Myasthenia Gravis Composite Scale Research Articles

Triple-seronegative myasthenia gravis: clinical and epidemiological characteristics.

Myasthenia gravis (MG) is an autoimmune disease usually caused by antibodies against the acetylcholine receptor (AChR-Abs), muscle-specific tyrosine kinase (MuSK-Abs), or low-density lipoprotein receptor-related protein 4 (LRP4-Abs). However, there are MG patients who do not have these antibodies and are thus said to have triple-seronegative (triple-SN) MG. This study aims to describe the frequency and clinical and epidemiological characteristics of patients with triple-SN MG. This was a retrospective cross-sectional study carried out through the analysis of medical records. Descriptive and analytical statistical analysis was performed comparing subgroups of myasthenic patients, classified according to serological profile. The sample population consisted of 93 MG patients: 85 were positive for antibodies, 80 (86%) with AChR-Abs, 5 (5.4%) with MuSK-Abs, and no MG patients with LRP4-Abs. Eight patients (8.6%) had triple-SN MG; they had a median age at disease onset of 30 years (21-45). Their most common initial symptoms were ptosis, diplopia, and generalized weakness. Most patients presented with mild symptoms at their last visit, reflecting a median MG composite scale score of 4 (0-6), and 75% of patients had an adequate response to treatment. Our study showed a low frequency of triple-SN MG in Brazilian MG patients. Triple-SN MG was predominant in females, who presented with ptosis, diplopia, and generalized weakness, and most patients had an adequate response to immunosuppressive treatment. There was no significant difference between triple-SN MG and the other subgroups.

Clinical characteristics and outcomes of COVID-19 infection among myasthenia gravis patients in a national referral hospital in Indonesia

Introduction. Due to their immunocompromised state from immunosuppressive therapy and respiratory efficiency, patients with Myasthenia Gravis (MG) are more likely to get infected with COVID-19. Currently, reports on MG patients exposed with COVID-19 in Indonesia are still limited. Therefore, this study was conducted to determine the clinical characteristics and outcomes of MG patients infected with COVID-19, especially in Jakarta, Indonesia. Methods. It is a retrospective study of MG patients in a National Referral Hospital in Indonesia from September 2021 to December 2022. The data obtained includes demographics, medical history, clinical characteristics, and severity of MG assessed by MG composite scale (MGCS), Myasthenia Gravis Foundation of America (MGFA) classification, symptoms, and therapy. Results. Of the 8 patients, 7 patients were female. The range of age at COVID-19 onset was 28-62 years old. On admission to the hospital, 2 patients were mild MG, 2 patients were moderate MG, 3 patients with severe MG, and one patient could not be identified because the patient came in an unconscious state. Five patients were classified as moderate COVID-19, two patients had severe COVID-19, and one patient was classified as critical COVID-19. Most patients experience MG exacerbation or require oxygen support during hospitalization. Only one patient deceased while receiving treatment at the emergency room, while the rest improved and were discharged home. Conclusion. MG patients exposed with COVID-19 tend to experience moderate, severe, and critical COVID-19 symptoms. However, with proper management of the infection and other comorbidities, MG patients with COVID-19 may achieve favorable outcome.

Physical and mental fatigue in myasthenia gravis and its correlation with other symptoms.

Muscle weakness and easy fatigability are the clinical hallmarks of myasthenia gravis (MG). However, fatigue perception, which can be seen quite often in myasthenic patients, and its effect on the quality of life, irrespective of motor deficit, has not been elucidated yet. The aim is to evaluate the frequency of fatigue in myasthenic patients with nearly full muscle strength and the effect of fatigue on quality of life by assessing its correlation with other symptoms. Fifty-three patients with ocular or mild generalized MG in remission or minimal manifestations completed the questionnaires measuring the severity of MG and quality of life (MG Composite Scale and MG-Activities of Daily Living Profile). Both patient group and control group (53 healthy volunteers)completed the scales assessing fatigue [Fatigue Assessment Scale (FAS) and Fatigue Impact Scale (FIS)], depression [Beck Depression Inventory (BDI)] and sleep (Epworth Sleepiness Scale). Disease severity was assessed using MG Foundation of America (MGFA) and MGFA Post-Intervention Status classifications. FAS, FIS physical and BDI scores were significantly higher in patients compared to the control group (p = 0.003, p = 0.001, and p = 0.003, respectively) and fatigue was associated with depression and daytime sleepiness. Inpatient group, depressive symptoms and daytime sleepiness were higher in females (p = 0.019 and p = 0.013). The mean values of FIS total and cognitive scores were higher in patients with generalized MG (p = 0.033 and p = 0.045). Fatigue scores correlated with motor signs. Fatigue can be seen in MG independently from muscle weakness and is an important symptom worsening the quality of life.

