Burden of illness in patients with treatment refractory myasthenia gravis

Engel-Nitz, Nicole; Boscoe, Audra; Wolbeck, Ryan; Johnson, Jonathan; Silvestri, Nicholas J.

doi:10.1002/mus.26114

Cited by 43 publications

(101 citation statements)

References 21 publications

(37 reference statements)

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“…The proportion of participants assigned to the refractory MG cohort in the present study (9%) accords with those reported from US medical and pharmacy claims (also 9%) and from a retrospective chart review in a US tertiary neuromuscular clinic (15%), despite any differences in the criteria for refractory disease. As acknowledged in a previous analysis conducted with the current study population, the proportion of women was higher (80%) when compared with other studies . This is likely to be because participants aged over 65 years were excluded, and women have an earlier mean age of MG onset …”

Section: Discussionsupporting

confidence: 81%

“…The criteria used to identify refractory disease in the present study were informed by the different definitions used in previous studies . The proportion of participants assigned to the refractory MG cohort in the present study (9%) accords with those reported from US medical and pharmacy claims (also 9%) and from a retrospective chart review in a US tertiary neuromuscular clinic (15%), despite any differences in the criteria for refractory disease. As acknowledged in a previous analysis conducted with the current study population, the proportion of women was higher (80%) when compared with other studies .…”

Section: Discussionmentioning

confidence: 83%

“…Acetylcholinesterase inhibitors, corticosteroids, and/or immunosuppressant therapies are successfully employed to alleviate MG symptoms in most patients. However, it is estimated that symptoms persist in up to 15% of those diagnosed with MG despite concerted therapeutic efforts; these individuals are considered to have refractory disease . The treatments used in patients with refractory disease, such as long‐term therapy with immunosuppressant treatments, intravenous immunoglobulin (IVIg), or plasmapheresis, may increase the burden of illness.…”

Section: Introductionmentioning

confidence: 99%

“…The treatments used in patients with refractory disease, such as long‐term therapy with immunosuppressant treatments, intravenous immunoglobulin (IVIg), or plasmapheresis, may increase the burden of illness. Patients with refractory MG are also significantly more likely than those with nonrefractory disease to experience myasthenic exacerbations and crises, and to visit an emergency department and/or be admitted to a hospital . Given the persistence of symptoms, patients with refractory MG may continue to experience problems with daily activities such as eating, personal grooming, reading, and driving.…”

Section: Introductionmentioning

confidence: 99%

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Employment in refractory myasthenia gravis: A Myasthenia Gravis Foundation of America Registry analysis

et al. 2019

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Introduction Labor‐market participation is potentially very difficult for patients with refractory myasthenia gravis (MG). In this study, employment status and work absences are compared between refractory and nonrefractory MG. Methods Adults (aged 18–64 years, all diagnosed ≥2 years previously) were included if enrolled in the Myasthenia Gravis Foundation of America Patient Registry during July 2013 to February 2018. Results Seventy‐six patients (9.2%) had refractory and 749 (90.8%) had nonrefractory disease; demographic data did not differ between groups. Relative to the nonrefractory group, the refractory group patients were more than twice as likely to work fewer hours per week (odds ratio [95% confidence interval]: currently employed, 2.777 [1.640–4.704]; employed over previous 6 months, 2.643 [1.595–4.380]), but those employed were not more likely to be absent from work. Discussion Because absence from the labor market adversely affects quality of life and personal finances, these findings reaffirm the considerable disease burden associated with refractory MG.

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Section: Discussionsupporting

confidence: 81%

Section: Discussionmentioning

confidence: 83%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Employment in refractory myasthenia gravis: A Myasthenia Gravis Foundation of America Registry analysis

et al. 2019

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“…The open‐label study not only supports and extends the body of evidence for the treatment effect of eculizumab but also provides new data regarding its positive impact on clinical burden in patients who experience persistent MG symptoms despite having a history of using multiple ISTs. Among such patients, rates of potentially life‐threatening exacerbations, with consequent hospitalizations and rescue therapy use, are typically high . We have demonstrated that eculizumab treatment significantly reduces the rates of MG exacerbations and MG‐related hospitalizations in comparison with the last complete year before entering the study and the rate of rescue therapy requirement compared with placebo‐treated patients in REGAIN.…”

Section: Discussionmentioning

confidence: 90%

Long‐term safety and efficacy of eculizumab in generalized myasthenia gravis

et al. 2019

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Introduction : Eculizumab is effective and well tolerated in patients with antiacetylcholine receptor antibody‐positive refractory generalized myasthenia gravis (gMG; REGAIN; NCT01997229). We report an interim analysis of an open‐label extension of REGAIN, evaluating eculizumab's long‐term safety and efficacy. Methods : Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients. Results : The safety profile of eculizumab was consistent with REGAIN; no cases of meningococcal infection were reported during the interim analysis period. Myasthenia gravis exacerbation rate was reduced by 75% from the year before REGAIN ( P < 0.0001). Improvements with eculizumab in activities of daily living, muscle strength, functional ability, and quality of life in REGAIN were maintained through 3 years; 56% of patients achieved minimal manifestations or pharmacological remission. Patients who had received placebo during REGAIN experienced rapid and sustained improvements during open‐label eculizumab ( P < 0.0001). Discussion : These findings provide evidence for the long‐term safety and sustained efficacy of eculizumab for refractory gMG. Muscle Nerve 2019

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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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Burden of illness in patients with treatment refractory myasthenia gravis

Cited by 43 publications

References 21 publications

Employment in refractory myasthenia gravis: A Myasthenia Gravis Foundation of America Registry analysis

Employment in refractory myasthenia gravis: A Myasthenia Gravis Foundation of America Registry analysis

Long‐term safety and efficacy of eculizumab in generalized myasthenia gravis

Consistent improvement with eculizumab across muscle groups in myasthenia gravis

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