Myasthenia Gravis Composite Score Research Articles

Eculizumab in Adolescent Patients With Refractory Generalized Myasthenia Gravis: A Phase 3, Open-Label, Multicenter Study

BackgroundThis study evaluated the efficacy and safety of eculizumab, a terminal complement C5 inhibitor, in juvenile generalized myasthenia gravis (gMG). MethodsAdolescents aged 12 to 17 years with refractory anti-acetylcholine receptor (AChR) antibody-positive gMG received eculizumab (weekly induction [one to two doses of 600 mg or four doses of 900 mg] followed by maintenance doses [300 to 1200 mg] every two weeks for up to 26 weeks) in a phase 3, open-label multicenter study (NCT03759366). Change from baseline to week 26 in Quantitative Myasthenia Gravis (QMG) total score (primary end point) and secondary end points including Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score, Myasthenia Gravis Composite score, Myasthenia Gravis Foundation of America postintervention status, EuroQol 5-Dimensions (Youth) and Neurological Quality-of-Life Pediatric Fatigue questionnaire scores, as well as pharmacokinetics, pharmacodynamics, and safety, were recorded. ResultsEleven adolescents (mean ± S.D. age 14.8 ± 1.8 years) were enrolled; 10 completed the primary evaluation period. Least-squares mean changes from baseline at week 26 were −5.8 (standard error [SE] 1.2; P = 0.0004) for QMG total score and −2.3 (SE 0.6; P = 0.0017) for MG-ADL total score. Overall, the primary and all secondary efficacy end point analyses met statistical significance from the first assessment and were sustained throughout. Complete terminal complement inhibition was sustained through 26 weeks in all patients. Treatment-emergent adverse events were all mild/moderate and predominantly unrelated to eculizumab. ConclusionsEculizumab was effective in reducing disease burden and was well tolerated in adolescents with refractory AChR antibody-positive gMG.

Effect of low-intensity aerobic cycling ergometer on disease severity and depression of patients with myasthenia gravis in “Dr. Soetomo” General Hospital, Surabaya

Background and objectives. Myasthenia gravis (MG) is a chronic autoimmune disease characterized by muscle weakness. Muscle weakness leads to impairment of daily activities and leads to psychiatric symptoms, including depression. The lack of exercise methods in MG patients is a problem in rehabilitation. This study evaluates the effect of an 8-week low-intensity aerobic exercise program on Myasthenia Gravis Composite (MGC) and Beck Depression Inventory (BDI) scores in MG patients. Materials and methods. This is a randomized controlled trial conducted from May to June 2023. Twenty subjects were randomly divided into treatment group and control group. The treatment group received low-intensity aerobic exercise with cycle ergometer 3 sessions per week for 8 weeks. Both groups were informed about lifestyle changes and effective breathing exercises. MGC and BDI scores were measured and compared before and after intervention. Results. In the treatment group, MGC and BDI scores was significantly decreased with strong effect size after 8 weeks (5.4 ± 2.32 to 2.4 ± 1.84, p = 0.001, Cohen’s D = 1.57; and 10.4 ± 2.88 to 5.9 ± 2.77, p = 0.001, Cohen’s D = 1.86). In the control group, MGC and BDI scores were not significantly different (5.4 ± 2.68 to 6.0 ± 2.49, p = 0.193, Cohen’s D = 0.44; and 10.4 ± 1.71 to 9.6 ± 1.65, p = 0.247, Cohen’s D = 0.39). The MGC score strongly correlated with the BDI score (r = 0.50). Conclusion. 8 weeks of low-intensity aerobic exercise with cycling ergometer reduces disease severity and depression severity in myasthenia gravis.

