Treatments conventionally used for myasthenia gravis (MG) can produce side effects that negatively impact the daily lives of patients and may deter them from taking their medications properly, according to a study which also highlights the need for more effective and tolerable treatment options.
The report, titled “Understanding side effects of therapy for myasthenia gravis and their impact on daily life,” was published in the journal BMC Neurology.
MG is a chronic autoimmune condition characterized by weakness and fatigue in several voluntary muscles, which may also affect the muscles that control breathing.
With no cure for their MG, patients can still live a normal lifespan using various therapies, including acetylcholinesterase inhibitors such as pyridostigmine (sold as Mestinon by Bausch Health Companies; generics also available), and immunomodulating therapies such as corticosteroids, azathioprine, and mycophenolate mofetil (brand name CellCept by Genentech).
Such therapies are, however, associated with significant side effects such as nausea, vomiting, gastrointestinal problems, increased infection risk, weight gain, or liver damage.
To better understand the impact of these side effects on patients, a team led by researchers from Evidera‘s Patient-Centered Research group conducted a two-part study that included one-on-one interviews and a web-based survey involving adult MG patients.
In the first part of the study, 14 patients recruited from an MG clinic at the University of South Florida were interviewed, while 246 patients invited from the MG patient registry entered the second part.
All 14 participants in the study’s first half reported experiencing side effects from MG treatments, with the most commonly reported being weight gain (57.1% of patients), diarrhea or gastrointestinal upset (42.9%), and headaches/migraines (35.7%).
Of note, most patients (85.7%) were taking Mestinon for MG.
The patients said that the side effects had a significant impact on their daily lives. The most common problems included having to take additional medication to cope with the side effects (64.3%), having to change their diet (42.9%), or having to buy new clothes as a result of weight gain (42.9%). Emotional problems (42.9%), and feeling bedridden or unable to leave the house (42.9%) were also cited.
According to the interviews, headaches and weight gain were the side effects with the greatest impact on the patients’ lives.
Despite that, all patients felt the medications worked to ease MG symptoms and most of them (71.4%) did not stop taking the medications nor did they decide to take a lower dose than prescribed. The remaining (28.6%) reported stopping or lowering their medications at least once.
In the study’s second part, 246 participants completed an initial brainstorming/concept-generation task and, of these, 242 also completed sociodemographic and clinical questionnaires.
The majority (69.0%) reported taking Mestinon. One-third (33.9%) also reported taking some type of oral corticosteroid, including prednisone, and 37.6% had undergone a thymectomy (surgery to remove the thymus gland).
The researchers invited participants once again to rate a group of 35 statements related to side effects and 23 statements related to impacts on daily life, which had been chosen from the prior brainstorming sessions and surveys.
According to patient ratings, the most severe and least tolerable side effects were those related to the development of blood clots, aseptic meningitis, sepsis (medical emergency caused by widespread inflammation due to an infection), pneumonia, weight gain, decreased immunity, and diarrhea.
Many patients rated no impact of side effects on their quality of life. But among those who felt such a burden, the most frequent and severe problems were difficulties in sleeping, and reduced physical and social activities. The least significant impacts were related to healthcare utilization issues, including additional medical procedures and hospitalizations.
Additionally, patients whose disease did not respond (refractory) to traditional MG treatments tended to report greater severity and impact of side effects on their daily lives, compared to non-refractory patients.
“This study has confirmed the serious burden of side effects resulting from traditional MG treatments and the negative impact of side effects on patient daily life,” the researchers wrote.
“Based on these findings, there is a need for more effective and more tolerable treatment options for patients with MG,” they said.