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.

Brazilian translation, cross-cultural adaptation and validation of the MG Composite scale and Quantitative Myasthenia gravis testing form: a multicentric study protocol.

Introduction: Myasthenia gravis (MG) is a chronic and inflammatory disease characterized by progressive weakness of the skeletal muscles due to changes in the synaptic junctions between nerves and muscle fibres. Typically, patients have a history of progressive weakness and fatigue in skeletal muscles, which occurs during sustained or repeated activities and improves after rest. Scoring systems to assess the degree of disease severity are commonly used in clinical drug trials. Further, specific instruments to assess health-related quality of life (HRQOL) have been designed to measure self-perception of symptoms and wellness during the disease or after disease treatment. HRQOL is an important component of patient evaluation, as some individuals can tolerate severe impairment and disability, while others may be very unhappy even with less severe symptom profiles. The objective of the proposed study is to translate and perform a cultural adaptation and validation of two specific instruments for assessing the clinical course of patients with MG. These instruments include the Quantitative Test for Myasthenia gravis (QMG) and Myasthenia gravis Composite Scale (MGC). Methods and analyses: The study design will follow a multi-method approach, and will be conducted in three stages consisting of translation, cultural adaptation, and validation of the QMG and MGC for Brazilian Portuguese patients. This multi-step process will consider aspects of construct and predictive validity as well as reliability and structure of the instrument. The entire process will follow the procedures proposed and developed in other linguistic and social-cultural contexts based on previous studies. Ethics: This study has received approval from the Human Research Ethics Committee of Nove de Julho University (Brazil) under process no. 360.488 based on the Helsinki declaration and is registered with the World Health Organization (WHO) under Universal Trial Number (UTN) U1111-1147-7853 and the Brazilian Registry of Clinical Trials (REBEC) RBR -7ckpdd.

Intravenous cyclophosphamide monthly pulses in refractory myasthenia gravis.

Refractory myasthenia gravis (MG) is defined as a failure to respond adequately to conventional therapies, the inability to reduce immunosuppressive therapy without clinical relapse or the need for ongoing rescue therapy, severe adverse effects from immunosuppressive therapy (treatment intolerant) or frequent myasthenic crisis even on therapy. Cyclophosphamide (CYC) is a DNA alkylating agent that causes important interference in transcription processes and DNA replication, it has been used in refractory MG with controversial results. We aim to determine the efficacy of CYC in refractory MG in the Mexican population. In an observational, longitudinal retrospective study, we identified eight refractory MG patients treated with 30-50mg/kg monthly CYC for at least 6months. The efficacy was assessed by Osserman scale considering significant improvement a ≥ 1 point reduction and Myasthenia Gravis Composite Scale. The relapse-free and remission-free period were also calculated using the Kaplan-Meier statistic. Clinical improvement was achieved in 75% of the patients. According to the Kaplan-Meier analysis, the median progression-free survival (PFS) was 9 (6.2-11.5) months and the median time to progression (TTP) was 4 (1-8) months. Response was independent of patient's characteristics, except for the MG age of onset (p = 0.0025). CYC was effective in all patients with refractory MG for a mean of 9months, with worsening thereafter, which could be associated with low cumulative dose. The symptomatic improvement with CYC was noted within the 1st month. We conclude that CYC is effective as an induction to remission therapy, although our data suggest it is not effective as a long-term therapy.

Muscle strength and psychiatric symptoms influence health-related quality of life in patients with myasthenia gravis

Myasthenia gravis (MG) is a neuromuscular autoimmune disease characterized by skeletal muscle weakness which can impact motor function and, furthermore, produce negative impact on the health-related quality of life (HRQOL). Objective: To evaluate the predictors for HRQOL in patients with MG. Methods: Eighty patients were evaluated with the MG Foundation of America classification and the MG Composite scale. HRQOL was estimated by the MGQOL15, while anxious and depressive symptoms were evaluated with the Hospital Anxiety and Depression Scale (HAD). Results: The mean age of patients was 41.9 years with mean illness duration of 13.5 years. Almost half of the patients (43.75%) had significant anxiety and more than a quarter (27.50%) had depressive symptoms. Factors that influenced the HRQOL in MG were skeletal muscle weakness and anxiety and depressive symptoms (p < .001 in logistic regression model). Conclusion: Anxiety and depressive symptoms, besides motor symptoms, influence HRQOL in MG. Mental health must be a clinical focus in addition to the treatment of somatic symptoms during the course of MG.

