Patient-reported burden of myasthenia gravis: baseline results of the international prospective, observational, longitudinal real-world digital study MyRealWorld-MG

Abstract

ObjectivesMyasthenia gravis (MG) is a rare, chronic, autoimmune neuromuscular disease which can affect functional and mental aspects of health and health-related quality of life (HRQoL). This study aims to obtain detailed knowledge of the impact of MG on HRQoL in a broad population from the perspective of the patient.DesignProspective, observational, digital, longitudinal real-world study.SettingAdult patients with MG from seven countries (USA, Japan, Germany, UK, Italy, Spain and Canada) downloaded a mobile application onto their phones and entered data about themselves and their MG.Outcome measuresData was collected using the following general and disease-specific patient-reported outcome measurements: EuroQol 5 Domains Health-Related Quality of Life Questionnaire (EQ-5D-5L), Myasthenia Gravis Activities of Daily Living (MG-ADL), Myasthenia Gravis Quality of Life 15-item revised scale (MG-QoL-15r), Hospital Anxiety and Depression Scale (HADS) and Health Utilities Index III (HUI3). Patients were categorised by their self-assessed Myasthenia Gravis Foundation of America (MGFA) class (I–V).ResultsBaseline results of 841 participants (mean age 47 years, 70% women) are reported . The distribution across the MGFA classes was: 13.9%, 31.0%, 38.1%, 15.5% and 1.6% for classes I–V. The MGFA class was a strong predictor of all aspects of HRQoL, measured with disease-specific and with generic instruments. The domains in which patients with MG most frequently mentioned problems were usual activities, anxiety and depression, tiredness, breathing and vision. The mean total MG-ADL Score was positively associated with increasing MGFA classes: 2.7, 4.4, 6.3 and 8.4 for MGFA classes I–IV. Mean baseline EQ-5D-5L utility was also associated with MGFA classes and was 0.817, 0.766, 0.648 and 0.530 for MGFA class I–IV.ConclusionsMG has a large impact on key aspects of health and HRQoL. The impact of this disease increases substantially with increasing disease severity.

Expert consensus recommendations for improving and standardising the assessment of patients with generalised myasthenia gravis

“…A patient-centred approach is important in gMG treatment. [EQ-5D-VAS]) [3,16,22] to determine patient satisfaction with symptom state and treatment can be used effectively in this setting.…”

“…Most patients with myasthenia gravis (MG) first present with ocular symptoms only; however, approximately 80% will have gMG within 2 years of initial disease onset [2]. gMG is classified by degree of muscle weakness, which, along with other symptoms of the disease, may vary greatly among individuals and fluctuate over time [1][2][3][4][5][6]. This variation in symptoms, combined with infrequent and isolated evaluations, means accurate tracking of symptom state and full comprehension of disease burden is difficult [4][5][6].…”

“…In addition, the impact of gMG on key areas of patient health and health-related quality of life (HRQoL) is profound, increasing substantially with increased disease severity [3]. Many patients also continue to experience substantial disease burden while on treatment, and poor HRQoL may persist even if gMG is considered to be well managed [4,5].…”

Efgartigimod efficacy and safety in refractory Myasthenia Gravis - UK’s first real-world experience

“…Approximately 15% of patients are refractory to standard therapies and are often dependent on costly treatments such as IVIg and TPE 3 . Real-world studies have shown that over 40% of patients with MG have unacceptable disease control 8 and MG is known to have a significant impact on quality of life 9 .…”

Association Between Myasthenia Gravis–Activities of Daily Living (MG-ADL) and EQ-5D-5L Utility Values: The Additional Effect of Efgartigimod on Utilities

“…Consequently, patients with gMG report lower health-related quality of life (HRQoL) than in the general population [ 8 , 9 ]. Several disease-specific and generic instruments have been validated in patients with gMG that can be used to assess patients’ symptom severity, functional status, and HRQoL.…”