Effectiveness of Therapeutic Plasma Exchange in Autoimmune Neurological Diseases in a Tertiary Care Hospital of South India

ABSTRACT Background and Objectives: Therapeutic plasma exchange (TPE) is commonly used as a treatment of certain autoimmune neurological diseases, and its main target is to remove pathogenical toxins or autoantibodies. Removed plasma with toxins and autoantibodies is replaced by crystalloids, colloids, and/or normal saline. It is more effective and cost-effective than immunoglobulins. The aim of this study was to know the effectiveness of TPE in autoimmune neurological diseases. Methods: This is a prospective study involving 376 autoimmune neurological cases, and the duration of this study was 5 years. All autoimmune neurological patients irrespective of age with complaints of weakness in limbs, respiratory distress, and acute myasthenia gravis (MG) crisis cases were included in the study. The Modified Rankin Score and MG Composite Score were used to analyze the severity of the disease, and the number of TPE procedures or cycles was planned. Results: The total number of cases included was 376 (322 newly registered cases and 54 old and relapse cases), and the total number of TPE procedures done was 1491 between the years 2017 and 2021. Male patients were 138 and female patients 238. The most number of cases were of MG, followed by Guillain–Barre syndrome. The mean number of TPE procedures done was 6.1 cycles in myasthenia crisis patients. There was a significant improvement in the patients after the treatment. The Modified Rankin Score and MG Composite Score were 1/6 and 3/50, respectively (P = 0.0321 and P = 0.0298, respectively) after completion of the TPE. Conclusion: TPE is the most effective method in cases of neurological autoimmune diseases. Most of the cases show improvement immediately after the first cycle, and more than half of the cases will be able to walk or do their routine activities after 2 or 3 cycles. It is a safe and cost-effective treatment modality with minimal side effects or complications.

Factors that impact health-related quality of life in 168 myasthenia gravis in West China: Disease severity, social support, and coping style

Background: Myasthenia gravis (MG) is associated with reduced health-related quality of life (HRQOL). This study aimed to investigate factors that impact HRQOL in MG in a patient population in West China. Methods: A series of questionnaires were completed by 168 patients to assess the relationships between HRQOL and social support and coping style. We also evaluated the contributions of clinical characteristics to HRQOL. One-way analysis of variance (ANOVA), correlation and regression analysis were conducted to identify predictors that negatively impact HRQOL in MG. Results: There were significant differences in HRQOL scores among patients with different ratio of disease cost to income each month (F = 5.831, P = 0.001) and frequency of MG symptoms (F = 9.128, P < 0.001). Spearman’s correlation analysis showed that reduced HRQOL had low correlation with Myasthenia Gravis Composite score (r = 0.461, P < 0.001), confrontation (r = 0.312, P < 0.001) and acceptance- resignation coping style (r = 0.433, P < 0.001). Stepwise regression analysis further revealed that acceptance-resignation coping style (β = 0.380, P < 0.001) and MGC score (β = 0.322, P < 0.001) were the main predictors of HRQOL. Conclusions: Our study revealed the factors that impact HRQOL in MG patients and provided the first demonstration that acceptance-resignation is the main independent predictor of poor HRQOL in MG, other than disease severity.

Efficacy and safety of iscalimab, a novel anti-CD40 monoclonal antibody, in moderate-to-severe myasthenia gravis: A phase 2 randomized study

