This study provides Class IV evidence that for patients with anti-MuSK MG, rituximab increased the probability of a favorable outcome.

The MG-QOL15r is now the preferred HRQOL instrument for MG because of improved clinimetrics and ease of use. This revision does not negate previous studies or interpretations of results using the MG-QOL15. Muscle Nerve 54: 1015-1022, 2016.

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Bulbar impairment represents a hallmark feature of Amyotrophic Lateral Sclerosis (ALS) that significantly impacts survival and quality of life. Speech and swallowing dysfunction are key contributors to the clinical heterogeneity of ALS and require well-timed and carefully coordinated interventions. The accurate clinical, radiological and electrophysiological assessment of bulbar dysfunction in ALS is one of the most multidisciplinary aspects of ALS care, requiring expert input from speech-language pathologists (SLPs), neurologists, otolaryngologists, augmentative alternative communication (AAC) specialists, dieticians, and electrophysiologists—each with their own evaluation strategies and assessment tools. The need to systematically evaluate the comparative advantages and drawbacks of various bulbar assessment instruments and to develop integrated assessment protocols is increasingly recognized. In this review, we provide a comprehensive appraisal of the most commonly utilized clinical tools for assessing and monitoring bulbar dysfunction in ALS based on the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) evaluation framework. Despite a plethora of assessment tools, considerable geographical differences exist in bulbar assessment practices and individual instruments exhibit considerable limitations. The gaps identified in the literature offer unique opportunities for the optimization of existing and development of new tools both for clinical and research applications. The multicenter validation and standardization of these instruments will be essential for guideline development and best practice recommendations.

Myasthenia gravis (MG) is the prototypical autoimmune disorder caused by specific autoantibodies at the neuromuscular junction. Broad-based immunotherapies, such as corticosteroids, azathioprine, mycophenolate, tacrolimus, and cyclosporine, have been effective in controlling symptoms of myasthenia. While being effective in a majority of MG patients many of these immunosuppressive agents are associated with long-term side effects, often intolerable for patients, and take several months to be effective. With advances in translational research and drug development capabilities, more directed therapeutic agents that can alter the future of MG treatment have been developed. This review focuses on the aberrant immunological processes in MG, the novel agents that target them along with the clinical evidence for efficacy and safety. These agents include terminal complement C5 inhibitors, Fc receptor inhibitors, B cell depleting agents (anti CD 19 and 20 and B cell activating factor [BAFF)]inhibitors), proteosome inhibitors, T cells and cytokine based therapies (chimeric antigen receptor T [CART-T] cell therapy), autologous stem cell transplantation, and subcutaneous immunoglobulin (SCIG). Most of these new agents have advantages over conventional immunosuppressive treatment (IST) for MG therapy in terms of faster onset of action, favourable side effect profile and the potential for a sustained and long-term remission.

Objective:We aimed to develop a measure of myasthenia gravis impairment using a previously developed framework and to evaluate reliability and validity, specifically face, content, and construct validity.Methods:The first draft of the Myasthenia Gravis Impairment Index (MGII) included examination items from available measures enriched with newly developed, patient-reported items, modified after patient input. International neuromuscular specialists evaluated face and content validity via an e-mail survey. Test–retest reliability was assessed in stable patients at a 3-week interval and interrater reliability was evaluated in the same day. Construct validity was assessed through correlations between the MGII and other measures and by comparing scores in different patient groups.Results:The first draft was assessed by 18 patients, and 72 specialists answered the survey. The second draft had 7 examination and 22 patient-reported items. Field testing included 200 patients, with 54 patients completing the reliability studies. Test–retest reliability of the total score was good (intraclass correlation coefficient 0.92; 95% confidence interval 0.79–0.94), as was interrater reliability of the examination component (intraclass correlation coefficient 0.81; 95% confidence interval 0.79–0.94). The MGII correlated well with comparison measures, with higher correlations with the MG–activities of daily living (r = 0.91) and MG-specific quality of life 15-item scale (r = 0.78). When assessing different patient groups, the scores followed expected patterns.Conclusions:The MGII was developed using a patient-centered framework of myasthenia-related impairments and incorporating patient input throughout the development process. It is reliable in an outpatient setting and has demonstrated construct validity. Responsiveness studies are under way.

ObjectivesTo estimate patient-acceptable symptom state (PASS) cut-points for myasthenia gravis (MG) health scales.MethodsWe conducted an electronic survey that included the Myasthenia Gravis Impairment Index (MGII), EuroQol 5-Dimension (EQ5D) and a simple PASS question. PASS anchored thresholds were estimated for the MGII questionnaire through receiver operating characteristic curves. We used the MGII PASS cut-point in a validation cohort of 257 patients to estimate PASS thresholds for other clinically relevant health scales such as the quantitative myasthenia gravis score (QMGS), MG activities of daily living (MG-ADL), Myasthenia gravis composite (MGC) and myasthenia quality of life (MG-QoL15) scale.ResultsOne hundred twenty-four of approximately 250 invited patients answered the electronic survey (49% response rate), and 80 considered their current symptom state acceptable (PASS-positive). These had lower MGII scores than PASS-negative patients (7.76 ± 9.37 vs 25.0 ± 13.7, p < 0.0001) and better EQ5D scores (0.86 ± 0.17 vs 0.69 ± 0.18, p < 0.0001). The MGII questionnaire threshold for PASS was ≤10 points. Using this threshold in an independent dataset of 257 patients, all patients in remission or minimal manifestation status were PASS-positive. In addition, some patients in Classes I, II and IIIA also achieved PASS status. PASS thresholds for the QMGS, MG-ADL, MGC and MG-QoL15 were ≤7, 2, 3 and 8 points, respectively.ConclusionsWe have estimated thresholds for commonly-used myasthenia health scales reflecting patient-acceptable states in myasthenia patients. These thresholds indicate a global state—“being well” —rather than a change in scores —or “being better.” Therefore, PASS thresholds can be used as secondary endpoints for myasthenia research.

QMGS MCID values provide clinically relevant information and are recommended in MG trials. MCID analysis shows that improvement in MG patients treated with IVIg reflects clinically meaningful changes.