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Published in: Neurology and Therapy 4/2022

Open Access 20-07-2022 | Myasthenia Gravis | Original Research

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

Authors: Milada Mahic, Ali M. Bozorg, Jonathan J. DeCourcy, Keisha J. Golden, Gregor A. Gibson, Christian F. Taylor, Angela Ting, Tyler J. Story, Anna Scowcroft

Published in: Neurology and Therapy | Issue 4/2022

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Abstract

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™, 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.
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Literature
1.
go back to reference Bacci ED, Coyne KS, Poon JL, Harris L, Boscoe AN. Understanding side effects of therapy for myasthenia gravis and their impact on daily life. BMC Neurol. 2019;19(1):335.PubMedPubMedCentralCrossRef Bacci ED, Coyne KS, Poon JL, Harris L, Boscoe AN. Understanding side effects of therapy for myasthenia gravis and their impact on daily life. BMC Neurol. 2019;19(1):335.PubMedPubMedCentralCrossRef
2.
go back to reference Szczudlik P, Sobieszczuk E, Szyluk B, Lipowska M, Kubiszewska J, Kostera-Pruszczyk A. Determinants of quality of life in myasthenia gravis patients. Front Neurol. 2020;11: 553626.PubMedPubMedCentralCrossRef Szczudlik P, Sobieszczuk E, Szyluk B, Lipowska M, Kubiszewska J, Kostera-Pruszczyk A. Determinants of quality of life in myasthenia gravis patients. Front Neurol. 2020;11: 553626.PubMedPubMedCentralCrossRef
3.
go back to reference Gilhus NE, Tzartos S, Evoli A, Palace J, Burns TM, Verschuuren J. Myasthenia gravis. Nat Rev Dis Primers. 2019;5(1):30.PubMedCrossRef Gilhus NE, Tzartos S, Evoli A, Palace J, Burns TM, Verschuuren J. Myasthenia gravis. Nat Rev Dis Primers. 2019;5(1):30.PubMedCrossRef
5.
go back to reference Melzer N, Ruck T, Fuhr P, Gold R, Hohlfeld R, Marx A, et al. Clinical features, pathogenesis, and treatment of myasthenia gravis: a supplement to the Guidelines of the German Neurological Society. J Neurol. 2016;263(8):1473–94.PubMedPubMedCentralCrossRef Melzer N, Ruck T, Fuhr P, Gold R, Hohlfeld R, Marx A, et al. Clinical features, pathogenesis, and treatment of myasthenia gravis: a supplement to the Guidelines of the German Neurological Society. J Neurol. 2016;263(8):1473–94.PubMedPubMedCentralCrossRef
6.
go back to reference Howard JF Jr. Myasthenia gravis: the role of complement at the neuromuscular junction. Ann N Y Acad Sci. 2018;1412(1):113–28.PubMedCrossRef Howard JF Jr. Myasthenia gravis: the role of complement at the neuromuscular junction. Ann N Y Acad Sci. 2018;1412(1):113–28.PubMedCrossRef
8.
go back to reference Gilhus NE, Verschuuren JJ. Myasthenia gravis: subgroup classification and therapeutic strategies. Lancet Neurol. 2015;14(10):1023–36.PubMedCrossRef Gilhus NE, Verschuuren JJ. Myasthenia gravis: subgroup classification and therapeutic strategies. Lancet Neurol. 2015;14(10):1023–36.PubMedCrossRef
9.
go back to reference Berrih-Aknin S, Claeys KG, Law N, Mantegazza R, Murai H, Saccà F, et al. Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG). BMJ Open. 2021;11(7): e048198.PubMedPubMedCentralCrossRef Berrih-Aknin S, Claeys KG, Law N, Mantegazza R, Murai H, Saccà F, et al. Patient-reported impact of myasthenia gravis in the real world: protocol for a digital observational study (MyRealWorld MG). BMJ Open. 2021;11(7): e048198.PubMedPubMedCentralCrossRef
10.
go back to reference Mahic M, Zaremba P, Rudnik J, Bozorg A, Dolin P. Currently used therapies for myasthenia gravis do not provide adequate disease control for all patients: a retrospective analysis of United States claims data. Am Assoc Neuromusc Electrodiagn Med. 2021;2021:5. Mahic M, Zaremba P, Rudnik J, Bozorg A, Dolin P. Currently used therapies for myasthenia gravis do not provide adequate disease control for all patients: a retrospective analysis of United States claims data. Am Assoc Neuromusc Electrodiagn Med. 2021;2021:5.