BackgroundIncreased morbidity in many patients with myasthenia gravis (MG) on long-term immunosuppression highlights the need for improved treatments.The aim of this study is to investigate the safety and efficacy of iscalimab (CFZ533), a fully human anti-CD40 monoclonal antibody, in patients with moderate-to-severe MG receiving standard-of-care (SoC) therapies. MethodsIn this double-blind, placebo-controlled phase 2 study, symptomatic patients (n = 44) despite SoC were randomized 1:1 to receive intravenous iscalimab (10 mg/kg; n = 22) or placebo (n = 22) every 4 weeks for 6 doses in total. Patients were followed up for 6 months after the last dose. The total duration of the study was 52 weeks. ResultsIn total, 34 of 44 patients (77.3 %) completed the study. The primary endpoint, Quantitative MG score, did not change significantly between baseline and week 25 for iscalimab (median [90 % CI], −4.07 [−5.67, −2.47]) versus placebo (−2.93 [−4.53, −1.33]); however, non-thymectomized patients (n = 29) showed more favorable results (iscalimab, −4.35 [−6.07, −2.64] vs placebo, −2.26 [−4.16, −0.36]). A statistically significant difference between iscalimab and placebo groups was observed in MG Composite score (adjusted mean change: −4.19 [−6.67, −1.72]; p = 0.007) at week 13, and MG-Activities of Daily Living score (−1.93 [−3.24, −0.62]; p = 0.018) at week 21. Adverse events were comparable between the iscalimab (91 %) and placebo (96 %) groups. ConclusionIscalimab showed favorable safety and improvements compared with placebo in non-thymectomized patients with moderate-to-severe MG. It did not show any protective effect in patients with moderate-to-severe MG.

Safety and clinical activity of autologous RNA chimeric antigen receptor T-cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-randomised phase 1b/2a study

Chimeric antigen receptor (CAR) T cells are highly effective in treating haematological malignancies, but associated toxicities and the need for lymphodepletion limit their use in people with autoimmune disease. To explore the use of CAR T cells for the treatment of people with autoimmune disease, and to improve their safety, we engineered them with RNA (rCAR-T)-rather than the conventional DNA approach-to target B-cell maturation antigen (BCMA) expressed on plasma cells. To test the suitability of our approach, we used rCAR-T to treat individuals with myasthenia gravis, a prototypical autoantibody disease mediated partly by pathogenic plasma cells. MG-001 was a prospective, multicentre, open-label, phase 1b/2a study of Descartes-08, an autologous anti-BCMA rCAR-T therapy, in adults (ie, aged ≥18 years) with generalised myasthenia gravis and a Myasthenia Gravis Activities of Daily Living (MG-ADL) score of 6 or higher. The study was done at eight sites (ie, academic medical centres or community neurology clinics) in the USA. Lymphodepletion chemotherapy was not used. In part 1 (phase 1b), participants with Myasthenia Gravis Foundation of America (MGFA) disease class III-IV generalised myasthenia gravis received three ascending doses of Descartes-08 to determine a maximum tolerated dose. In part 2 (phase 2a), participants with generalised myasthenia gravis with MGFA disease class II-IV received six doses at the maximum tolerated dose in an outpatient setting. The primary objective was to establish safety and tolerability of Descartes-08; secondary objectives were to assess myasthenia gravis disease severity and biomarkers in participants who received Descartes-08. This trial is registered with clinicaltrials.gov, NCT04146051. We recruited 16 individuals for screening between Jan 7, 2020 and Aug 3, 2022. 14 participants were enrolled (n=3 in part 1, n=11 in part 2). Ten participants were women and four were men. Two individuals did not qualify due to low baseline MG-ADL score (n=1) or lack of generalised disease (n=1). Median follow-up in part 2 was 5 months (range 3-9 months). There was no dose-limiting toxicity, cytokine release syndrome, or neurotoxicity. Common adverse events were headache (six of 14 participants), nausea (five of 14), vomiting (three of 14), and fever (four of 14), which resolved within 24 h of infusion. Fevers were not associated with increased markers of cytokine release syndrome (IL-6, IL-2, and TNF). Mean improvements from baseline to week 12 were -6 (95% CI -9 to -3) for MG-ADL score, -7 (-11 to -3) for Quantitative Myasthenia Gravis score, -14 (-19 to -9) for Myasthenia Gravis Composite score, and -9 (-15 to -3) for Myasthenia Gravis Quality of Life 15-revised score. In this first study of an rCAR-T therapy in individuals with an autoimmune disease, Descartes-08 appeared to be safe and was well tolerated. Descartes-08 infusions were followed by clinically meaningful decreases on myasthenia gravis severity scales at up to 9 months of follow-up. rCAR-T therapy warrants further investigation as a potential new treatment approach for individuals with myasthenia gravis and other autoimmune diseases. Cartesian Therapeutics and National Institute of Neurological Disorders and Stroke of the National Institutes of Health.

