Clinical meaningfulness and psychometric robustness of the MG Symptoms PRO scales in clinical trials in adults with myasthenia gravis DOI Creative Commons
Antoine Regnault, Ali A. Habib,

Kristin Creel

et al.

Frontiers in Neurology, Journal Year: 2024, Volume and Issue: 15

Published: June 24, 2024

Objectives The objective of this research was to generate psychometric evidence supporting the myasthenia gravis (MG) symptoms patient-reported outcome (PRO) scales as a fit-for-purpose measure severity core MG and provide information allowing their meaningful interpretation using data from phase 3 study in MG. Methods Data MycarinG study, rozanolixizumab patients with generalized who experience moderate severe ( ClinicalTrials.gov Identifier: NCT03971422) were analyzed both classical test theory (CTT) Rasch measurement (RMT). Meaningful within-individual change group-level estimated for three Symptoms PRO anchor- distribution-based methods. Anchor-based methods used patient global impression (PGIS) (PGIC) anchors. Results Good properties shown sample 200 participants: good excellent reliability (test–retest internal consistency reliability) validity (associations between items scores within other clinical outcomes—MG ADL, QMG score, MGC MGFA classes—were expected); showed coverage continuum fit model. Triangulation method results led definition clinically within-patient improvement Muscle Weakness Fatigability (−16.67), Physical Fatigue (−20.00), Bulbar associated ranges. Benchmarks are also proposed results. Conclusion strong performance generated guide its supports use trials demonstrating benefits new treatments targeting (muscle weakness fatigability, physical fatigue, bulbar muscle weakness, respiratory ocular weakness).

Language: Английский

Application of Digital Tools and Artificial Intelligence to the Myasthenia Gravis Core Examination DOI
Marc Garbey, Quentin Lesport, Helen Girma

et al.

medRxiv (Cold Spring Harbor Laboratory), Journal Year: 2024, Volume and Issue: unknown

Published: July 19, 2024

Advances in video image analysis and artificial intelligence provide the opportunity to transform approach patient evaluation through objective digital evaluation.

Language: Английский

Citations

1
Experience of Daily Life with Generalized Myasthenia Gravis: A Qualitative Investigation and Assessment of Instrument Content Validity DOI Creative Commons
Christopher Hartford, Steven Sherman, Stella Karantzoulis

et al.

Neurology and Therapy, Journal Year: 2023, Volume and Issue: 12(6), P. 2079 - 2099

Published: Sept. 25, 2023

Generalized myasthenia gravis (gMG) is a rare autoimmune disease. Symptoms of gMG are diverse, and understanding their impact on patients limited. This qualitative study aimed to provide an in-depth exploration patients' daily experiences gMG. Published studies were reviewed identify the most important signs, symptoms, functional impacts related patient experience in Semi-structured hybrid concept elicitation interviews (allowing spontaneous generation disease concepts) cognitive debriefing (assessing validity existing assessments) conducted with clinicians adult Signs, understand which salient (i.e., reported by at least 50% patients, disturbance rating 5 or higher [10-point numeric scale]); saturation was also assessed. The conceptual model updated. Existing clinical outcomes assessments (COAs) that capture how feel, function, survive Interviews (n = 24) identified seven new signs symptoms 37 compared literature. Concept reached. Signs as (salient) shortness breath, general fatigue, muscle weakness arms, legs, neck, dysphonia, dysarthria, trouble swallowing liquids, choking, heat sensitivity. Patient-identified work life, depression, difficulty walking, grooming hair, showering, brushing teeth, eating, personal relationships, family participating social activities. Clinicians considered ocular, respiratory, swallowing, speech/talking, extremity function key manifestations Patients found outcome be conceptually relevant comprehensive. research provides holistic experienced observed clinicians. highlights range everyday lives, emphasizing humanistic unmet needs.

