The impact of myasthenia gravis on daily life is significant, but overlooked


People with myasthenia gravis, especially women, have a high symptom burden that significantly affects their health-related quality of life, but the human cost of the disease is rarely adequately studied, a review finds. systematics of the literature.

Symptoms generally improved after treatment and disease remission. Still, there is a need “to better understand the impact of the disease from the perspective of the patient,” the journal scientists wrote, noting that “although the clinical manifestations of myasthenia gravis (MG) are well understood, its humanistic impact is not.”

The article, “The humanistic burden of myasthenia gravis: a systematic review of the literaturewas published in the Journal of Neurological Sciences.

recommended reading

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, leading to disability and affecting quality of life.

Few high-quality studies have examined MG outcomes from the perspective of patients, the scientists noted, and those that have done so lack standardization.

“The humanistic impacts of the disease … are also not routinely discussed clinically,” 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 patient studies adults published between January 2009 and December 2019 in MEDLINE and Embase databases.

Two independent researchers screened potential studies and from a list of 658 studies, 67 were reviewed in detail. Fourteen of these studies reported data on psychological symptoms such as depression, anxiety, fatigue, and sleep quality; 21 focused on health-related quality of life (HRQoL); and 40 reported treatment tolerance.

Although the tools used to analyze psychological symptoms varied across studies, in general, patients with more severe symptoms and requiring longer hospital stays had higher levels of depression and anxiety.

Symptoms of depression and anxiety were found to be more severe in patients with antibodies targeting muscle-specific kinase (MuSK) compared to those with anti-acetylcholine receptor (AChR) antibodies. They also affected women more often than men.

Fatigue and sleep problems eased as MG severity decreased or patients went into remission.

MG patients have a worse HRQOL than the general population, with poorer outcomes in MG patients with AChR antibodies and among those with refractory or treatment-resistant disease. HRQoL scores generally improved after treatment including eculizumab (Soliris), amifampridine (Firdapse), and efgartigimod (Vyvgart), the study notes.

“Therapeutic options that improve humanistic outcomes for these populations are especially vital,” the researchers said.

A total of 40 studies looked at the safety and tolerability of the treatment. Adverse events varied widely, from 1.8% to 52.3% depending on study design and population. These occurred in less than 20% of patients and serious adverse events in less than 5%.

Use of immunosuppressants was associated with excessive weight gain (46.6%), gastrointestinal side effects (60%), infections (52%) and indigestion (46.7%). Intravenous immunoglobulins were associated with headache (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%), bleeding or gastrointestinal disturbances (3.0%), osteoporosis or brittle bones (13.6%), and nervous system disorders (30%).

Discontinuations of treatment varied across studies, but a high proportion of patients discontinued treatment due to lack of efficacy.

Overall, this systematic review suggests that “symptoms associated with MG improve with improvement and remission of the disease”.

However, “additional options in an effective treatment that adequately address disease-related symptoms and also improve HRQoL may contribute to beneficial outcomes in more patients with MG,” they wrote.


Comments are closed.