnaire: Persian version.

Background: The 8-item myasthenia gravis activity of daily living (MG-ADL) questionnaire is a valid and reliable instrument for evaluating myasthenia gravis (MG)-associated disability. This study aims to assess its validity and reliability in the Iranian population. Methods: A total number of 58 patients with MG were qualified for the examination. All 58 patients completed the Persian translation of 15-item Myasthenia Gravis Quality of Life (MG-QOL15) and MG-ADL questionnaires initially, and 30 filled out the MG-ADL questionnaire 2 to 4 weeks after the initial visit. Pearson correlation coefficient of questionnaires, internal consistency using Cronbach's alpha (α), and test-retest repeatability of the questionnaire were evaluated. Results: The Pearson correlation coefficient of Persian versions of MG-QOL and MG-ADL was 0.93 (P < 0.01). The Persian MG-ADL showed satisfactory internal consistency (Cronbach’s α = 0.89) and test-retest reliability (r = 0.99, P < 0.01). Conclusion: The Persian MG-ADL is a valid and reliable questionnaire for the determination of activities of daily life in Iranian patients with MG.

Rest or 30 min walk as exercise intervention (RESTOREX) in myasthenia gravis: A randomized controlled trial

Introduction There is a lack of evidence about the usefulness of exercise or rest in myasthenia gravis (MG). This study is aimed to evaluate the efficacy and safety of exercise or rest in MG. Methods In a single-center open-labeled randomized controlled trial, the patients with mild to moderate MG were randomized to 30-min walk or rest in addition to the standard treatment. The primary endpoint was 50% improvement in the MG Quality of Life (MG-QOL15), and secondary endpoints were change in the Myasthenic Muscle Score (MMS), MG Activities of Daily Living (MGADL), grip strength, dose of acetylcholine esterase inhibitor and prednisone, 6-min walk test (6MWT), decrement in trapezius on the low-rate repetitive nerve stimulation test, and adverse events. The outcomes were defined at 3 months, by >50% improvement in these outcome parameters. Results Forty patients with MG were randomized to the exercise or rest arm. The 2 arms were matched for demographic and clinical parameters. The patients in the exercise arm had significantly better QOL evidenced by MG-QOL15 (p = 0.02). The secondary endpoints, distance covered in 6MWT (p = 0.007), were also better in the exercise arm without any adverse event. Conclusion Regular exercise for 30 min in mild and moderate MG improves quality of life and walking distance compared to rest and is safe. Clinical trial registration The clinical trial registration number is CTRI/2019/11/021869.

Do people with Myasthenia Gravis need speech-language pathology services? A national survey of consumers’ experiences and perspectives

Purpose: This study explored experiences of bulbar (speech, voice and swallowing) symptoms in Australian adults with Myasthenia Gravis (MG), and the relationship between these symptoms and community participation and quality-of-life. Further, it examined access to and experiences with speech-language pathology (SLP) services, and awareness and perceptions of the SLP’s role. Method: A cross-sectional mixed methods online survey collected data using researcher-designed questions and patient-reported outcome measures (PROMs) including the Dysarthria Impact Profile (DIP), Voice Handicap Index (VHI), Dysphagia Handicap Index (DHI), Community Integration Questionnaire–Revised (CIQ-R), and Myasthenia Gravis Quality of Life–Revised 15 (MG-QOL15r). Analyses included descriptive and non-parametric statistics, and content analysis. Result: Participants were 111 adults with MG living in Australia. 74% of respondents reported experiencing symptoms of speech difficulties but only 20% of those were referred to SLP services. Similarly, 85% of respondents reported experiencing swallowing difficulties, but only 26% were referred to SLP. Voice handicap (VHI) was strongly correlated with the psychosocial impact of dysarthria (DIP) and dysphagia handicap (DHI). Increased voice and dysphagia handicap were moderately correlated with worse quality-of-life. There was generally poor awareness of the SLP’s role and many were reluctant to attend SLP appointments. Conclusion: These findings highlight unmet consumer needs for MG-related bulbar symptoms, and a lack of awareness of SLP services. It is suggested that, in addition to other strategies, consumer education and increased awareness are required to address these unmet needs.

