Article
Clinical Neurology
Nakul Katyal, Karen Halldorsdottir, Raghav Govindarajan, Perry Shieh, Suraj Muley, Phoebedel Reyes, Kenneth K. Leung, Jeffrey Mullen, Shadi Milani-Nejad, Manisha Korb, Namita A. Goyal, Tahseen Mozaffar, Neelam Goyal, Ali A. Habib, Srikanth Muppidi
Summary: This study aimed to describe the early experience of using efgartigimod in patients with acetylcholine receptor antibody-positive generalized myasthenia gravis. The results showed that efgartigimod led to clinically meaningful improvement in myasthenia gravis activities of daily living (MG-ADL) scores in most patients.
Article
Medicine, Research & Experimental
Sarah Dewilde, Cynthia Z. Qi, Glenn Phillips, Sergio Iannazzo, Mathieu F. Janssen
Summary: For patients with generalized myasthenia gravis (gMG), the association between symptom severity (measured with MG-ADL) and utility values is unknown. This study analyzed data from the ADAPT trial and found a significant association between MG-ADL scores and utility values in gMG patients. Furthermore, treatment with efgartigimod showed significant improvements in both symptoms and quality of life.
ADVANCES IN THERAPY
(2023)
Article
Clinical Neurology
Yukiko Ozawa, Akiyuki Uzawa, Manato Yasuda, Yuta Kojima, Yosuke Onishi, Fumiko Oda, Tetsuya Kanai, Keiichi Himuro, Naoki Kawaguchi, Satoshi Kuwabara
Summary: Approximately 60% of MG patients achieved the treatment goal within 2 years after treatment initiation. More frequent plasmapheresis and higher PSL dose within 3 months after treatment initiation were associated with difficulty in achieving the 5-mg MM status at 2 years.
JOURNAL OF NEUROLOGY
(2021)
Article
Clinical Neurology
Young-A Heo
Summary: Efgartigimod alfa is a novel and effective treatment for generalised myasthenia gravis that has shown significant and durable clinical benefits in clinical trials.
Article
Clinical Neurology
Vincenzo Di Stefano, Paolo Alonge, Nicasio Rini, Massimiliano Militello, Antonino Lupica, Angelo Torrente, Filippo Brighina
Summary: Stiff-person syndrome (SPS) is a rare autoimmune neurological disorder characterized by high levels of antibodies against glutamic acid decarboxylase (GAD), leading to impaired inhibitory neurotransmission. Efgartigimod, a new Fc receptor blocker, has shown promising results in neurological autoimmune disorders and has been approved for the treatment of AChR-seropositive generalized myasthenia gravis (MG). In this study, three patients with both MG and anti-GAD-seropositive SPS showed improvement in symptoms of both conditions after treatment with efgartigimod.
JOURNAL OF NEUROLOGY
(2023)
Article
Genetics & Heredity
Milada Mahic, Ali Bozorg, Jonathan DeCourcy, Keisha Golden, Gregor Gibson, Christian Taylor, Anna Scowcroft
Summary: Myasthenia gravis (MG) is a rare, chronic, debilitating neuromuscular disease. Real-world data from five European countries showed that MG imposes a high burden on patients despite current treatment options.
ORPHANET JOURNAL OF RARE DISEASES
(2023)
Article
Clinical Neurology
Vera Bril, James F. Howard Jr, Chafic Karam, Jan L. De Bleecker, Hiroyuki Murai, Kimiaki Utsugisawa, Peter Ulrichts, Edward Brauer, Sihui Zhao, Renato Mantegazza, Tuan Vu, ADAPT Study Grp
Summary: Efgartigimod is beneficial in improving muscle function and strength across all muscle groups in patients with gMG by reducing IgG concentration and showing significant improvements in Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scores.
