MG Symptoms Weigh on Life Quality, Particularly for Women
People with myasthenia gravis, particularly women, have a high symptom burden that considerably affects their health-related quality of life, but the disease’s human cost is rarely given adequate study, a systematic literature review has found.
Symptoms generally eased following treatment and disease remission. Still, there is a need “to better understand the impact of disease from the perspective of the patient,” the review’s scientists wrote, noting that “while the clinical manifestations of myasthenia gravis (MG) are well understood, its humanistic impact is not.”
The review, “The humanistic burden of myasthenia gravis: A systematic literature review,” was published in the Journal of the Neurological Sciences.
MG is an autoimmune disease characterized by the production of self-reactive antibodies that attack the connection between nerve and muscle cells, called the neuromuscular junction. This leads to muscle weakness and fatigue, causing disability and affecting quality of life.
Few high-quality studies have looked into MG outcomes from the patients’ perspective, the scientists noted, and those that did lack standardization.
“Humanistic impacts of disease … are also not routinely discussed in clinic,” they added.
To understand how the disease and its treatments affect patients and caregivers, scientists at Argenx, which markets Vyvgart (efgartigimod) for generalized MG, and colleagues at a research consulting firm reviewed studies into adult patients published between January 2009 and December 2019 in the MEDLINE and Embase databases.
Two independent researchers screened for potential studies and out of a list of 658 studies, 67 were reviewed in detail. Fourteen of these studies reported data on psychological symptoms like depression, anxiety, fatigue, and sleep quality; 21 addressed health-related quality of life (HRQoL); and 40 reported on treatment tolerability.
Although the tools used to analyze psychological symptoms varied across studies, in general, patients with more severe symptoms and requiring longer hospitalizations showed higher levels of depression and anxiety.
Depression and anxiety symptoms were found to be more severe in patients with antibodies targeting muscle-specific kinase (MuSK) compared with those having anti-acetylcholine receptor (AChR) antibodies. They also affected women more often than men.
Fatigue and sleep issues eased when MG severity lessened or patients went into remission.
MG patients experience a worse HRQoL than the general population, with poorer results seen in MG patients with AChR antibodies and among those with refractory, or treatment resistant, disease. HRQoL scores generally improved following treatment that included eculizumab (Soliris), amifampridine (Firdapse), and efgartigimod (Vyvgart), the study noted.
According to the researchers, “therapeutic options that improve the humanistic outcomes for these populations are particularly vital.”
A total of 40 studies addressed treatment safety and tolerability. Adverse events varied greatly, from 1.8% to 52.3% depending on the study design and population. These occurred in less than 20% of patients and serious adverse events in less than 5%.
Immunosuppressant use was associated with excessive weight gain (46.6%), gastrointestinal side effects (60%), infections (52%), and indigestion (46.7%). Intravenous immunoglobulins were associated with headaches (77.3%), injection site reactions (63.6%), and nervous system disorders (42.9%).
Adverse events associated with corticosteroids were rare and included fractures (4.9%), gastrointestinal bleeds or disorders (3.0%), osteoporosis or weak bones (13.6%), and nervous system disorders (30%).
Treatment discontinuations varied across studies, but a high proportion of patients stopped treatment due to lack of efficacy.
Overall, this systematic review suggests that “symptoms associated with MG get better with disease improvement and remission.”
However, “additional options in efficacious therapy that adequately address the disease-related symptoms and also improve HRQoL may contribute to beneficial outcomes in a greater number of patients with MG,” they wrote.
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