Physician-Reported Perspectives on Myasthenia Gravis in the United States: A Real-World Survey

Mahic, Milada; Bozorg, Ali; Decourcy, Jonathan; Golden, Keisha J.; Gibson, Gregor A.; Taylor, Christian F.; Ting, Angela; Story, Tyler J.; Scowcroft, Anna

doi:10.1007/s40120-022-00383-3

Cited by 10 publications

(7 citation statements)

References 36 publications

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“…The proportion of patients who had more than just ocular symptoms (MGFA Class II–V) increased following diagnosis and throughout the course of disease, suggesting generalization of the disease and an increase in disease severity over time. This describes the usual disease course and aligns with previous observations [ 4 , 6 , 19 ]. Despite 96% of patients being treated, the mean number of symptoms per patient overall was the same at diagnosis and at the time of survey completion, and the five most frequently reported symptoms experienced at time of survey completion were also similar to those reported at diagnosis.…”

Section: Discussionsupporting

confidence: 90%

Physician- and patient-reported perspectives on myasthenia gravis in Europe: a real-world survey

Mahic

Bozorg

Decourcy

et al. 2023

Orphanet J Rare Dis

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Background Myasthenia gravis (MG) is a rare, chronic, debilitating, unpredictable, and potentially life-threatening neuromuscular disease. There is a lack of real-world data on disease management that could be used to further understand and address unmet patient needs and burden. We aimed to provide comprehensive real-world insights in the management of MG in five European countries. Methods Data were collected using the Adelphi Real World Disease Specific Programme™ in MG, a point-in-time survey of physicians and their patients with MG in France, Germany, Italy, Spain, and the United Kingdom (UK). Physician- and patient-reported clinical data were collected, including demographics, comorbidities, symptoms, disease history, treatments, healthcare resource utilization (HCRU), and quality of life outcomes. Results In total, 144 physicians completed 778 patient record forms from March to July 2020 in the UK, and from June to September 2020 in France, Germany, Italy and Spain. Mean patient age at symptom onset was 47.7 years, with a mean time from symptom onset to diagnosis of 332.4 days (10.97 months). At diagnosis, 65.3% of patients were classified as Myasthenia Gravis Foundation of America Class II or above. Mean number of symptoms reported at diagnosis per patient was five, with ocular myasthenia reported in at least 50% of patients. At time of survey completion, the mean number of symptoms reported per patient was five and ocular myasthenia and ptosis were each still present in more than 50% of patients. Acetylcholinesterase inhibitors were the most commonly prescribed chronic treatments in all countries. Of 657 patients treated with chronic treatment at the time of the survey, 62% continued to experience moderate-to-severe symptoms. On average, 3.1 healthcare professionals (HCPs) were involved in patient management, 6.2 consultations were made per patient with any HCP over the last 12 months, and 178 (22.9%) patients were hospitalized in the last 12 months. Overall, HCRU and disease management were similar across all countries. Conclusions Our findings demonstrated the high burden of MG despite current treatment options for patients with MG.

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Section: Discussionsupporting

confidence: 90%

Physician- and patient-reported perspectives on myasthenia gravis in Europe: a real-world survey

Mahic

Bozorg

Decourcy

et al. 2023

Orphanet J Rare Dis

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“…Early control of symptoms will depend on a combination of timely diagnosis and subsequent prompt implementation of effective treatment. The length of time between the onset of MG symptoms and diagnosis is likely to vary widely between individuals; surveys in Europe conducted between 2018 and 2020 reported mean times ranging from approximately 11 months to 2.1 years 21,24,25 . Along with acetylcholinesterase inhibitors, most patients are likely to be treated initially with corticosteroids and subsequently with nonsteroidal ISTs.…”

Section: Discussionmentioning

confidence: 99%

Efficacy of ravulizumab in patients with generalized myasthenia gravis by time from diagnosis: A post hoc subgroup analysis of the CHAMPION MG study

