Correlation between myasthenia gravis−activities of daily living (MG‐ADL) and quantitative myasthenia gravis (QMG) assessments of anti−acetylcholine receptor antibody−positive refractory generalized myasthenia gravis in the phase 3 regain study

Vissing, John; O’Brien, Fanny; Wang, Jing Jing; Howard, James F.

doi:10.1002/mus.26152

Cited by 5 publications

(5 citation statements)

References 3 publications

(11 reference statements)

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“…We previously reported that the correlation between MG-ADL and QMG total scores was stronger when assessing response to treatment than baseline status 23 and that there was a strong correlation between scores after 26 weeks of eculizumab treatment. 24 Correlation analysis of the final OLE data confirms these observations for long-term eculizumab treatment (median treatment duration was over 2.5 years). The strong correlations between MG-ADL and QMG total scores, both for changes from eculizumab baseline to last OLE assessment and at last OLE assessment, demonstrate that the long-term effect of eculizumab is consistent between the MG-ADL and QMG assessments.…”

Section: Discussionsupporting

confidence: 55%

Consistent improvement with eculizumab across muscle groups in myasthenia gravis

Mantegazza¹,

O’Brien²,

Yountz³

et al. 2020

Ann Clin Transl Neurol

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Objective To assess whether eculizumab, a terminal complement inhibitor, improves patient‐ and physician‐reported outcomes (evaluated using the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale, respectively) in patients with refractory anti‐acetylcholine receptor antibody‐positive generalized myasthenia gravis across four domains, representing ocular, bulbar, respiratory, and limb/gross motor muscle groups. Methods Patients with refractory anti‐acetylcholine receptor antibody‐positive generalized myasthenia gravis were randomized 1:1 to receive either placebo or eculizumab during the REGAIN study (NCT01997229). Patients who completed REGAIN were eligible to continue into the open‐label extension trial (NCT02301624) for up to 4 years. The four domain scores of each of the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale recorded throughout REGAIN and through 130 weeks of the open‐label extension were analyzed. Results Of the 125 patients who participated in REGAIN, 117 enrolled in the open‐label extension; 61 had received placebo and 56 had received eculizumab during REGAIN. Patients experienced rapid improvements in total scores and all four domain scores of both the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale with eculizumab treatment. These improvements were sustained through 130 weeks of the open‐label extension. Interpretation Eculizumab treatment elicits rapid and sustained improvements in muscle strength across ocular, bulbar, respiratory, and limb/gross motor muscle groups and in associated daily activities in patients with refractory anti‐acetylcholine receptor antibody‐positive generalized myasthenia gravis.

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Section: Discussionsupporting

confidence: 55%

Consistent improvement with eculizumab across muscle groups in myasthenia gravis

Mantegazza¹,

O’Brien²,

Yountz³

et al. 2020

Ann Clin Transl Neurol

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“…37 A mean ± SD baseline QMG score of 17.1 ± 5.3 (range 6–34; that is, mild to severe MG) was reported in the phase III clinical study of eculizumab in patients ( n = 125) with refractory generalized MG who were categorized as having MGFA class II–IV disease and who had an MG activities of daily living (MG-ADL) score of ⩾6, suggesting moderate to severe impairment of activities of daily living. 29,38 Thus, in general, QMG scores reported in these studies suggest higher disability in patients with refractory MG compared with those with nonrefractory MG.…”

