Tobias Ruck M.D., Niklas Huntemann M.D., Menekse Öztürk M.D., Stefanie Schreiber M.D., Stefanie Lichtenberg Ph.D., Lars Masanneck M.D., Christopher Nelke M.D., Hend Ben Moussa M.D., Thomas Ulrych M.D., Marc Seifert Ph.D., Dimitrios Mougiakakos M.D., Sascha Dietrich M.D., Sven G. Meuth M.D. Ph.D.
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引用次数: 0
Abstract
In this case series, we report the first off-label use of the CD19xCD3 T-cell engager blinatumomab in two patients with generalized myasthenia gravis (MG). Refractory MG remains a major therapeutic challenge, with patients experiencing severe disability and potentially life-threatening crises despite intensive immunotherapy. This study evaluates the clinical efficacy and safety of short-term blinatumomab treatment in two patients with severe, refractory generalized MG. Both individuals had been experiencing a persistent disease burden with myasthenic crises leading to severe disability, despite multimodal immunotherapy. Following treatment with blinatumomab, both patients showed rapid and sustained clinical improvements, reflected in significant reductions in MG-specific scores (MG-ADL, QMG, and revised MG Quality of Life-15), further patient-reported outcomes, digital activity markers, and gait analyses. Laboratory findings revealed persistent B-cell depletion in patient #1, whereas patient #2 demonstrated clinical improvement and autoantibody reduction despite B-cell repopulation by day 106. Both patients experienced grade 1 cytokine release syndrome during initial treatment phases, but no neurotoxicity or severe adverse events were observed. This report underscores the potential of CD19xCD3 T-cell engagers as a promising therapeutic approach in severe autoimmune neuroimmunological disorders, warranting further investigation in clinical trials and mechanistic studies.
期刊介绍:
Molecular Therapy is the leading journal for research in gene transfer, vector development, stem cell manipulation, and therapeutic interventions. It covers a broad spectrum of topics including genetic and acquired disease correction, vaccine development, pre-clinical validation, safety/efficacy studies, and clinical trials. With a focus on advancing genetics, medicine, and biotechnology, Molecular Therapy publishes peer-reviewed research, reviews, and commentaries to showcase the latest advancements in the field. With an impressive impact factor of 12.4 in 2022, it continues to attract top-tier contributions.