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.

SEROPOSITIVE MYASTHENIA GRAVIS AS THE CAUSE OF MULTIPLE FAILED EXTUBATIONS AND A PROLONGED ICU STAY FOR COVID-19 PNEUMONIA: A CASE REPORT AND BRIEF LITERATURE REVIEW

SEROPOSITIVE MYASTHENIA GRAVIS AS THE CAUSE OF MULTIPLE FAILED EXTUBATIONS AND A PROLONGED ICU STAY FOR COVID-19 PNEUMONIA: A CASE REPORT AND BRIEF LITERATURE REVIEW

Leflunomide combined with low-dose prednisone inhibits proinflammatory T cells responses in myasthenia gravis patients.

We previously found that leflunomide combined with low-dose prednisone rapidly improved the clinical symptoms of myasthenia gravis (MG), but we had not investigated the mechanism of this phenomenon. This study documents the effect of leflunomide combined with low-dose prednisone on pro-inflammatory T cells in MG patients. We compared 32 treated MG patients with 18 controls. We collected peripheral blood before treatment and 4, 8, and 12 weeks after treatment. We extracted peripheral blood mononuclear cells (PBMCs) and stimulated them with phorbol 12-myristate 13-acetate (PMA) + ionomycin and quantified IFN-γ, IL-4, IL-17, and IL-9 secretion through ELISA. We quantified T helper (Th) cells Th1 (CD3+CD4+IFN-γ+), Th2 (CD3+CD4+IL-4+), Th17 (CD3+CD4+IL-17A+) and Th9 (CD3+CD4+IL-9+) among PBMCs. The treatment significantly reduced IL-17 and IL-9 secretion in peripheral blood but did not affect IFN-γ levels. Significant decreases in IL-17 and IL-9 appeared at week 12, and the trend of change was similar to that of the MG composite score. Flow cytometry indicated that leflunomide combined with low-dose prednisone significantly reduced the frequency of Th1 and Th17 cells. These findings demonstrate the potential of this treatment as an alternative immunosuppressive therapy for MG.

Clinical evaluation of efficacy of leflunomide combined with low-dose prednisone for treatment of myasthenia gravis.

This study evaluated the clinical efficacy of leflunomide combined with low-dose prednisone (0.25mg/kg/day) for treatment of myasthenia gravis (MG). We enrolled 32 MG patients treated with leflunomide combined with low-dose prednisone. In the control group, 14 patients were treated with low-dose prednisone. Improvement in MG composite (MGC) score of ≥ 3 points from enrollment to 12-week follow-up indicated that the treatment was effective. In the leflunomide combined low-dose prednisone group, the median of MGC score at the time of enrollment was 8.5 points. After 12weeks, the MGC score dropped to four points. There was statistically significant difference in MGC score before and after treatment (p < 0.001). In the low-dose prednisone group also followed up for 12weeks, the median of MGC score of the patients decreased from 7 to 4 points, and the change was not statistically significant (p = 0.05). In the leflunomide combined low-dose prednisone group, the improvement of clinical symptoms occurred mainly in the first 4weeks and the last 4weeks. Relatively, the decline of the score was mostly seen during the first 8weeks in the low-dose prednisone group. In leflunomide combined with low-dose prednisone group, the effective rate of generalized MG(gMG) was significantly higher than ocular MG(oMG) (χ2 test, p = 0.036). However, there is no significant difference in the effective rate between AChR-Ab-positive and -negative groups (Fisher's Exact Test, p = 0.625). No serious side effects were observed in any of the subjects. Leflunomide combined with low-dose prednisone rapidly improved the clinical symptoms of patients with MG. It may be a promising treatment for gMG.

