Understanding the Symptom Burden and Impact of Myasthenia Gravis from the Patient’s Perspective: A Qualitative Study

Jackson, Kristi; Parthan, Anju; Lauher-Charest, Miranda; Broderick, Lynne; Law, Nancy; Barnett, Carolina

doi:10.1007/s40120-022-00408-x

Cited by 21 publications

(25 citation statements)

References 32 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Of note, 80% of patients in our study were >40 years of age, which may result in a higher rate of comorbidities compared with a younger population. Similarly, the particular symptoms identified in our study are broadly in line with those highlighted in other studies and have previously been reported as the most bothersome 15–17 . The prominence of slurred speech in work‐related domains may reflect the particular importance of effective communication in the workplace, and has not been previously noted.…”

Section: Discussionsupporting

confidence: 92%

The impact of myasthenia gravis severity on work and daily activities

Pesa,

Choudhry,

de Courcy

et al. 2024

Muscle and Nerve

View full text Add to dashboard Cite

Introduction/AimsPeople with myasthenia gravis (MG) experience impaired quality of life. However, the impact of MG symptoms on work productivity has not been well‐studied. We aimed to evaluate this impact and to examine associations between disease severity and the degree of impairment.MethodsData were drawn from the Adelphi MG Disease‐Specific Programme™, a multinational (USA, France, Germany, Italy, Spain, UK) survey completed by physicians and their patients with MG in 2020. Patient‐reported measures included the Work Productivity and Activity Impairment (WPAI): Specific Health Problem questionnaire.ResultsThe WPAI questionnaire was completed by 330 patients. Among those currently employed, the mean percentage of work time missed (absenteeism) was 13.3% (N = 116), percentage impairment of productivity at work (presenteeism) was 26.7% (N = 121), and overall work impairment was 30.0% (N = 110). Across all patients, impairment of non‐work‐related activities due to health problems (ADL impairment) was 39.2% (N = 330). Regression analysis indicated that impairment differed according to MG Foundation of America (MGFA) class (p = .0147, p < .0001, p < .0001 and p < .0001 for absenteeism, presenteeism, overall work impairment and ADL impairment, respectively). Being MGFA class III/IV was a predictor of presenteeism, overall work impairment and ADL impairment in a predictor model.DiscussionPatients with MG experience substantial work impairment particularly those with more severe symptoms, highlighting an important way in which patient quality of life is negatively affected. More effective treatment strategies would enable patients to lead more productive lives and could impact decisions relating to work and career.

show abstract

Section: Discussionsupporting

confidence: 92%

The impact of myasthenia gravis severity on work and daily activities

Pesa,

Choudhry,

de Courcy

et al. 2024

Muscle and Nerve

View full text Add to dashboard Cite

show abstract

“…The positive effects of NMD670, on top of said immunomodulatory treatment, thus further support the idea that ClC-1 inhibition is additive to other types of MG therapies. In a recent study, patients with MG reported that the nature of the fluctuating and often unpredictable symptom severity necessitates better treatment options that allow patients to keep symptoms under control ( 27 ). From the current work, NMD670 appears to have a fast onset of action and good oral bioavailability, adding further value to this new treatment approach.…”

Section: Discussionmentioning

confidence: 99%

The ClC-1 chloride channel inhibitor NMD670 improves skeletal muscle function in rat models and patients with myasthenia gravis

Skov,

Ruijs,

Grønnebæk

et al. 2024

Sci. Transl. Med.

View full text Add to dashboard Cite

Myasthenia gravis (MG) is a neuromuscular disease that results in compromised transmission of electrical signals at the neuromuscular junction (NMJ) from motor neurons to skeletal muscle fibers. As a result, patients with MG have reduced skeletal muscle function and present with symptoms of severe muscle weakness and fatigue. ClC-1 is a skeletal muscle specific chloride (Cl − ) ion channel that plays important roles in regulating neuromuscular transmission and muscle fiber excitability during intense exercise. Here, we show that partial inhibition of ClC-1 with an orally bioavailable small molecule (NMD670) can restore muscle function in rat models of MG and in patients with MG. In severely affected MG rats, ClC-1 inhibition enhanced neuromuscular transmission, restored muscle function, and improved mobility after both single and prolonged administrations of NMD670. On this basis, NMD670 was progressed through nonclinical safety pharmacology and toxicology studies, leading to approval for testing in clinical studies. After successfully completing phase 1 single ascending dose in healthy volunteers, NMD670 was tested in patients with MG in a randomized, placebo-controlled, single-dose, three-way crossover clinical trial. The clinical trial evaluated safety, pharmacokinetics, and pharmacodynamics of NMD670 in 12 patients with mild MG. NMD670 had a favorable safety profile and led to clinically relevant improvements in the quantitative myasthenia gravis (QMG) total score. This translational study spanning from single muscle fiber recordings to patients provides proof of mechanism for ClC-1 inhibition as a potential therapeutic approach in MG and supports further development of NMD670.

show abstract

“…An Australian study among 165 MG patients found that 40% had stopped working and 20% had changed their occupation due to MG [99]. In a qualitative study from the United States, all 28 MG participants reported impacts on their working activities and financial situation [100].…”

Section: Rehabilitation and Workmentioning

confidence: 99%

Generalized myasthenia gravis with acetylcholine receptor antibodies: A guidance for treatment

Gilhus,

Andersen,

Andersen

et al. 2024

Euro J of Neurology

View full text Add to dashboard Cite

BackgroundGeneralized myasthenia gravis (MG) with antibodies against the acetylcholine receptor is a chronic disease causing muscle weakness. Access to novel treatments warrants authoritative treatment recommendations. The Nordic countries have similar, comprehensive health systems, mandatory health registers, and extensive MG research.MethodsMG experts and patient representatives from the five Nordic countries formed a working group to prepare treatment guidance for MG based on a systematic literature search and consensus meetings.ResultsPyridostigmine represents the first‐line symptomatic treatment, while ambenonium and beta adrenergic agonists are second‐line options. Early thymectomy should be undertaken if a thymoma, and in non‐thymoma patients up to the age of 50–65 years if not obtaining remission on symptomatic treatment. Most patients need immunosuppressive drug treatment. Combining corticosteroids at the lowest possible dose with azathioprine is recommended, rituximab being an alternative first‐line option. Mycophenolate, methotrexate, and tacrolimus represent second‐line immunosuppression. Plasma exchange and intravenous immunoglobulin are used for myasthenic crises and acute exacerbations. Novel complement inhibitors and FcRn blockers are effective and fast‐acting treatments with promising safety profiles. Their use depends on local availability, refunding policies, and cost–benefit analyses. Adapted physical training is recommended. Planning of pregnancies with optimal treatment, information, and awareness of neonatal MG is necessary. Social support and adaptation of work and daily life activities are recommended.ConclusionsSuccessful treatment of MG rests on timely combination of different interventions. Due to spontaneous disease fluctuations, comorbidities, and changes in life conditions, regular long‐term specialized follow‐up is needed. Most patients do reasonably well but there is room for further improvement. Novel treatments are promising, though subject to restricted access due to costs.

show abstract

Understanding the Symptom Burden and Impact of Myasthenia Gravis from the Patient’s Perspective: A Qualitative Study

Cited by 21 publications

References 32 publications

The impact of myasthenia gravis severity on work and daily activities

The impact of myasthenia gravis severity on work and daily activities

The ClC-1 chloride channel inhibitor NMD670 improves skeletal muscle function in rat models and patients with myasthenia gravis

Generalized myasthenia gravis with acetylcholine receptor antibodies: A guidance for treatment

Contact Info

Product

Resources

About