11.
go back to reference Cutter G, Xin H, Aban I, Burns TM, Allman PH, Farzaneh-Far R, et al. Cross-sectional analysis of the Myasthenia Gravis Patient Registry: disability and treatment. Muscle Nerve. 2019;60(6):707–15.PubMedPubMedCentralCrossRef Cutter G, Xin H, Aban I, Burns TM, Allman PH, Farzaneh-Far R, et al. Cross-sectional analysis of the Myasthenia Gravis Patient Registry: disability and treatment. Muscle Nerve. 2019;60(6):707–15.PubMedPubMedCentralCrossRef
12.
go back to reference Mantegazza R, Antozzi C. When myasthenia gravis is deemed refractory: clinical signposts and treatment strategies. Ther Adv Neurol Disord. 2018;11:1756285617749134.PubMedPubMedCentralCrossRef Mantegazza R, Antozzi C. When myasthenia gravis is deemed refractory: clinical signposts and treatment strategies. Ther Adv Neurol Disord. 2018;11:1756285617749134.PubMedPubMedCentralCrossRef
13.
go back to reference Schneider-Gold C, Hagenacker T, Melzer N, Ruck T. Understanding the burden of refractory myasthenia gravis. Ther Adv Neurol Disord. 2019;12:1756286419832242.PubMedPubMedCentralCrossRef Schneider-Gold C, Hagenacker T, Melzer N, Ruck T. Understanding the burden of refractory myasthenia gravis. Ther Adv Neurol Disord. 2019;12:1756286419832242.PubMedPubMedCentralCrossRef
14.
go back to reference Guptill JT, Sharma BK, Marano A, Soucy A, Krueger A, Sanders DB. Estimated cost of treating myasthenia gravis in an insured US population. Muscle Nerve. 2012;45(3):363–6.PubMedCrossRef Guptill JT, Sharma BK, Marano A, Soucy A, Krueger A, Sanders DB. Estimated cost of treating myasthenia gravis in an insured US population. Muscle Nerve. 2012;45(3):363–6.PubMedCrossRef
15.
go back to reference Xin H, Harris LA, Aban IB, Cutter G. Examining the impact of refractory myasthenia gravis on healthcare resource utilization in the United States: analysis of a myasthenia gravis foundation of America Patient Registry sample. J Clin Neurol. 2019;15(3):376–85.PubMedPubMedCentralCrossRef Xin H, Harris LA, Aban IB, Cutter G. Examining the impact of refractory myasthenia gravis on healthcare resource utilization in the United States: analysis of a myasthenia gravis foundation of America Patient Registry sample. J Clin Neurol. 2019;15(3):376–85.PubMedPubMedCentralCrossRef
16.
go back to reference Landfeldt E, Pogoryelova O, Sejersen T, Zethraeus N, Breiner A, Lochmuller H. Economic costs of myasthenia gravis: a systematic review. Pharmacoeconomics. 2020;38(7):715–28.PubMedCrossRef Landfeldt E, Pogoryelova O, Sejersen T, Zethraeus N, Breiner A, Lochmuller H. Economic costs of myasthenia gravis: a systematic review. Pharmacoeconomics. 2020;38(7):715–28.PubMedCrossRef
17.
go back to reference Anderson P, Benford M, Harris N, Karavali M, Piercy J. Real-world physician and patient behaviour across countries: Disease-Specific Programmes—a means to understand. Curr Med Res Opin. 2008;24(11):3063–72.PubMedCrossRef Anderson P, Benford M, Harris N, Karavali M, Piercy J. Real-world physician and patient behaviour across countries: Disease-Specific Programmes—a means to understand. Curr Med Res Opin. 2008;24(11):3063–72.PubMedCrossRef
18.
