Abstract
Introduction: Inflammation underlies the pathogenesis of numerous neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS). In ALS, the complement system has been implicated in the neuropathology of disease and disease progression. Pegcetacoplan, a subcutaneously administered C3 complement inhibitor, is being investigated in hematology, nephrology, and neurology. The current clinical study (NCT04579666) is investigating whether pegcetacoplan can improve survival and function in people diagnosed with apparent sporadic ALS. Objectives and Methodology: Evaluate the efficacy and safety of pegcetacoplan compared to placebo among people diagnosed with ALS in a global, multicenter, randomized, double-blind, placebo-controlled, phase 2 study. Approximately 228 patients diagnosed with apparent sporadic ALS, ≥18 years of age and with an ALS Functional Rating Scale-Revised (ALSFRS-R) score ≥30, slow vital capacity (SVC) ≥60% of the predicted value at screening, and with symptom onset within 72 weeks before screening, are eligible for enrollment. After screening, patients will be randomized 2:1 to treatment groups receiving either subcutaneous pegcetacoplan (1080 mg) or placebo twice weekly for a duration of 52 weeks. The primary efficacy endpoint is the difference in the Combined Assessment of Function and Survival (CAFS) ranked score at 52 weeks after treatment initiation. Additional, secondary functional efficacy (ALSFRS-R, percent SVC, muscle strength, quality of life, and caregiver burden) and safety endpoints will be analyzed at 52 weeks. After the placebo-controlled period, all patients will have the option to receive pegcetacoplan in an open-label period for an additional 52 weeks. Results: This ongoing study is currently enrolling participants. Conclusions: Results of this study will determine the role of complement and C3 inhibition in patients with ALS.
Highlights
Capsaicin is able to induce mast cell degranulation, an event probably related to the pathophysiology of a migraine attack
The present review study aimed to address the mechanisms of action of capsaicin and other chemical inducers in mast cell degranulation and an interaction of nerves and events that happen in the dura mater with the activation of mast cells
Conclusion: the analyzed data indicate that the polymorphisms contributed to the susceptibility to Parkinson’s disease (PD), further studies related to the polymorphisms and their relationship to PD are still needed for more ethnic groups, and early diagnosis is possible
Summary
Capsaicin is able to induce mast cell degranulation, an event probably related to the pathophysiology of a migraine attack. Methods: A literature review and an observational, descriptive and transversal data collect on treatment for acute demyelinating polyradiculoneuritis, available at DATASUS from January 2008 to December 2020, and articles available at Scielo and PubMed. Results: There were 7,917 hospitalizations, representing a total expenditure of R$ 9,392,552.04, 2009 being the year with the highest number of hospitalizations (809) and 2017 with the highest amount spent during the period (R$ 967,284.65). Methods: A literature review and an observational, descriptive and transversal data collect on surgical treatment was carried out, available from January 2008 to December 2020 and articles available in Scielo, Lilacs and PubMed. Results: There were 15,148 hospitalizations for surgical procedures for the treatment of chronic subdural hematoma, representing a total expenditure of R$ 45,365,258.21, with 2018 being the year with the highest number of hospitalizations (1,418) and with the highest amount spent during the period (R$ 4,570,334.28). Conclusions: The “Coca-Cola Bottle sign” is a classic sign of Graves’ disease, some signs, such as, unilateral and single orbital musculature involvement, may be suggestive of involvement by other etiologies, suggesting the benefit of an early expanded investigation
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