Amifampridine phosphate in the treatment of muscle-specific kinase myasthenia gravis: a phase IIb, randomized, double-blind, placebo-controlled, double crossover study

Objective:The aim of this study is to determine the safety and the efficacy of amifampridine phosphate in muscle-specific kinase antibody-positive myasthenia gravis, in a 1:1 randomized, double-blind, placebo-controlled, switchback, double crossover study.Methods:Eligible patients had muscle-specific kinase myasthenia gravis, >18 years of age, and Myasthenia Gravis Foundation of America class II–IV with a score of ⩾9 on Myasthenia Gravis Composite scale. After the run-in phase, during which amifampridine phosphate was titrated to a tolerable and effective dosage, patients were randomized to receive placebo–amifampridine–placebo sequence or amifampridine–placebo–amifampridine sequence daily for 7 days. Then, patients switched treatment arms twice, for a total of 21 days of double-blind treatment. Safety was determined by serial assessments of adverse events/serious adverse events, physical examinations, and clinical and laboratory tests. The co-primary outcome measures included changes from baseline of Quantitative Myasthenia Gravis score and Myasthenia Gravis–specific Activities of Daily Living Profile score. The secondary outcome measures comprised changes from baseline of Myasthenia Gravis Composite score, Myasthenia Gravis Quality of Life scale—15 questions, Fatigue Severity Scale, and Carlo Besta Neurological Institute–Myasthenia Gravis scale. Statistical analyses were assessed using a switchback model for three-period, two-treatment crossover design.Results:A total of 10 patients were screened, enrolled, and treated. Transient paresthesias (60%) were the only amifampridine phosphate–related adverse events reported. Four patients were randomized to receive placebo–amifampridine–placebo sequence and three patients to receive amifampridine–placebo–amifampridine sequence. The co-primary objectives were statistically met (Quantitative Myasthenia Gravis score: p = 0.0003 and Myasthenia Gravis–specific Activities of Daily Living Profile score: p = 0.0006), as well as all the secondary endpoints (Myasthenia Gravis Composite score: p < 0.0001, Myasthenia Gravis Quality of Life scale—15 questions: p = 0.0025, Fatigue Severity Scale: p = 0.0061, and Carlo Besta Neurological Institute–Myasthenia Gravis scale: p = 0.0014).Conclusion:Despite the low number of patients, MuSK-001 study provided evidence that amifampridine phosphate, in the range of 30–60 mg daily dose, was safe and effective in treating muscle-specific kinase myasthenia gravis, suggesting the need for a large multi-center trial to confirm these results.

Quantitative Myasthenia Gravis Score: a Brazilian multicenter study for translation, cultural adaptation and validation.

To perform the translation, cross-cultural adaptation and validation of the Quantitative Myasthenia Gravis Score (QMGS) to Brazilian Portuguese in accordance with international ethical standards. The following steps were taken: (1) implementation of the translation protocol and transcultural adaptation, (2) validation of the adapted content, and (3) assessment of reliability. To check intra- and inter-observer reproducibility, each patient underwent two interviews with interviewer-A and one with B. The QMGS was compared to the Myasthenia Gravis Composite Scale and Myasthenia-specific Quality of Life Questionnaire. Our study group consisted of 30 patients, with a mean age of 47.6±11.4 years and a mean duration of illness of 11.33±8.49 years. Correlation between the QMGS and MGC was very strong (r = 0.928; p < 0.001) and substantial between the QMGS and MG-QOL 15 (r = 0.737; p < 0.001). The Brazilian Portuguese translation, and validation of the QMGS was successfully performed.

Brazilian-Portuguese translation, cross-cultural adaptation and validation of the Myasthenia Gravis Composite scale. A multicentric study.

To perform the translation, cultural adaptation and validation of the Myasthenia Gravis Composite (MGC) scale in Brazil. The study was conducted at three neuromuscular disease research centers in accordance with the international ethical standards, following a multi-modal approach and was conducted in three steps consisting of translation, cultural adaptation, and validation according to international guidelines. The final version of the MGC was applied in a sample of 27 MG patients and the total score was compared to a Portuguese version of the MG-QOL-15. The internal consistency verified by Cohen's Kappa test was excellent (0.766). The correlation between the MGC and MG-QOL-15 was strong (R = 0.777; p = 0.000). No significant differences were found between the responses of patients in the first and second applications of the MGC. The MGC scale, validated into Brazilian Portuguese, has proven to be a reliable instrument that is easy to use, and is highly reproducible.