Howard,

Vu,

Mantegazza

et al. 2024

Muscle and Nerve

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Introduction/AimsThe CHAMPION MG study demonstrated that ravulizumab significantly improved Myasthenia Gravis‐Activities of Daily Living (MG‐ADL) and Quantitative Myasthenia Gravis (QMG) total scores versus placebo in adults with acetylcholine receptor antibody‐positive generalized myasthenia gravis (AChR+ gMG). This post hoc analysis aimed to assess these outcomes by time from MG diagnosis.MethodsChanges from baseline to week 26 in MG‐ADL and QMG total scores were analyzed by time from MG diagnosis to study entry (≤2 vs. >2 years). Within each subgroup, least‐squares (LS) mean changes for ravulizumab and placebo were compared using mixed models for repeated measures.ResultsIn ravulizumab‐treated patients, differences in LS mean (standard error of the mean) changes from baseline to week 26 were not statistically significant in the ≤2‐years subgroup versus the >2‐years subgroup for MG‐ADL (−4.3 [0.70] vs. −2.9 [0.37]; p = .0511) or QMG (−4.3 [0.94] vs. −2.5 [0.50]; p = .0822) scores. No clear trends were observed in the placebo group. LS mean changes from baseline were significantly greater for ravulizumab versus placebo in both the ≤2 and >2 years from diagnosis subgroups for MG‐ADL and QMG scores (all p < .05). The difference in treatment effect between the ≤2‐years and >2‐years subgroups was not statistically significant. No clinically meaningful between‐subgroup differences in treatment‐emergent adverse events were observed in ravulizumab‐treated patients.DiscussionRavulizumab treatment improved clinical outcomes for patients with AChR+ gMG regardless of time from diagnosis. A numerical trend was observed favoring greater treatment effect with earlier versus later treatment after diagnosis. Further studies are required for confirmation.

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“…Patients describe MG as an “invisible illness” and may find it difficult to explain their challenges to others, which increases the feelings of frustration, loneliness and depression [ 6 ], and such feelings can be exacerbated by fatigue [ 5 , 6 , 9 ]. In a study of real-world clinical practice conducted in the United States, fatigue was included in the five most “troublesome” symptoms for patients with MG together with diplopia, ocular myasthenia, ptosis and weakness in the legs, as reported by physician-completed questionnaires [ 10 ]. In a recent study assessing daily activities of patients with MG, general fatigue was identified as one of the most salient symptoms, as reported by the patients [ 11 ].…”

Section: Introductionmentioning

confidence: 99%

Improvement of fatigue in generalised myasthenia gravis with zilucoplan

Weiss,

Freimer,

Leite

et al. 2024

J Neurol

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Background Fatigue is a debilitating symptom of myasthenia gravis (MG). The impact of fatigue on MG can be assessed by Quality of Life in Neurological Disorders (Neuro-QoL) Short Form Fatigue scale. Transformation of raw Neuro-QoL fatigue scores to T-scores is a known approach for facilitating clinical interpretation of clinically meaningful and fatigue severity thresholds. Methods In the Phase 3, double-blind, placebo-controlled RAISE study (NCT04115293), adults with acetylcholine receptor autoantibody-positive generalised MG (MG Foundation of America Disease Class II–IV) were randomised 1:1 to daily subcutaneous zilucoplan 0.3 mg/kg or placebo for 12 weeks. Patients completing RAISE could opt to receive zilucoplan 0.3 mg/kg in an ongoing, open-label extension study, RAISE-XT (NCT04225871). In this post-hoc analysis, we evaluated the long-term effect of zilucoplan on fatigue in RAISE patients who entered RAISE-XT. We report change in Neuro-QoL Short Form Fatigue T-scores and fatigue severity levels from RAISE baseline to Week 60. Results Mean Neuro-QoL Short Form Fatigue T-scores improved from baseline to Week 12 in the zilucoplan group (n = 86) with a clinically meaningful difference versus placebo (n = 88; least squares mean difference: − 3.61 (nominal p-value = 0.0060]), and these improvements continued further to Week 60. At Week 12, more patients on zilucoplan (n = 34, 47.2%) experienced improvements in ≥ 1 fatigue severity level from baseline versus placebo (n = 23, 28.4%; p = 0.017). At Week 60, most (n = 55, 65.5%) patients had mild fatigue or none. Conclusion Treatment with zilucoplan demonstrated statistical and clinically meaningful improvements in fatigue scores and severity versus placebo during RAISE, which were sustained to Week 60 in RAISE-XT.

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Physician-Reported Perspectives on Myasthenia Gravis in the United States: A Real-World Survey

Cited by 10 publications

References 36 publications

Physician- and patient-reported perspectives on myasthenia gravis in Europe: a real-world survey

Physician- and patient-reported perspectives on myasthenia gravis in Europe: a real-world survey

Efficacy of ravulizumab in patients with generalized myasthenia gravis by time from diagnosis: A post hoc subgroup analysis of the CHAMPION MG study

Improvement of fatigue in generalised myasthenia gravis with zilucoplan

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