Robotic thymectomy for thymoma in patients with myasthenia gravis: neurological and oncological outcomes.

The goal of this study was to analyse the outcomes in 53 patients with thymoma, 34 of whom had myasthenia gravis (MG), who were treated with robotic surgery. The oncological outcomes of the whole series of patients were analysed. Furthermore, because consistent data are not yet available in the literature, the main focus was the analysis of the neurological results of the patients affected by MG and thymoma. The clinical outcomes of 53 patients with a diagnosis of thymoma who underwent robotic thymectomy between January 2014 and December 2019 in our institution were collected and evaluated; 34 of these patients had a concomitant diagnosis of MG. The neurological status of the patients was determined from a clinical evaluation according to the Osserman classification and on pre- and post-surgery Myasthenia Gravis Composite scores, whereas neurological clinical outcomes were assessed using the Myasthenia Gravis Foundation of America Post-Intervention Score. Reduction of steroid therapy was also considered. The recurrence rate, adjuvant radiotherapy and overall survival of the patients with a thymoma were evaluated. Neurological outcomes: improvement of the clinical conditions was obtained in 26 patients (76.5%) following the operation: complete stable remission was observed in 5 patients (14.7%), pharmacological remission in 10 (29.4%) and minimal manifestation in 11 (32.3%). Four patients (11.8%) exhibited no substantial change from the pretreatment clinical manifestations or reduction in MG medication and 4 (11.8%) patients experienced worsening of clinical conditions. In 21 patients (61.7%) a reduction of the dosage of steroid therapy was obtained. Oncological outcomes: at an average follow-up of 36 months, the overall survival was 96%, 4 patients (7.5%) had pleural relapses and 12 patients (22.6%) underwent postoperative radiotherapy, according to their stage. In accordance with Masaoka staging, 34% were in stage I, 56.6% in stage II and 9.4% in stage III. Our results suggest that robotic surgical treatment of patients with thymoma and concomitant MG is effective in improving the neurological outcomes. Moreover, the oncological results obtained in this series confirm the efficacy of robotic surgery for the treatment of thymic malignancies, with results in line with those of open surgery. However, due to the indolent growth of thymomas, further observations with longer follow-up are necessary.

P-PN015. Rituximab therapy in post thymectomy refractory myasthenia gravis: A case report

Introduction . Myasthenia gravis (MG) is one of the autoimmune diseases caused by disruption of synaptic transmission at the neuromuscular junction. An estimated 10–20% patients with MG will still experience frequent relapses even after receiving the standard MG treatment with pyridostigmine and immunosuppressants. Rituximab can be considered as a therapeutic option in MG patients that show unsatisfactory response to standard therapy. Case report: A female patient, 31 years old, was diagnosed with myasthenia gravis in 2017, and tested positive for acetylcholine receptor antibody (AChR antibody). Upon diagnosis, her Myasthenia Gravis Composite (MGC) score was 22. She was administered with pyridostigmine 4x60 mg per oral (PO) and methylprednisolone 3x16 mg PO. She was hospitalized three times in two months due to MG relapses. In addition, the patient suffered an episode of myasthenic crisis with mechanical ventilator support one year after diagnosis, after which she had thymectomy. Post thymectomy, MGC score was 13, with medication pyridostigmine 4x60 mg PO, methylprednisolone 1x8 mg PO, and azathioprine 1x200 mg PO. The patient was still experiencing MG relapses and was admitted to hospital five times in two months. She was then administered rituximab. After rituximab therapy, her MGC score was 4, she only had relapse once within a year. Pyridostigmine 2x60 mg and methylprednisolone 1x8 mg PO were still continued. Results . A recent systematic review shows that rituximab administration gives satisfactory results in 72% of MuSK patient, 30% of AChR antibody patients, and 44% in combined both groups. In this patient, it was found that initial treatment with thymectomy surgery had unsatisfactory improvement, and relapses were remained relatively frequent. However, with additional treatment with rituximab, clinical improvement was significant. Conclusion . Rituximab is an effective therapeutic choice for refractory MG patients who fail to attain good disease control even after thymectomy.