Rest or 30-Min Walk as Exercise Intervention (RESTOREX) in Myasthenia Gravis: A Randomized Controlled Trial

Introduction: There is a lack of evidence about the usefulness of exercise or rest in myasthenia gravis (MG). This study is aimed to evaluate the efficacy and safety of exercise or rest in MG. Methods: In a single-center open-labeled randomized controlled trial, the patients with mild to moderate MG were randomized to 30-min walk or rest in addition to the standard treatment. The primary endpoint was 50% improvement in the MG Quality of Life (MG-QOL15), and secondary endpoints were change in the Myasthenic Muscle Score (MMS), MG Activities of Daily Living (MGADL), grip strength, dose of acetylcholine esterase inhibitor and prednisone, 6-min walk test (6MWT), decrement in trapezius on the low-rate repetitive nerve stimulation test, and adverse events. The outcomes were defined at 3 months, by >50% improvement in these outcome parameters. Results: Forty patients with MG were randomized to the exercise or rest arm. The 2 arms were matched for demographic and clinical parameters. The patients in the exercise arm had significantly better QOL evidenced by MG-QOL15 (p = 0.02). The secondary endpoints, distance covered in 6MWT (p = 0.007), were also better in the exercise arm without any adverse event. Conclusion: Regular exercise for 30 min in mild and moderate MG improves quality of life and walking distance compared to rest and is safe. Clinical Trial Registration: The clinical trial registration number is CTRI/2019/11/021869.

Determinants of Quality of Life in Myasthenia Gravis Patients.

Background: Although approximately half of myasthenia gravis (MG) patents achieve remission, for the remaining group MG is often a life-long disease. Better understanding of the determinants of Quality of Life (QoL) in MG is needed to optimize treatment goals in chronic cases.Materials and Methods: We performed a single center cross-sectional study in 339 MG adult patients (64.9% women), with ocular or generalized disease. SF-36 and a structured questionnaire was administered, including information on previous and current MG severity, medications, comorbidities, education, occupation and BMI of the patient. Mean disease duration was 7.5 + 9.3 years. Current age was 51.6 + 18.3 years, 55% had Early-Onset (<50 years) MG.Results: There were no statistically significant differences in mean SF-36 subscores between women and men. Worse MGFA class was related to lower QoL in physical (PCS) and mental (MCS) subscore (p = 0.000 for both). Patients with MGFA I-II class had significantly better QoL in physical and mental subscores than patients with more severe MG (p < 0.005). Late-onset MG patients had worse QoL than EOMG in physical score domain PCS (p = 0.049). Overweight and obese patients had lower PCS (p = 0.002) and MCS (p = 0.038) than patients with normal BMI. University education was related to statistically higher PCS (p = 0.015) and MCS (p = 0.006). QoL in currently employed was better in PCS and MCS (p = 0.000), with white collar workers reporting higher PCS (p = 0.049) than the remaining group. Patients living with family evaluated their MCS (p = 0.015) better than living alone. Moderate physical activity (twice a week) improved PCS (p = 0.045).Conclusion: Our study confirmed that greater severity of symptoms, age, age of onset but also BMI, type of work, education status and physical activity affect QoL in MG.