Language: Английский

Citations

2
Impact of Ravulizumab on Patient Outcomes and Quality of Life in Generalized Myasthenia Gravis DOI Creative Commons
Carlo Antozzi, Renato Mantegazza

Patient Related Outcome Measures, Journal Year: 2023, Volume and Issue: Volume 14, P. 305 - 312

Published: Oct. 1, 2023

Myasthenia gravis (MG) is an autoimmune ion channel disorder in which antibodies to different end-plate antigens impair neuromuscular transmission, ultimately leading muscle weakness and fatigability. In about 85% of patients with MG, autoantibodies against the acetylcholine receptor (AChR) activate complement cascade, causing damage junction. MG a chronic for standard therapies corticosteroids, immunosuppressive drugs, immunomodulation plasma exchange or intravenous immunoglobulins modify course disease, but residual burden physical, psychological, social disability highlights several unmet needs, among these need specific, targeted, well tolerated able improve patients' quality life. Complement inhibition paved way precision medicine since, first time, specific therapy targeting crucial pathogenetic step has been designed, tested, proven be effective controlled fashion. Ravulizumab represents long-acting inhibitor approved treatment generalized provide rapid, complete, sustained inhibition. improved Activity Daily Living scale other clinical parameters up 26 weeks as shown by CHAMPION trial, its open label extension, added value being administered every 8 weeks. The schedule administration likely adherence hence their introduction will considerably change traditional therapeutic strategy MG.

Language: Английский

Citations

1
THE NEUROMUSCULAR JUNCTION AND MYASTHENIC SYNDROMES DOI Creative Commons
Vasylyna Senkiv,

Paula Dominika

GRUNDLAGEN DER MODERNEN WISSENSCHAFTLICHEN FORSCHUNG, Journal Year: 2024, Volume and Issue: unknown

Published: May 24, 2024

The neuromuscular junction (NMJ) is a specialized synapse in which motor neurons communicate with skeletal muscle fibers, facilitating voluntary contraction. Dysfunction at the NMJ can result disorders such as myasthenia gravis, Lambert-Eaton myasthenic syndrome, and botulism characterized by impaired transmission. Diagnosis management of pathologies involve combination clinical assessments, serological testing, symptomatic treatment. This article examines etiology, pathophysiology, manifestations, diagnosis, conditions affecting nervous system muscles, to enhance understanding these practice.

Language: Английский

Citations

0
Clinical meaningfulness and psychometric robustness of the MG Symptoms PRO scales in clinical trials in adults with myasthenia gravis DOI Creative Commons
Antoine Regnault, Ali A. Habib,

Kristin Creel

et al.

Frontiers in Neurology, Journal Year: 2024, Volume and Issue: 15

Published: June 24, 2024

Objectives The objective of this research was to generate psychometric evidence supporting the myasthenia gravis (MG) symptoms patient-reported outcome (PRO) scales as a fit-for-purpose measure severity core MG and provide information allowing their meaningful interpretation using data from phase 3 study in MG. Methods Data MycarinG study, rozanolixizumab patients with generalized who experience moderate severe ( ClinicalTrials.gov Identifier: NCT03971422) were analyzed both classical test theory (CTT) Rasch measurement (RMT). Meaningful within-individual change group-level estimated for three Symptoms PRO anchor- distribution-based methods. Anchor-based methods used patient global impression (PGIS) (PGIC) anchors. Results Good properties shown sample 200 participants: good excellent reliability (test–retest internal consistency reliability) validity (associations between items scores within other clinical outcomes—MG ADL, QMG score, MGC MGFA classes—were expected); showed coverage continuum fit model. Triangulation method results led definition clinically within-patient improvement Muscle Weakness Fatigability (−16.67), Physical Fatigue (−20.00), Bulbar associated ranges. Benchmarks are also proposed results. Conclusion strong performance generated guide its supports use trials demonstrating benefits new treatments targeting (muscle weakness fatigability, physical fatigue, bulbar muscle weakness, respiratory ocular weakness).

Language: Английский

Citations

0