Section: Burden Of Refractory Myasthenia Gravismentioning

confidence: 68%

Understanding the burden of refractory myasthenia gravis

Schneider‐Gold

Hagenacker

Melzer

et al. 2019

Ther Adv Neurol Disord

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Myasthenia gravis (MG) is an autoantibody-mediated disease that compromises the acetylcholine receptors or associated structures of the postsynaptic membrane of the neuromuscular junction. This leads to impaired neuromuscular transmission and subsequent fluctuating fatigability and weakness of ocular, bulbar, and limb skeletal muscles. Over the past few decades, there have been significant advances in our understanding of the disease pathophysiology and improvements in prognosis due to intensive care medicine and immunomodulation. Despite this, an estimated 10–20% of patients with MG do not achieve an adequate response, are intolerant to conventional treatment, or require chronic treatment with intravenous immunoglobulins or plasma separation procedures. Such patients are regarded as having MG that is ‘refractory’ to treatment and may represent a distinct clinical subgroup. Because the majority of patients with MG have well-controlled disease, the burden of illness in the minority with refractory disease is poorly understood and may be underestimated. However, clinically these patients are liable to experience extreme fatigue, considerable disability owing to uncontrolled symptoms, and frequent myasthenic crises and hospitalizations. Both acute adverse effects and an increased risk of comorbidity from treatment regimens may contribute to reduced quality of life. As yet, little is known concerning the impact of refractory MG on mental health and health-related quality of life. This review aims to highlight the burden of disease and unmet needs in patients with refractory MG.

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“…A correlation between changes in patient-reported MG-ADL scores and physician-assessed QMG scores has been described previously [19,20]. In REGAIN and its open-label extension, patient-reported improvements were reflected in improvements in physician-reported outcomes assessed using QMG scoring.…”

Section: Discussionmentioning

confidence: 59%

‘Minimal symptom expression’ in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab

et al. 2020

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Background The efficacy and tolerability of eculizumab were assessed in REGAIN, a 26-week, phase 3, randomized, doubleblind, placebo-controlled study in anti-acetylcholine receptor antibody-positive (AChR+) refractory generalized myasthenia gravis (gMG), and its open-label extension. Methods Attainment of 'minimal symptom expression' was evaluated using patient-reported outcome measures of gMG symptoms [MG activities of daily living scale (MG-ADL), 15-item MG quality of life questionnaire (MG-QOL15)] at the completion of REGAIN and during the open-label extension. 'Minimal symptom expression' was defined as MG-ADL total score of 0-1 or MG-QOL15 total score of 0-3. Results At REGAIN week 26, more eculizumab-treated patients achieved 'minimal symptom expression' versus placebo [MG-ADL: 21.4% vs 1.7%; difference 19.8%; 95% confidence interval (CI) 8.5, 31.0; p = 0.0007; MG-QOL15: 16.1% vs 1.7%; difference 14.4%; 95% CI 4.3, 24.6; p = 0.0069]. During the open-label extension, the proportion of patients in the placebo/eculizumab group who achieved 'minimal symptom expression' increased after initiating eculizumab treatment and was sustained through 130 weeks of open-label eculizumab (MG-ADL: 1.7 to 27.8%; MG-QOL15: 1.7 to 19.4%). At extension study week 130, similar proportions of patients in the eculizumab/eculizumab and placebo/eculizumab groups achieved 'minimal symptom expression' (MG-ADL: 22.9% and 27.8%, respectively, p = 0.7861; MG-QOL15: 14.3% and 19.4%, respectively, p = 0.7531). The long-term tolerability of eculizumab was consistent with previous reports. Conclusions Patients with AChR+ refractory gMG who receive eculizumab can achieve sustained 'minimal symptom expression' based on patient-reported outcomes. 'Minimal symptom expression' may be a useful tool in measuring therapy effectiveness in gMG. Trial registration ClinicalTrials.gov NCT01997229, NCT02301624.

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Correlation between myasthenia gravis−activities of daily living (MG‐ADL) and quantitative myasthenia gravis (QMG) assessments of anti−acetylcholine receptor antibody−positive refractory generalized myasthenia gravis in the phase 3 regain study

Cited by 5 publications

References 3 publications

Consistent improvement with eculizumab across muscle groups in myasthenia gravis

Consistent improvement with eculizumab across muscle groups in myasthenia gravis

Understanding the burden of refractory myasthenia gravis

‘Minimal symptom expression’ in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab

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