Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)

Berrih‐Aknin, Sonia; Claeys, Kristl G.; Law, Nancy; Mantegazza, Renato; Murai, Hiroyuki; Saccà, Francesco; Dewilde, Sarah; Janssen, Mathieu F.; Bagshaw, Emma; Kousoulakou, H.; Larkin, Mark; Beauchamp, Jon; Leighton, Trevor; Paci, Sandra

doi:10.1136/bmjopen-2020-048198

Cited by 16 publications

(32 citation statements)

References 31 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…MG is a rare disease, with an estimated 60,000 cases of MG in the United States (US) [ 7 , 8 ]. As with many rare diseases, because of the relatively low prevalence compared with more common diseases, there is a lack of real-world studies in MG to understand and address patients’ unmet needs and the burden of disease [ 9 ]. While randomized clinical trials are necessary to obtain robust efficacy and safety data on a treatment, observational real-world studies are also important as they can provide information on larger, more representative populations compared with clinical trials, and an understanding of the true progression and management of the disease [ 9 ].…”

Section: Introductionmentioning

confidence: 99%

“…As with many rare diseases, because of the relatively low prevalence compared with more common diseases, there is a lack of real-world studies in MG to understand and address patients’ unmet needs and the burden of disease [ 9 ]. While randomized clinical trials are necessary to obtain robust efficacy and safety data on a treatment, observational real-world studies are also important as they can provide information on larger, more representative populations compared with clinical trials, and an understanding of the true progression and management of the disease [ 9 ]. An analysis of insurance claims between 2010 and 2019 from the IBM® MarketScan® database showed that 91% of patients living with MG in the US received drug therapy, and 40% of those received non-steroidal immunosuppressive treatment (IST) or biologics [ 10 ].…”

Section: Introductionmentioning

confidence: 99%

“…Adelphi Real World Disease Specific Programmes™ (DSPs) are an established methodology for investigating treatment practices across a large range of diseases using point-in-time surveys [ 17 ]. They collect real-world data from clinical practice, including patient demographics, treatment practices and physician-reported healthcare resource utilization (HCRU) [ 9 ].…”

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Physician-Reported Perspectives on Myasthenia Gravis in the United States: A Real-World Survey

et al. 2022

View full text Add to dashboard Cite

Introduction: Myasthenia gravis (MG) is a rare, debilitating, chronic disorder caused by the production of pathogenic immunoglobulin G autoantibodies against the neuromuscular junction. A lack of real-world studies in rare diseases reflects a relatively limited understanding of the significant unmet needs and burden of disease for patients. We aimed to provide comprehensive real-world insights into the management and burden of MG from treating physicians in the United States (US). Methods: Data were collected using the Adelphi Real World MG Disease Specific Programme TM , a point-in-time survey of physicians and their patients with MG, in the US between March and July 2020. Physician-reported clinical data, including demographics, comorbidities, symptoms, disease history, treatments, and healthcare resource utilization, were collected. Results: In total, 456 patient record forms were completed by 78 physicians based in the US. At time of survey completion, patient mean age was 54.5 years. Mean time from symptom onset to diagnosis was 9.0 months (n = 357). Ocular symptoms were reported in 71.7% of patients. General fatigue affected 47.1% of patients and over half of those reported the severity as moderate or severe (59.5%, n = 128). Acetylcholinesterase inhibitors and/or steroids were the most frequently prescribed first-line treatment type among patients receiving treatment at time of survey completion and with moderate-to-severe symptoms (77.9%, n = 159/204). High-dose steroids (n = 14) and intravenous immunoglobulin (n = 13) were the most prescribed acute treatments among those receiving an acute treatment at time of survey completion (n = 36), with symptom exacerbations or myasthenic crises being the most common reasons for acute treatment. On average, 2.5 healthcare professionals were involved in patient management and 5.0 consultations were made per patient over the last 12 months. Conclusions: Our findings indicated that, despite treatment, there is a proportion of patients with MG in the US who had a significant need for improved disease management.

show abstract

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Physician-Reported Perspectives on Myasthenia Gravis in the United States: A Real-World Survey

et al. 2022

View full text Add to dashboard Cite

show abstract

“…In addition, they have provided input into the publication and communication plan of the study and are offered the opportunity to coauthor resulting academic papers. 9 …”

Section: Methodsmentioning

confidence: 99%

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

Dewilde¹,

Philips²,

Paci

et al. 2023

BMJ Open

Self Cite

View full text Add to dashboard Cite

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.

show abstract

“…In recent years, interest has been growing in the emotional, socio-familial, and humanistic aspects of MG, as well as the effects of medical interventions on disease burden [ 6 , 7 , 8 , 9 , 10 ]. This scenario is especially crucial for patients with treatment-refractory MG [ 11 , 12 ].…”

Section: Introductionmentioning

confidence: 99%

New Targeted Agents in Myasthenia Gravis and Future Therapeutic Strategies

Sánchez-Tejerina

Sotoca

Llauradó

et al. 2022

JCM

View full text Add to dashboard Cite

Myasthenia gravis (MG) is a chronic autoimmune disease for which multiple immunomodulatory therapies are available. Nevertheless, MG has a significant impact on patient quality of life. In recent years, experts’ main efforts have focused on optimizing treatment strategies, since disease burden is considerably affected by their safety and tolerability profiles, especially in patients with refractory phenotypes. This article aims to offer neurologists caring for MG patients an overview of the most innovative targeted drugs specifically designed for this disease and summarizes the recent literature and more recent evidence on agents targeting B cells and plasmablasts, complement inhibitors, and neonatal fragment crystallizable receptor (FcRn) antagonists. Positive clinical trial results have been reported, and other studies are ongoing. Finally, we briefly discuss how the introduction of these novel targeted immunological therapies in a changing management paradigm would affect not only clinical outcomes, disease burden, safety, and tolerability, but also health spending in a condition that is increasingly managed based on a patient-centred model.

show abstract

Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG)

Cited by 16 publications

References 31 publications

Physician-Reported Perspectives on Myasthenia Gravis in the United States: A Real-World Survey

Physician-Reported Perspectives on Myasthenia Gravis in the United States: A Real-World Survey

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

New Targeted Agents in Myasthenia Gravis and Future Therapeutic Strategies

Contact Info

Product

Resources

About