Self-Assessment: Immune Mediated Peripheral Neuropathies | Paradigm Clashes: Debate & Exploration in Choosing Traditional vs Novel Immunotherapeutics for MG-Paradigm Clashes: Dr. Goyal Presentation

Paradigm Clashes: Dr. Goyal Presentation

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The document outlines the rationale and evidence supporting the expanded use of novel immunotherapies in the management of Myasthenia Gravis (MG), particularly in non-classical cases like seronegative, juvenile, ocular, thymoma-associated, pregnancy-related, and checkpoint inhibitor-induced MG. Dr. Neelam Goyal, Clinical Professor at Stanford University, presents an analysis of these therapies, emphasizing FcRn and complement inhibitors such as efgartigimod, rozanolixizumab, eculizumab, ravulizumab, and zilucoplan.<br /><br />Seronegative MG, characterized by the absence of detectable antibodies, is proposed to still be antibody-mediated, with evidence of a complement-mediated damage pathway. Retrospective studies and case reports suggest these patients respond to immunosuppression and specific novel therapies.<br /><br />In juvenile MG, eculizumab has shown promising results in clinical trials for adolescents, with ongoing studies for other therapies. Novel treatments aim to offer improved treatment options for managing symptoms and reducing the risk of disease progression and medication toxicity.<br /><br />Ocular MG treatments focus on preventing disease generalization and managing symptoms, although controlled trial data is limited.<br /><br />In MG with thymoma and pregnancy-related cases, the document discusses the complexities of integrating novel therapies due to factors like surgery stabilization needs and safety during pregnancy, with limited trial data available.<br /><br />For MG crises, where traditional treatments may not suffice, these novel therapies are considered due to their rapid efficacy and ability to be used in varying dosing regimens.<br /><br />Lastly, early use of these therapies is hypothesized to yield better patient outcomes, supported by studies like the ELEVATE Study showing benefit in early treatment initiation. While clinical trial data is available for certain scenarios, significant gaps remain, highlighting the need for ongoing research.

Keywords

Myasthenia Gravis

immunotherapies

seronegative MG

FcRn inhibitors

complement inhibitors

novel treatments

juvenile MG

ocular MG

thymoma-associated MG

pregnancy-related MG