#AANAM – Patient-led Study Highlights Challenges of Living With MG

Editor’s note: The Myasthenia Gravis News team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. Go here to read the latest stories from the conference.

People with myasthenia gravis (MG) have used a patient-led analysis to document and share with healthcare professionals their experiences of living with MG.

The goal of the study is to try to improve disease management and outcomes with better understanding by healthcare providers of the challenges posed by this chronic autoimmune disorder.

The study identified several key themes, including the need for patients to constantly adapt their activities and lives due to the unpredictable nature of MG symptoms. Respondents also described their need to make sacrifices and trade-offs in dealing with their treatment, and the perceived differences between patients and their healthcare providers regarding symptom control and quality of life.”

Findings from this patient-led study were presented at the 2021 American Academy of Neurology (AAN) Virtual Annual Meeting, in a poster titled “The Lived Experience of Myasthenia Gravis (MG): A Patient-led Analysis.” The AAN meeting was held online April 17-22.

In MG, the body’s immune system mistakenly attacks the neuromuscular junction — the place where nerves and muscles communicate — leading to extreme muscle weakness and fatigue. In some cases, muscle weakness can become life-threatening when it compromises a patient’s ability to swallow or breathe. These potentially life-threatening episodes are known as MG crises. 

Here, researchers noted that there is little data on how MG impacts daily life from a patient’s perspective. 

“Understanding the reality of living with MG may provide insights that can improve management and outcomes for patients,” the team wrote.

To that end, UCB Pharma, a developer of investigational MG therapeutics, along with the Myasthenia Gravis Foundation of America (MGFA), convened an international patient global council (PGC) to reveal the lived experience of MG from the patient perspective. The PGC was comprised of nine individuals living with MG who serve as local or national patient advocates in seven countries. Those countries included the U.S. and several European nations.

“Despite thousands of people across the world living with the unpredictable and, in many cases, debilitating impact of MG, there is still relatively little research describing true lived experiences faced by the MG patient community, from their perspective,” Nancy Law, former CEO and current board chair for the MGFA, said in a press release.

“Most of the current data are about others’ perception of patient lived experience,” added Law, co-author of the study’s abstract.

Insights into the daily lives and challenges of people with MG were consolidated from various sources, including a literature review, a qualitative research study of 54 MG patients and caregivers in seven countries, and a PCG meeting held in 2019. The council now prioritized those insights and discussed them in depth in a virtual workshop. 

A total of 114 patient insights were organized into nine subject areas or domains, such as physical, psychological, social, parenting, and activity participation. Disease-related insights included MG control, flare-ups and MG crises, treatment burden, and unmet needs. 

Representative experiences were described in a series of consensus statements. The patient authors identified a total of five overarching themes. 

First, they highlighted how the fluctuating and unpredictable nature of MG symptoms — with periods of disease worsening followed by remission — can have a substantial impact on their lives. 

“Every patient will have muscle weakness, but the difficulty to live with is that it is so unstable, the fluctuation is even worse to live with than the muscle weakness itself,” said one of the patients.

Second, as a consequence of living with fluctuating symptoms, many individuals felt they were in a constant state of adaptation and continual assessment. That led to trade-offs in all aspects of life, including crucial areas such as work, family planning, and treatment. 

“You feel it from the moment you wake up, and you have to adjust your routines and expectations; I live day by day,” said one of the patients of the qualitative study. “Those bad days you need to prioritize the most important activities, or the most basic, and try to work with your medication.”

Third, the authors noted that both patients and healthcare providers (HCPs) can be reluctant to leave their comfort zone in regards to MG treatment, often leading to “treatment inertia” and undertreatment. This can occur due to a lack of consensus on optimal disease control, concerns over side effects, and the time needed to see benefits following a change in treatment, among other factors.

“If you don’t know something is going to work, and it doesn’t work, you feel like you’ve wasted six months, which can be very frustrating,” said one of the participants.

Furthermore, some patients who are not treated by specialists can feel their HCPs do not fully understand their condition.

Fourth, many patients reported a sense of disconnect with their HCPs. The lack of connection was driven mainly by obstacles in communication, such as limited time, and by gaps in the perception of both disease and treatment burden. Having different treatment goals also added to that sense of disconnect.

According to patients, some HCPs may focus on managing symptoms and side effects, but these approaches fail to address the impact MG has on their overall daily lives. Patients stressed a lack of understanding by providers about the side effects and other difficulties people with MG have to live with as part of their condition.

“He may think I’m in remission, but I’m taking 20–30 mg of prednisone, I have all these side effects. It’s not adequate control,” said one of the patients. 

Fifth, the condition can lead to feelings of anxiety, frustration, anger, guilt, loneliness, and depression. These feelings are driven not only by the symptoms themselves, but also by social isolation, loss of control, and a lack of support. 

“I remember a time when I couldn’t go out for dinner with friends after the theatre because I was so exhausted. I cried a lot that evening,” a participant said. 

Summary statements from the physical domain of the analysis described how unstable muscle weakness and fatigue were experienced by almost all people with MG. Such symptoms are difficult to live with and often led to disrupted sleep. 

Socially, living with MG brought additional difficulties in making plans, which in turn had a negative impact on relationships, resulting in feelings of loneliness. However, participants found comfort in connecting with other MG patients who understand how they feel.

Participating in activities needs more planning, can take longer, and may need to be limited, impacting employment and schooling, the study’s participants said. 

Psychologically, MG is primarily an “invisible” illness, meaning that others may not fully understand its impact. That, in turn, might lead to feelings of guilt.

In contrast, living with MG can bring out feelings of optimism, hope, and resilience. Emotional support also is essential, the patients said. 

People with MG also said they had concerns about pregnancy and child rearing, such as the impact the disease and its treatment might have on their ability to cope as a parent. MG also leads to trade-offs for people with children. More education and guidance are needed for family planning, the respondents stressed. 

Regarding the disease, the participants believed MG could be better controlled in a significant proportion of patients. Moreover, many also noted how flare-ups and crises after a period of disease stability or remission can be discouraging. In this sense, resilience may help those with MG to cope. 

While a cure for MG is the ultimate goal, better longer-term treatment efficacy and tolerability would already be an improvement for those with the disease, the respondents said when discussing unmet needs. There also was an expressed need for improved treatment — and patients want to hear about new treatments in development. 

It also was noted that technology can improve engagement and education in those with MG, and that psychological patient support could be further improved.

“This patient-driven analysis enriches our understanding of the reality of living with MG,” the scientists wrote in the poster.

“A greater understanding of what it means to live with MG could help healthcare professionals to better communicate with patients,” they said.

The MGFA said it was pleased that the AAN included this patient-led study in its presentations for the 73rd annual forum.

“It is heartening that in accepting this data for presentation at their meeting, the American Academy of Neurology has recognized the importance of healthcare professionals truly understanding the perspectives of patients on their experiences,” Law said.

“Patient collaborations, such as this one with the pharmaceutical industry, have potential to raise the patient voice and build better understanding about this rare neuromuscular disease,” she said.