Catalyst Pharmaceuticals reports positive results from MuSK-MG trial of Firdapse to treat MG
Catalyst Pharmaceuticals has reported positive top-line results from the MuSK-MG clinical trial of Firdapse to treat patients with myasthenia gravis (MG).
Conclusion: The decreased mitochondrial membrane potential and mitophagy in Tregs in the MG group may be related to a decreased inhibition of Treg proliferation. The mitochondrial membrane potential was increased after adding the autophagy agent Rapa to enhance mitophagy, and the proliferation inhibition function of Tregs was also enhanced. The autophagy agent 3-MA down-regulated mitophagy, which decreased the mitochondrial membrane potential and inhibitory effect of Tregs. These results reveal the possible cellular immune mechanism of Treg dysfunction in MG.
Publication date: Available online 29 March 2020Source: Journal of Clinical NeuroscienceAuthor(s): Paulo José Lorenzoni, Cláudia Suemi Kamoi Kay, Raquel Cristina Arndt, Nyvia Milicio Coblinski Hrysay, Renata Dal-Pra Ducci, Otto H. Jesus Fustes, Ana Töpf, Hanns Lochmüller, Lineu Cesar Werneck, Rosana Herminia Scola
ConclusionsIn Japanese LEMS patients, the long ‐term neurological outcome is generally favorable. Our results also suggest that even in LEMS with SCLC patients, successful treatment for their cancer could result in sustained improvement of neurological symptoms.
With interest we read the article by Sugimoto et al. about a retrospective study on the “initial deterioration” of 51 patients with myasthenia gravis receiving 750–3000 mg/cycle intravenous methyl-prednisolone (IVMP) . “Initial deterioration” was qualitatively and quantitatively defined, quantitatively as increase of two or more points on the myasthenia gravis activities of daily living (MG-ADL) score . It was concluded that IVMP can be repeated after “initial deterioration” during a first cycle and that IVMP may lead to clinical improvement .
CONCLUSION: The findings of this study indicates that late Masaoka-Koga staging and histology types are significantly associated with extended overall survival. Similarly, surgical resection and multimodality treatments play a significant role in thymic malignancies neoplasms therapy strategies to prolong survival rates. PMID: 32212790 [PubMed - in process]
Corona Virus Disease 2019 (COVID-19) is a new illness caused by a novel coronavirus, severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). Symptoms are variable but typically include fever, cough, respiratory symptoms, diarrhea, reduction of smell and taste sensation. Severity ranges from mild to severe and the virus may lead to pneumonia, acute respiratory distress syndrome and death, in some patients. Nearly every country in the world has been affected by this virus and is currently defined as a pandemic, by the World Health Organization.
We report a case of pembrolizumab-induced myasthenia gravis that occurred in an 84-year-old Japanese female with metastatic urothelial carcinoma in multiple organs. She developed right ptosis 3 days after the second pembrolizumab treatment. Although prednisolone was administered, her symptoms did not change and dysphagia appeared. She needed the steroid pulse therapy for treatment eventually. On the other hand, 9 weeks after the first pembrolizumab treatment, reductions in the sizes of liver and adrenal metastases was observed. However, unfortunately, the severe immune-related adverse events did not allow her to...
Conclusions: RAMIT with simultaneous bilateral thoracoscopy is a feasible approach that may allow for enhanced visualization and more complete thymic resection compared to existing unilateral minimally invasive operations. Comparative studies and long-term follow up are needed to adequately assess the potential benefits of RAMIT. PMID: 32190361 [PubMed]
Conclusion S ingle-utility port VATS extended thymectomy is a safe and feasible minimally invasive procedure for MG. During the perioperative period, special attention should be paid to prevent myasthenic crisis, pulmonary complications, and incision complications. DOI: 10.3779/j.issn.1009-3419.2020.03.04
ConclusionsAge, Masaoka stage, and recurrence were prognostic factors of OS. Presence of bulbar symptoms was an independent risk factor for POMC. Age and World Health Organization classification influence the postoperative effect of MG.Key pointsSignificant findings of the studyAge, Masaoka stage, and recurrence were prognostic factors of OS for MG with thymomas. The presence of bulbar symptoms was an independent risk factor for POMC. Age and World Health Organization classification may influence the postoperative effect of MG.What this study addsOur study had a relatively large sample size of MG patients with thymomas onl...