go back to reference Babineaux SM, Curtis B, Holbrook T, Milligan G, Piercy J. Evidence for validity of a national physician and patient-reported, cross-sectional survey in China and UK: the Disease Specific Programme. BMJ Open. 2016;6(8): e010352.PubMedPubMedCentralCrossRef Babineaux SM, Curtis B, Holbrook T, Milligan G, Piercy J. Evidence for validity of a national physician and patient-reported, cross-sectional survey in China and UK: the Disease Specific Programme. BMJ Open. 2016;6(8): e010352.PubMedPubMedCentralCrossRef
19.
go back to reference Higgins V, Piercy J, Roughley A, Milligan G, Leith A, Siddall J, et al. Trends in medication use in patients with type 2 diabetes mellitus: a long-term view of real-world treatment between 2000 and 2015. Diabetes Metab Syndr Obes. 2016;9:371–80.PubMedPubMedCentralCrossRef Higgins V, Piercy J, Roughley A, Milligan G, Leith A, Siddall J, et al. Trends in medication use in patients with type 2 diabetes mellitus: a long-term view of real-world treatment between 2000 and 2015. Diabetes Metab Syndr Obes. 2016;9:371–80.PubMedPubMedCentralCrossRef
21.
go back to reference Spillane J, Higham E, Kullmann DM. Myasthenia gravis. BMJ Br Med J. 2012;345: e8497.CrossRef Spillane J, Higham E, Kullmann DM. Myasthenia gravis. BMJ Br Med J. 2012;345: e8497.CrossRef
23.
go back to reference Hoffmann S, Ramm J, Grittner U, Kohler S, Siedler J, Meisel A. Fatigue in myasthenia gravis: risk factors and impact on quality of life. Brain Behav. 2016;6(10): e00538.PubMedPubMedCentralCrossRef Hoffmann S, Ramm J, Grittner U, Kohler S, Siedler J, Meisel A. Fatigue in myasthenia gravis: risk factors and impact on quality of life. Brain Behav. 2016;6(10): e00538.PubMedPubMedCentralCrossRef
24.
go back to reference Andersen JB, Gilhus NE, Sanders DB. Factors affecting outcome in myasthenia gravis. Muscle Nerve. 2016;54(6):1041–9.PubMedCrossRef Andersen JB, Gilhus NE, Sanders DB. Factors affecting outcome in myasthenia gravis. Muscle Nerve. 2016;54(6):1041–9.PubMedCrossRef
25.
go back to reference Basta IZ, Pekmezović TD, Perić SZ, Kisić-Tepavčević DB, Rakočević-Stojanović VM, Stević ZD, et al. Assessment of health-related quality of life in patients with myasthenia gravis in Belgrade (Serbia). Neurol Sci. 2012;33(6):1375–81.PubMedCrossRef Basta IZ, Pekmezović TD, Perić SZ, Kisić-Tepavčević DB, Rakočević-Stojanović VM, Stević ZD, et al. Assessment of health-related quality of life in patients with myasthenia gravis in Belgrade (Serbia). Neurol Sci. 2012;33(6):1375–81.PubMedCrossRef
26.
go back to reference Boldingh MI, Dekker L, Maniaol AH, Brunborg C, Lipka AF, Niks EH, et al. An up-date on health-related quality of life in myasthenia gravis—results from population-based cohorts. Health Qual Life Outcomes. 2015;13:115.PubMedPubMedCentralCrossRef Boldingh MI, Dekker L, Maniaol AH, Brunborg C, Lipka AF, Niks EH, et al. An up-date on health-related quality of life in myasthenia gravis—results from population-based cohorts. Health Qual Life Outcomes. 2015;13:115.PubMedPubMedCentralCrossRef
27.
go back to reference Jeong A, Min J-H, Kang YK, Kim J, Choi M, Seok JM, et al. Factors associated with quality of life of people with Myasthenia Gravis. PLoS ONE. 2018;13(11): e0206754.PubMedPubMedCentralCrossRef Jeong A, Min J-H, Kang YK, Kim J, Choi M, Seok JM, et al. Factors associated with quality of life of people with Myasthenia Gravis. PLoS ONE. 2018;13(11): e0206754.PubMedPubMedCentralCrossRef
28.