Transcultural Adaptation and Validation of a Spanish Version of the Myasthenia Gravis Composite Scale (P3.131)

Transcultural Adaptation and Validation of a Spanish Version of the Myasthenia Gravis Composite Scale (P3.131)

Brazilian translation, cross-cultural adaptation and validation of the Myasthenia Gravis Composite scale and Quantitative Myasthenia gravis testing form: a multicentric study protocol.

Introduction: Myasthenia gravis (MG) is a chronic and inflammatory disease characterized by progressive weakness of the skeletal muscles due to changes in the synaptic junctions between nerves and muscle fibres. Typically, patients have a history of progressive weakness and fatigue in skeletal muscles, which occurs during sustained or repeated activities and improves after rest. Scoring systems to assess the degree of disease severity are commonly used in clinical drug trials. Further, specific instruments to assess health-related quality of life (HRQOL) have been designed to measure self-perception of symptoms and wellness during the disease or after disease treatment.HRQOL is an important component of patient evaluation, as some individuals can tolerate severe impairment and disability, while others may be very unhappy even with less severe symptom profiles. The objective of the proposed study is to translate and perform a cultural adaptation and validation of two specific instruments for assessing the clinical course of patients with MG. These instruments include the Quantitative Test for Myasthenia gravis (QMG) and Myasthenia gravis Composite Scale (MGC). Methods and analyses: The study design will follow a multi-method approach, and will be conducted in three stages consisting of translation, cultural adaptation, and validation of the QMG and MGC for Brazilian Portuguese patients. This multi-step process will consider aspects of construct and predictive validity as well as reliability and structure of the instrument. The entire process will follow the procedures proposed and developed in other linguistic and social-cultural contexts based on previous studies. Ethics: This study has received approval from the Human Research Ethics Committee of Nove de Julho University (Brazil) under process no. 360.488 based on the Helsinki declaration and is registered with the World Health Organization (WHO) under Universal Trial Number (UTN) U1111-1147-7853 and the Brazilian Registry of Clinical Trials (REBEC) RBR -7ckpdd.

Cross-sectional analysis and prospective observational study of sleep quality, lung function, ventilatory muscle strength, ventilatory patterns, autonomic nervous activity, disease severity, clinical status and quality of life in Myasthenia Gravis: a rese

Introduction: The presence of sleep disorders in Myasthenia gravis (MG) patients, can negatively affect the ventilation and the mechanics of breathing in patients with neuromuscular diseases. Some studies have shown a poor quality of sleep, excessive daytime sleepiness and the presence of sleep disordered breathing, while others did not observe a positive correlation. The aim of the study is to investigate sleep quality, lung function, ventilatory patterns, autonomic nervous activity, disease severity, clinical status and quality of life in clinically stable MG patients. Method: We propose a cross sectional analysis and prospective observational study of sleep quality, lung function, ventilatory patterns, autonomic nervous activity, disease severity, clinical status and quality of life in MG patients which will be conducted at the Sleep Laboratory of Nove de Julho University and the Neuromuscular Research Department of the Federal University of São Paulo. The design, conduct, and reporting of this study will follow the rules of The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) Statement. The protocol will includes body weight; height; body mass index; circumferences of the neck, waist, and hip; heart and respiratory rates; Mallampati index; maximum ventilatory pressures; spirometry; and standard overnight polysomnography, as well as the administration of specific questionnaires addressing the risk of obstructive sleep apnoea, excessive daytime sleepiness and HRQoL. Moreover, specific questionnaires addressing sleep apnoea; excessive daytime sleepiness; symptoms of stress, depression, anxiety, disease severity, clinical status and quality of life will be administered. The 8 outcome variables which will be measured include subjective sleepiness Epworth Sleepiness Scale (ESS), risk of OSA through The Berlin Questionnaire, stress and its manifestations by The Inventory of Symptoms of Stress for Adults (LIPP), anxiety and depression by The Hospital Anxiety and Depression Scale (HAD), general quality of life questionnaire using The Short Form 36 (SF36) and specific outcome measure MGQOL 15, MG Composite Scale and The Quantitative Myasthenia Gravis Score. Discussion: This protocol expects to identify the presence and severity of sleep disorders, changes of autonomic nervous activity, HRQoL and consequently contribute to an earlier diagnosis, thus reducing the impact on quality of life of MG patients. Trial registration: The protocol for this study is registered with the Brazilian Registry of Clinical Trials (ReBEC RBR-7ckpdd) and World Health Organization under Universal Trial Number UTN: (U1111-1147-7853).

Factors associated with depressive state in patients with myasthenia gravis: a multicentre cross-sectional study

ObjectivesThe objective of this study was to examine clinical factors associated with depressive state in patients with myasthenia gravis (MG).DesignCross-sectional study.Setting and participantsWe evaluated 287 consecutive cases of MG seen...