Rest or Exercise (RESTOREX) in Myasthenia Gravis: A Randomized Controlled Trial

Background: In Myasthenia Gravis (MG), the effect of exercise is not well documented in controlled trial. In the present study, the efficacy and safety of exercise in MG in comparison to rest is presented. Methods: In this single-center open-labeled randomized clinical trial the patients were randomized in 1:1 ratio to exercise (30 min walk) or rest arm in addition to standard treatment of MG. The Primary endpoint was 50% change in Myasthenia Gravis Quality of Life (MG-QOL15) at 3 months and secondary endpoints were change in Myasthenic Muscle Score (MMS), Myasthenia Gravis Activities of Daily Living (MGADL), grip strength, AChEI and prednisone dose, 6 minute walk test (6MWT), decrement in trapezius muscle and adverse events at 3 months. The Outcome was defined at three months by 50% change compared to the baseline parameters. Results: There were 20 patients in each arm which were matched for demographic and clinical parameters. The patients in exercise arm had significantly better QOL as evidenced by 50% improvement in MG-QOL15 (P = 0.02), increase in number of steps (P = 0.03) and the distance covered in 6MWT (P = 0.003). The scores of MG-QOL15 (P = 0.03), MMS (P = 0.048), and distance travelled (P <0.001) also revealed significant group difference. Intragroup comparison revealed that both exercise and rest arm significantly improvement with respect to MG-QOL15 in exercise (P = 0.001) and rest (P = 0.001), MMS in exercise (P = 0.001) and rest (P = 0.001), reduction in pyridostigmine (P = 0.03) and prednisone (P =0.001) dose in exercise, increase in number of steps in exercise (P =0.001) and increase in walking distance in 6MWT in both exercise (P =0.001) and rest arm (P = 0.023). There was no adverse event in any group. Interpretations: The study provides class II evidence of improved quality of life in mild to moderate MG by 30 min walk in comparison to rest. Trial Registration: The trial was registered in Clinical Trial Registry of India (CTRI/2019/11/021869). Funding Statement: World Congress of Neurology, the authors received no funding for this study. Declaration of Interests: All authors declare no competing interest. Ethics Approval Statement: This study was approved by Institutional Ethics committee (PGI/BE/632/2018), SGPGIMS, Lucknow.

Preliminary Findings of a Dedicated Ocular Myasthenia Gravis Rating Scale: The OMGRate

ABSTRACT There is a pressing need for a robust rating scale for ocular myasthenia gravis (OMG). Rating scales for myasthenia gravis (MG) research have a predominant focus on generalised disease. We present results of the first dedicated rating scale for OMG: the ocular myasthenia gravis rating scale (OMGRate). The OMGRate was developed through an international collaboration between neuromuscular and neuro-ophthalmology experts in OMG. It comprises two components: a physician- examination (OMGRate-e) and a patient questionnaire (OMGRate-q).. The OMGRate was prospectively validated in patients attending a neuro-ophthalmology clinic from April 2017 to October 2018. External validity and reliability of OMGRate were evaluated using validated MG rating scales: the Myasthenia Gravis Composite (MGC), the Myasthenia Gravis Quality of Life (MG-QOL), and the ocular component from the Myasthenia Gravis Impairment Index questionnaire (MGII). Two hundred and eleven assessments were completed in 104 patients (67 males, mean age 55 y, range 18–86 y). There was very good external validity of the OMGRate: good correlation between OMGRate-e and MGC (r = 0.64, 95% confidence intervals [CI] 0.54–0.74, p < .0001); excellent correlation between OMGRate-q and MGII (r = 0.85, 95% CI 0.78–0.91, p < .0001) and good correlation between OMGRate and MG-QOL (r = 0.68, 95% CI 0.60–0.77, p < .0001). A higher correlation of OMGRate and MG-QOL compared with MGC and MG-QOL (r = 0.47, 95% CI 0.34–0.59, p < .0001) suggests that OMGRate is better able to capture significant QOL information in patients with OMG. It had excellent reliability with an intraclass correlation coefficient of 0.83 (95% CI 0.67–0.92). Feedback from examiners and patients indicated that the OMGRate was easy to use. In conclusion, OMGRate is an easy-to-use, valid and reliable rating scale for monitoring the severity of OMG.

The Relationship between Restless Legs Syndrome and Quality of Life in Patients with Myasthenia Gravis

Background: Restless legs syndrome (RLS) is characterized for an uncomfortable sensation in legs and an irresistible desire to move them. This disorder has been more recently recognized in patients with myasthenia gravis (MG) and can interfere with the quality of life (QOL). Objectives: The aims of this study are to describe the prevalence of RLS and its severity and influence on the QOL in patients with MG. Method: This was a cross-sectional study conducted from May to June 2016 in Recife, Brazil. A sample of 42 patients was interviewed using a sociodemographic questionnaire, MG QOL questionnaire-15 and The RLS Rating Scale. Results: RLS was present in 47.6% of patients and of these 40.5% met moderate to severe RLS criteria. Patients were 45 years on average (SD ± 14.4) and women represented 57.1% of the study population. Among patients with RSL, the quality-of-life scores were worse (p = 0.010) on average. There was no association of RLS with the duration of MG, use of immunosuppressant or clinical conditions that could mimic the occurrence of RLS. Conclusion: RLS is a prevalent condition in patients with MG, and may be severe enough to negatively impact QOL.