Clinical experience with maintenance therapeutic plasma exchange in refractory generalized myasthenia gravis.

Despite the availability of several immunomodulatory therapies, about 20% of myasthenia gravis (MG) patients remain refractory to conventional treatments. There is limited evidence to support the use of maintenance therapeutic plasma exchange (TPE) therapy for refractory generalized MG. Retrospective chart review of 14 patients with refractory generalized MG treated for 12 months with maintenance TPE therapy. Outcome measures were myasthenia gravis composite (MGC) score, myasthenia gravis activities of daily living (MG-ADL), number of acute exacerbations, medication changes, and adverse events. Data were collected at 3 monthly intervals for 12 months before and after initiation of TPE therapy. Clinically meaningful reductions in mean MG-ADL (>2 points) (mean MG-ADL score: 9.9 ± 0.5; 12-month pre-TPE to 5.2 ± 0.9; 12-month post-TPE) and MGC (>3 points) (mean MGC score: 25.2 ± 1.6; 12-month pre-TPE to 11.7 ± 1.4; 12-month post-TPE) were observed at 3months following initiation of TPE and were maintained up to 12 months in all patients. After 12 months of TPE therapy, all patients had a significant reduction in daily prednisone and pyridostigmine use. Patients previously on IVIG or rituximab therapy were successfully weaned off both treatments. There was a significant reduction in acute MG exacerbations; 7.8 ± 1.1 mean exacerbations/patient (12-month pre-TPE) to 2 ± 1.1 mean exacerbations/patient (12-month post-TPE). Over a period of 12 months, maintenance TPE therapy improved MG-ADL, and MGC with decreased immunosuppressant requirement, while being well-tolerated.

Rituximab in Refractory Myasthenia Gravis: Experience in a Single Healthcare Center in Mexico.

Background: Ten to fifteen percent of patients with myasthenia gravis (MG) have treatment-refractory disease. In short series and case reports, rituximab has proven to be effective in refractory MG.Methods: A retrospective, longitudinal study was conducted. Recruitment was performed in an MG cohort from a single third-level healthcare center in Mexico. The selection included refractory MG patients that were treated with rituximab. Response after rituximab therapy was assessed with MG composite score (MGCS) and prednisone dose reduction at 6, 12, and 18 months after initiation. Wilcoxon signed-rank test was used to evaluate differences between related groups for non-continual variables. P<0.05 was considered statistically significant.Results: Ten patients (7%) fulfilled criteria for refractory MG, and eight of them were treated with rituximab. The mean age at MG diagnosis was 25.5 (±2) years, with a female predominance (75%). All our patients (100%) had positive acetylcholine receptor (AchR) antibodies. The median MG duration was six years (interquartile range [IQR] 4.2-6) before rituximab initiation. All patients were previously treated with azathioprine and 50% additionally with cyclophosphamide. The median prednisone doses before rituximab treatment and 18-month follow-up were 50 mg (IQR 30-50 mg) and 10 mg (IQR 0-20 mg), respectively (p=0.011). The median baseline MGCS and at 18-month follow-up were 19.5 (IQR 11-31) and 6 (IQR0-16), respectively (p = 0.012).Conclusion: Rituximab appears to be associated with clinical improvement and prednisone dose reduction in Latin-American patients diagnosed with anti-AchR MG. Our findings need to be interpreted in light of the limitations mentioned.

Disease Severity Assessment and Short-Term Outcome in Patients with Myasthenia Gravis.