EUROPEAN JOURNAL OF NEUROLOGY
(2023)
Article
Immunology
Jessica Barreto Ribeiro Dos Santos, Rosangela Maria Gomes, Michael Ruberson Ribeiro da Silva
Summary: In this study, efgartigimod was found to be an effective and safe treatment for patients with generalized myasthenia gravis who are positive for AChR antibodies. Efgartigimod reduces antibody levels and improves symptoms. The most common adverse events were headache, nasopharyngitis, nausea, and diarrhea, with urinary and upper respiratory tract infections being more frequent in efgartigimod-treated patients.
EXPERT REVIEW OF CLINICAL IMMUNOLOGY
(2022)
Article
Immunology
Shigeaki Suzuki, Akiyuki Uzawa, Hiroyuki Murai
Summary: This study provides an overview of the therapeutic strategy for generalized MG in Japan, and reviews the clinical efficacy, safety, and tolerability of efgartigimod as a promising biological drug for MG.
EXPERT REVIEW OF CLINICAL IMMUNOLOGY
(2022)
Review
Immunology
Li Jiao, Honghao Li, Shougang Guo
Summary: Eculizumab, a recombinant humanized monoclonal antibody targeting complement protein C5, has been approved by the FDA for treating AchR-positive generalized myasthenia gravis (gMG) patients. Despite demonstrating efficacy in refractory gMG patients in the REGAIN trial, there is limited information on its effectiveness in other subgroups of MG patients.
JOURNAL OF NEUROIMMUNOLOGY
(2022)
Article
Immunology
Yingkai Li, John S. Yi, James F. Howard, Manisha Chopra, Melissa A. Russo, Jeffrey T. Guptill
Summary: The study found that ECU treatment in MG patients can affect the activation state and distribution of immune cells, including decreased activation markers in memory CD4(+) T cell subsets, increased regulatory T cell populations, and reduced frequencies of Tfh subsets and migratory memory B cells.
CLINICAL IMMUNOLOGY
(2021)
Article
Clinical Neurology
Linda Kahr Andersen, Anna Sofie Jakobsson, Karoline Lolk Revsbech, John Vissing
Summary: This study explored the association between patients' satisfaction with symptoms and various disease-specific and generic outcome measures in 100 patients with generalized myasthenia gravis (gMG). Dissatisfaction with symptom levels was found to be high and correlated with disease severity, duration, depression, fatigue, and lower quality of life. The results highlight the importance of a patient-centered approach in MG treatment to optimize patient satisfaction.
JOURNAL OF NEUROLOGY
(2022)
Article
Clinical Neurology
Tuan Vu, Stephan Ortiz, Masahisa Katsuno, Djillali Annane, Renato Mantegazza, Kathleen N. Beasley, Rasha Aguzzi, James F. Howard
Summary: This study analyzed the pharmacokinetics, pharmacodynamics, and potential immunogenicity of the C5 inhibitor ravulizumab in patients with AChR Ab+ gMG. The results showed that ravulizumab can provide immediate, complete, and sustained inhibition of C5 in the treatment of these patients, supporting its use every 8 weeks.
JOURNAL OF NEUROLOGY
(2023)
Article
Clinical Neurology
Madeline Singer, Sami Khella, Shawn Bird, Paul McIntosh, Bandhu Paudyal, Anil Wadhwani, Colin Quinn, Chafic Karam
Summary: This study describes the use of efgartigimod in the treatment of gMG and finds it to be efficacious, well tolerated, and safe. Efgartigimod can be considered as an add-on therapy, a bridge therapy, or as a monotherapy for patients with difficulty tolerating other treatments.
Article
Clinical Neurology
Malin Petersson, Amalia Feresiadou, Daniel Jons, Andreea Ilinca, Fredrik Lundin, Rune Johansson, Anna Budzianowska, Anna-Karin Roos, Viktor Kagstrom, Martin Gunnarsson, Peter Sundstrom, Fredrik Piehl, Susanna Brauner
Summary: The study investigated the relationship between MG-ADL and clinical characteristics in a large Swedish nationwide cohort, finding that higher MG-ADL scores were associated with female sex, obesity, and diagnostic delay, and inversely correlated with high educational attainment. Almost half of the population reported an unsatisfactory symptom state, indicating a need for improved treatment options.