Pratique Neurologique - FMC, Год журнала: 2024, Номер 15(4), С. 215 - 222
Опубликована: Ноя. 1, 2024
Journal of Neurology, Год журнала: 2024, Номер 271(9), С. 6209 - 6219
Опубликована: Июль 30, 2024
Eculizumab, a complement active antibody, and efgartigimod, an Fc fragment that blocks neonatal receptor, are both approved to treat generalized myasthenia gravis (gMG) patients. The objective of this study is describe the clinical response treatments in real-life setting.
Язык: Английский
Процитировано
15
European Journal of Neurology, Год журнала: 2024, Номер 31(7)
Опубликована: Март 24, 2024
Regular and consistent disease assessment could provide a clearer picture of burden in generalised myasthenia gravis (gMG) improve patient care; however, the use tools practice lacks standardisation. This modified Delphi approach was taken to review current evidence on tool gMG develop expert-derived consensus recommendations for good practice.
Язык: Английский
Процитировано
12
BioDrugs, Год журнала: 2025, Номер unknown
Опубликована: Янв. 27, 2025
Язык: Английский
Процитировано
1
Journal of Neurology, Год журнала: 2024, Номер 271(9), С. 6114 - 6126
Опубликована: Июль 25, 2024
The phase 3 REGAIN study and its open-label extension demonstrated the efficacy of complement C5 inhibitor eculizumab in patients with treatment-refractory, acetylcholine receptor antibody-positive generalized myasthenia gravis (gMG). aim ELEVATE was to assess effectiveness clinical practice adults MG United States.
Язык: Английский
Процитировано
4
Frontiers in Neurology, Год журнала: 2025, Номер 15
Опубликована: Янв. 15, 2025
Myasthenia Gravis (MG) is a common neuromuscular junction disorder that primarily mediated by anti-acetylcholine receptor antibodies (AChR-Ab). However, using AChR-Ab titers to predict MG severity and improvement remains controversial. This study aims explore the relationship between rate of change (RR-AChR-Ab, %) scores. We used prospective approach, included 62 patients with generalized (GMG) who were positive for AChR-Ab. measured titers, MGFA-QMGS, MG-ADL scores at baseline (before treatment) 3 6 months into treatment. Pearson Spearman correlation analyses relationships changes in rates change, (1) At baseline, there was no age, duration illness, gender, MGFA classification, or presence thymic abnormalities. (2) The trend decreasing matched reduced QMGS ADL (3) Six treatment,there titer (4) Three treatment, RR-AChRAb showed same time point. found decreased after standardized treatment consistent reductions Additionally, did reflect improvements activities daily living patients.
Язык: Английский
Процитировано
0
EMJ Neurology, Год журнала: 2025, Номер unknown, С. 2 - 7
Опубликована: Янв. 16, 2025
There have been great advances in the field of generalised myasthenia gravis (gMG) recent years, but many patients remain dissatisfied with their disease state despite being on treatment. is a growing consensus among specialists that minimising symptom burden, sustained treatment efficacy, best way to improve outcomes. gMG chronic autoimmune disorder, characterised by fluctuating muscle weakness. Traditional treatments focus immunosuppression, often face challenges efficacy and side effects, including corticosteroid complications. Up 20% do not respond standard still experience symptoms. In this key opinion leader article, Carlos Casasnovas, Bellvitge University Hospital, Barcelona, Spain; Kristl Claeys, Department Neurology, Hospitals Leuven, Belgium; John Vissing, Copenhagen Neuromuscular Centre, Rigshospitalet, Denmark, share expert knowledge past, present, future management. Speaking EMJ October November 2024, they discussed how ongoing symptoms can an important impact daily activities quality life affected patients, achieving traditional approaches, emerging novel therapies may help address critical unmet medical need.