Background:Myasthenia gravis (MG) is an autoimmune disorder with a chronic fluctuating course. The outcome measures encapsulate disease severity, functional impact at diagnosis, and objective evaluation of clinical benefit from therapeutic interventions.Aims and Objective:To assess the disease severity, correlation between various outcome measures, and to evaluate the short-term outcome at 3 months and 6 months in a cohort of MG patients.Materials and Methods:Quantitative myasthenia gravis (QMG) score, myasthenia gravis composite (MGC) score, and myasthenia gravis quality of life-15 (MG-QoL-15) score were applied to 54 patients at first visit, 3 months and 6 months follow-up.Results:Mean quality of life-15 (QoL-15) score at base line was 15.241. Mean QMG and MGC scores at baseline were 14.63 ± 8.37 and 15.87 ± 9.14, respectively. QMG score showed a strong positive correlation with both MGC and MG-QoL-15 scores. QMG and MGC scores showed a moderate correlation with acetylcholine receptor antibody (AChR Ab) titers. Mean QMG at follow-up was 9.95 ± 5.49 at 3 months and 6.74 ± 4.74 at 6 months. Mean MGC at follow-up was 10.75 ± 5.58 at 3 months and 6.51 ± 4.36 at 6 months.Conclusion:The combination of physician-evaluated and patient-reported outcome measures provided a more discerning picture of patient status and response to treatment. Incorporating MG outcome measures into clinical practice would aid in modulating therapies.

Cladribine in myasthenia gravis: a pilot open‐label study

The aim of this study was to assess the effectiveness of cladribine treatments in a population of patients with refractory myasthenia gravis (MG). In a prospective open-label study of cladribine in refractory MG, 13 patients received cladribine at baseline with repetitive cycles driven by clinical response. A Myasthenia Gravis Composite (MGC) score was obtained and a standard dose of steroids was administered. A total of 11 patients achieved significant clinical improvement in MGC score during their therapy. The mean MGC score declined from 15.1 to 6.3 points within 4months of observation. The dosage of prednisolone declined from 9.5 to 1.9mg. None of the patients required intravenous immunoglobulin or plasma exchange treatments and no adverse events occurred in the study period. Cladribine seems to be a safe and effective emergency therapy in a population of patients with refractory MG.

QUALITY OF SLEEP IN PATIENTS WITH MYASTHENIA GRAVIS

Myasthenia gravis (MG) is a chronic neuromuscular disease that leads to progressive weakness, fatigue of the skeletal muscles, and is often associated with psychological changes, especially with poorer quality of sleep. To evaluate the quality of sleep in patients suffering from MG in relation to sociodemographic and clinical characteristics of the disease. A total of 70 adult patients have been classified according to Myasthenia Gravis Foundation of America classification and divided into groups with regard to the age of onset, gender, employment status and type of work, presence or absence of pathological changes on thymus and presence or absence of anti-nAchr antibodies. Severity of clinical manifestations was evaluated by using quantitative MG scores and MG composite scores. Pittsburgh questionnaire was used to assess the subjective quality of sleep. In addition, Hamilton's anxiety and depression scales and questionnaires for quality of life assessment were also implemented. The results of our research show a correlation between poor quality of sleep and prolonged duration of the disease, pathological changes on thymus, positive anti-nAchr antibodies. The correlation between poor quality of sleep with more severe clinical presentation, poor quality of life, anxiety and depression was confirmed. Quality of sleep is impaired in patients with MG, especially in the case of severe clinical manifestations and prolonged duration of the disease. Considering the lack of literature on the subject, a better understanding of the prevalence and severity of sleep disorders in MG requires further research.

Safety of an Abbreviated Transition Period When Switching From Intravenous Immunoglobulin to Eculizumab in Patients with Treatment-Refractory Myasthenia Gravis: A Case Series.

