Health‐related quality of life and treatment strategies in myasthenia gravis

Abstract

The spread of immune treatments based on oral steroids since the 1970s has led to a non-lethal disease course and decreased severity of illness for many patients with myasthenia gravis (MG). 1-6 However, the rate of long-term complete stable remission (CSR) has not been increased and remains quite low. 1-6 Even today, many patients experience difficulties with daily activities as a result of residual fatigability and long-term side-effects of medications, such as oral steroids, and they are unable to live a normal lifestyle. 1,2,5,6 Such conditions negatively influence health-related quality of life (QOL). 5-8 In Japan until the 2000s, a considerable proportion of neurologists were content with the better clinical symptoms after immune treatment and the preferable condition compared with the period when MG had been a severe disease with a high mortality rate. MG symptoms fluctuate and limit the utility of measuring disease status based solely on simple examination during clinical visits. 9,10 Given the delay in the adoption of quantitative estimation of fatigability

Safety and effectiveness of eculizumab in Japanese patients with generalized myasthenia gravis: Analysis of 1‐year postmarketing surveillance

“…Although corticosteroids have a relatively fast onset of action, they act nonspecifically, not only in suppressing the immune system, but also in reducing bone density, and affecting the central nervous, vascular, and endocrine systems. Their long‐term use is associated with a range of adverse side effects and can negatively impact on quality of life in patients with MG 10,11,17,36–39 . Conversely, achieving minimal manifestations status or better with a prednisolone dose of ≤5 mg/d has been shown to have a significantly positive effect on health‐related quality of life in Japanese patients with MG 10 .…”

“…Their long-term use is associated with a range of adverse side effects and can negatively impact on quality of life in patients with MG. 10,11,17,[36][37][38][39] Conversely, achieving minimal manifestations status or better with a prednisolone dose of ≤5 mg/d has been shown to have a significantly positive effect on health-related quality of life in Japanese patients with MG. 10 The current analysis demonstrated that eculizumab treatment was associated with an increase over time in the proportion of patients who were treated with a low daily oral corticosteroid dose of ≤5 mg, the current target recommended in treatment guidelines for MG in Japan. 1 This is consistent with the results of a post-hoc analysis of immunosuppressant use in the REGAIN OLE, which showed that eculizumab treatment was accompanied by significant reductions in the mean daily dose of oral prednisone and other corticosteroids-almost half of patients decreased dosage or stopped their corticosteroid during the OLE.…”

“…Clinical practice guidelines for MG in Japan recommend repeated treatment with high‐dose intravenous immunoglobulin (IVIg) and/or plasmapheresis, with the goal of achieving minimal manifestations or better as rapidly as possible, with an oral prednisolone dose of ≤5 mg/d 1 . However, despite aggressive and repeated treatment with such approaches, a proportion of patients do not show clinical improvement and at least 30% of patients with gMG do not achieve minimal manifestations with prednisolone ≤5 mg/d 5,9,16,17 …”

“…1 However, despite aggressive and repeated treatment with such approaches, a proportion of patients do not show clinical improvement and at least 30% of patients with gMG do not achieve minimal manifestations with prednisolone ≤5 mg/d. 5,9,16,17 The muscle weakness that characterizes MG is a result of impairment of neuromuscular transmission. 18 The majority ($85%) of patients with gMG have detectable autoantibodies to the acetylcholine receptor (AChR).…”

Development of infections among patients with myasthenia gravis undergoing immunotherapy

“…A complement component C5 inhibitor, eculizumab, 3 and neonatal Fc receptor inhibitor, efgartigimod, 4 were recently approved for refractory cases. A few patients can achieve a complete remission with these treatments, 5 and many patients remain requiring long‐term immunotherapies. Infections are adverse effects of immunotherapy.…”