Язык: Английский
Процитировано
0
Frontiers in Neurology, Год журнала: 2025, Номер 16
Опубликована: Апрель 2, 2025
Background There are indeed several studies addressing the severity of Coronavirus disease 2019 (COVID-19) infection in myasthenia gravis (MG) patients. However, data on post-COVID-19 sequelae MG patients remain limited. To address this gap, we collected clinical condition and prognosis with COVID-19 infection, aiming to investigate factors influencing both occurrence at 1 12 months after recovery. Method This was a retrospective analysis 150 from November 1, 2022 March 2023 First Affiliated Hospital Guangzhou University Chinese Medicine, including patient demographics, characteristics, sequelae. Multivariable binary logistic linear regression models were employed ascertain variables severity. The evolution analyzed using McNemar's test. Result In patients, 128 (85.3%) presented 23 (18%) hospitalized. associated daily corticosteroid dose (OR = 1.08, p 0.02) frequency crises pre-COVID-19 ( b 7.8, t 2.14, 0.035). Compared normal more likely experience such as insomnia, myalgia, dizziness, cough, expectoration, sore throat Among these, prevalence rash, vision impairment significantly higher. Conclusion prone developing severe which is pre-COVID-19. Additionally, they experiencing sequelae, throat,
Язык: Английский
Процитировано
0
medRxiv (Cold Spring Harbor Laboratory), Год журнала: 2025, Номер unknown
Опубликована: Апрель 16, 2025
Accurate prediction of patient outcomes in clinical trials is crucial for timely assessment treatment efficacy. This study introduces a novel approach to predict response by constructing temporal trajectories from longitudinal data. We aim extrapolate these forecast individual and identify when new patients align with established patterns. Utilizing data the MGTX trial myasthenia gravis patients, we evaluate predictability discuss potential confounding factors. Furthermore, our analysis yields an automatic clustering based on success, revealing associations age smoking status.
Язык: Английский
Процитировано
0
PLoS ONE, Год журнала: 2025, Номер 20(4), С. e0322168 - e0322168
Опубликована: Апрель 22, 2025
Background The therapeutic landscape in generalized myasthenia gravis (gMG) has been continuously evolving recent years, with over five products approved, each different efficacy, safety, and administration profiles. With the availability of new targeted treatments, physicians are faced challenge decision-making tailored to traditional goals, individual preferences, personal experience, seeking optimal disease control a positive safety profile. In this context uncertainty multiple novel choices, study aims provide insights into preferred treatment choices neurologists managing gMG identify demographic, professional or behavioral factors influencing process. Methods This is non-interventional, cross-sectional, web-based involving 150 treating patients collaboration Spanish Society Neurology. primary endpoint will be assess preferences for attributes using conjoint analysis create hypothetical scenarios. Therapeutic inertia, described as lack initiation intensification when goals not met, evaluated secondary through 7 case scenarios simulating real clinical practice situations. Neurologists also answer survey composed professional, characteristics (user resistance behavior, care-related regret, burnout, risk attitude, empathy, work fulfilment, personality traits) recognize possible decisions. Conclusions findings contribute better understanding under changing options, which have role affecting those
Язык: Английский
Процитировано
0
Muscle & Nerve, Год журнала: 2025, Номер unknown
Опубликована: Апрель 22, 2025
ABSTRACT Introduction/Aims Myasthenia gravis (MG) specific outcome measures are being used in clinical trials to evaluate therapeutic effectiveness. These validated tools also becoming a necessity practice, with payors the US market often requiring them be monitor disease state. There is considerable variation and subjectivity regarding their use. This study aimed develop consensus‐based recommendations for standardization of MG practice. Methods A panel 10 US‐based specialists developed based on three rounds formal voting using UCLA‐RAND appropriateness method after surveying myasthenia clinicians developing focus group. Results Twenty one expert consensus statements six themes were following clinician survey result review group theme development. Some key include: MGFA Clinical Classification assesses at that examination should updated intervals 3–6 months reflect current status. PIS represents overall judgment evaluator without requirement defined change scores any measure. Patient‐reported items, such as MG‐ADL MGC, referenced previous 1 week optimize recall. Additional include scoring presence co‐morbidity, physical exam findings, clarification administration measures. Discussion provided use MG‐specific findings help standardize how they
Язык: Английский
Процитировано
0