Case seriesPatient: —Final Diagnosis: Generalized myasthenia gravisSymptoms: Muscle weaknessMedication: IVIg • eculizumabClinical Procedure: Transition from IVIg to eculizumabSpecialty: NeurologyObjective:Unusual setting of medical careBackground:Eculizumab is a terminal complement inhibitor used to treat myasthenia gravis in patients refractory (because of insufficient efficacy or intolerance) to other therapies, including intravenous immunoglobulin. However, information is lacking on how to transition patients from intravenous immunoglobulin to eculizumab, while avoiding a crossover effect of intravenous immunoglobulin and minimizing the risk of a transient worsening of symptoms if treatment that may be at least partially effective is interrupted. The aim of this study was to determine whether eculizumab can be safely initiated before complete intravenous immunoglobulin washout, using a standardized protocol.Case Report:A series of 13 patients with generalized treatment-refractory myasthenia gravis were transitioned to eculizumab 10–14 days after their last intravenous immunoglobulin infusion. Patients’ clinical status was assessed before and 6 weeks after transition using the Myasthenia Gravis Composite Score. Most patients (8/13; 62%) had received ≥3 immunosuppressants as well as intravenous immunoglobulin. The median (range) Myasthenia Gravis Composite Score before and 6 weeks after transition was 21 (11–29) and 12 (6–18), respectively. Clinically significant improvements (score decrease ≥3) were observed in all patients. Two patients experienced mild myalgia during transition.Conclusions:In this case series, patients with treatment-refractory myasthenia gravis were successfully transitioned to eculizumab 10–14 days after their last intravenous immunoglobulin infusion without any significant safety concerns.

Quality of Life of Myasthenia Gravis Patients in Regard to Epidemiological and Clinical Characteristics of the Disease.

Myasthenia gravis (MG) affects overall quality of life (QoL). The aim of the research was to evaluate QoL in patients suffering from MG in regard to epidemiological and clinical factors of the disease. The study included 70 patients. The severity of clinical manifestation was estimated using quantitative MG score and MG composite score. Patients were classified by using Myasthenia Gravis Foundation of America Classification. The Questionnaire of Life Quality Specific for Myasthenia Gravis-15 items (MGQOL15) is developed for QoL assessment in patients suffering from MG. In addition to the MGQOL15 revised version (MGQOL15r), a 36-item health survey of the Medical Outcomes Study Short Form (SF36) questionnaire, the Hamilton scales for the assessment of anxiety (HAM-A), and depression (HAM-D) were also used. MGQOL15r scores are negatively correlated with SF36 scores and positively correlated with scores on HAM-A and HAM-D scales (P<0.001). Patients with longer disease duration and severe clinical manifestation often manifest anxiety, depression, and have poorer QoL (P<0.05). In relation to the presence of the anti-nAchR antibody, statistically significant differences were found in relation to the summed scores on scales for assessing QoL, as well as on a HAM-A scale (P<0.05). Physical workers and unemployed patients had worse scores in relation to retired and administrators/intellectual workers on all scales (P<0.001). MGQOL15r is a life quality assessment questionnaire that could be used in routine practice in patients with MG. It is much simpler for use in these patients than SF36 and provides relevant data.

Association between musk antibody concentrations and the myasthenia gravis composite score in 3 patients: A marker of relapse?

Muscle-specific tyrosine kinase (MuSK) autoantibody related myasthenia gravis is characterized by bulbar and respiratory manifestations, a poor response to anticholinergics, and a generally good response to plasma exchange and rituximab. It is not known if MuSK-antibody (Ab) levels could be used to predict the clinical course Methods: Three patients for whom frequent long-term monitoring of MuSK-Ab levels and the Myasthenia Gravis Composite (MGC) scores were performed are described. A close relationship existed between the MuSK-Ab concentrations and the MGC score. Furthermore, a rise in Ab concentration preceded a more serious clinical relapse in all patients Conclusions: These findings suggest that MuSK-Ab concentrations may be a useful biomarker for the long-term monitoring of MuSK myasthenia gravis, particularly while in clinical remission. This may allow preemptive escalation of therapy to prevent clinical relapse, and conversely permitting greater weaning of unnecessary immunosuppression. Muscle Nerve, 2019.