Journal of Clinical Neuromuscular Disease最新文献

Objectives: To evaluate the impact of treatment with eculizumab, a terminal complement inhibitor, on academic and employment status in patients with refractory generalized myasthenia gravis (MG).

Methods: Case review of 7 US patients.

Results: Six patients were aged ≤65 years; one was a full-time student and the remainder were in employment before MG diagnosis. After diagnosis, all patients gave up work (n = 3) or reduced their study/working hours (n = 4). In the 12 months after eculizumab initiation, patients who had stopped work resumed working in some capacity, whereas those who had changed their work/study hours returned to their original work/study pattern. Patients also experienced a reduction in the number of MG exacerbations, and a clinically significant improvement in MG-Activities of Daily Living scores, and were able to reduce other MG medications.

Conclusions: These results suggest that treatment with eculizumab may help maintain education/employment activity in patients with refractory generalized MG.

Objectives: To evaluate the utility of repetitive nerve stimulation test (RNS) for differentiating multifocal motor neuropathy (MMN) and progressive muscular atrophy (PMA).

Methods: We retrospectively enrolled 20 patients with MMN or PMA. We extracted the results of the initial 3-Hz RNS in the ulnar and accessory nerves and compared the percentage and frequency of abnormal decremental responses between both groups.

Results: RNS was performed in 8 ulnar and 9 accessory nerves in patients with MMN, and in 8 ulnar and 10 accessory nerves in patients with PMA. Patients with MMN had a significantly lower decrement percentage (0.6 ± 4.0% in MMN vs. 10.3 ± 6.5% in PMA, P < 0.01) and frequency of abnormal decremental response (0 of 9 in MMN vs. 6 of 10 in PMA, P = 0.01) than patients with PMA in the accessory nerve.

Conclusions: The RNS has clinical utility for differentiating MMN from PMA.

Abstract: This update covers a number of treatment topics starting with Fc receptor inhibitors and the Federal Drug Administration approval of efgartigimod. Some uncertainties regarding the use of corticosteroids are addressed, namely the risk of exacerbation with initiation of treatment and how to taper. The presence and potential importance of antibody overshoot following plasmapheresis is noted and the evolving increase in usefulness of acetylcholine receptor antibodies in diagnosing ocular myasthenia. Several recent series and case reports regarding coronavirus 2019 and myasthenia gravis are reviewed. The topics of myasthenia gravis and pregnancy, and another look at thymectomy in MG are provided. Finally, a couple of case reports on Lambert-Eaton myasthenic syndrome concentrate on the ice pack test and an autoantibody association with paraneoplastic cerebellar degeneration and Lambert-Eaton myasthenic syndrome in the same patient.

Abstract: Multifocal motor neuropathy is a rare, immune-mediated motor neuropathy with asymmetric, often debilitating progressive weakness. The efficacy of intravenous immunoglobulin in this disease is well established; however, the response typically wanes over time. No other agent has shown similar therapeutic efficacy. We describe a case of anti-ganglioside GM1 IgM-positive multifocal motor neuropathy with typical incomplete and diminishing response to intravenous immunoglobulin over time. Sixteen years after symptom onset, rituximab was administered at 2 g/m2 over 2 weeks. No significant progression of disease has occurred over the following 10 years despite no additional treatments, including intravenous immunoglobulin, being given. Only case reports and small, mostly uncontrolled studies have reported the use of rituximab in multifocal motor neuropathy with mixed results. However, given its potential benefits and lack of an established second-line agent, treatment with rituximab may be considered in select patients with refractory multifocal motor neuropathy.