go back to reference Masuda M, Utsugisawa K, Suzuki S, Nagane Y, Kabasawa C, Suzuki Y, et al. The MG-QOL15 Japanese version: validation and associations with clinical factors. Muscle Nerve. 2012;46(2):166–73.PubMedCrossRef Masuda M, Utsugisawa K, Suzuki S, Nagane Y, Kabasawa C, Suzuki Y, et al. The MG-QOL15 Japanese version: validation and associations with clinical factors. Muscle Nerve. 2012;46(2):166–73.PubMedCrossRef
30.
go back to reference Twork S, Wiesmeth S, Klewer J, Pöhlau D, Kugler J. Quality of life and life circumstances in German myasthenia gravis patients. Health Qual Life Outcomes. 2010;8:129.PubMedPubMedCentralCrossRef Twork S, Wiesmeth S, Klewer J, Pöhlau D, Kugler J. Quality of life and life circumstances in German myasthenia gravis patients. Health Qual Life Outcomes. 2010;8:129.PubMedPubMedCentralCrossRef
31.
go back to reference Ruiter AM, Verschuuren JJGM, Tannemaat MR. Prevalence and associated factors of fatigue in autoimmune myasthenia gravis. Neuromuscul Disord. 2021;31(7):612–21.PubMedCrossRef Ruiter AM, Verschuuren JJGM, Tannemaat MR. Prevalence and associated factors of fatigue in autoimmune myasthenia gravis. Neuromuscul Disord. 2021;31(7):612–21.PubMedCrossRef
33.
go back to reference Copher R, Kee A, Gerds A. Treatment patterns, health care resource utilization, and cost in patients with myelofibrosis in the United States. Oncologist. 2022;27(3):228–35.PubMedPubMedCentralCrossRef Copher R, Kee A, Gerds A. Treatment patterns, health care resource utilization, and cost in patients with myelofibrosis in the United States. Oncologist. 2022;27(3):228–35.PubMedPubMedCentralCrossRef
34.
go back to reference Fowler NH, Chen G, Lim S, Manson S, Ma Q, Li FY. Treatment patterns and health care costs in commercially insured patients with follicular lymphoma. J Health Econ Outcomes Res. 2020;7(2):148–57.PubMedPubMedCentralCrossRef Fowler NH, Chen G, Lim S, Manson S, Ma Q, Li FY. Treatment patterns and health care costs in commercially insured patients with follicular lymphoma. J Health Econ Outcomes Res. 2020;7(2):148–57.PubMedPubMedCentralCrossRef
35.
go back to reference Lu M, Goodwin B, Vera-Llonch M, Williams J. Disease burden and treatment patterns associated with eosinophilic esophagitis in the United States: a retrospective claims study. J Clin Gastroenterol. 2022;56(2):133–40.PubMedCrossRef Lu M, Goodwin B, Vera-Llonch M, Williams J. Disease burden and treatment patterns associated with eosinophilic esophagitis in the United States: a retrospective claims study. J Clin Gastroenterol. 2022;56(2):133–40.PubMedCrossRef
36.
37.
go back to reference Cortes-Vicente E, Alvarez-Velasco R, Segovia S, Paradas C, Casasnovas C, Guerrero-Sola A, et al. Clinical and therapeutic features of myasthenia gravis in adults based on age at onset. Neurology. 2020;94(11):e1171–80.PubMedPubMedCentralCrossRef Cortes-Vicente E, Alvarez-Velasco R, Segovia S, Paradas C, Casasnovas C, Guerrero-Sola A, et al. Clinical and therapeutic features of myasthenia gravis in adults based on age at onset. Neurology. 2020;94(11):e1171–80.PubMedPubMedCentralCrossRef
38.
go back to reference Guastafierro E, Tramacere I, Toppo C, Leonardi M, Mantegazza R, Bonanno S, et al. Employment in myasthenia gravis: a systematic literature review and meta-analysis. Neuroepidemiology. 2020;54(4):304–12.PubMedCrossRef Guastafierro E, Tramacere I, Toppo C, Leonardi M, Mantegazza R, Bonanno S, et al. Employment in myasthenia gravis: a systematic literature review and meta-analysis. Neuroepidemiology. 2020;54(4):304–12.PubMedCrossRef
Metadata
Title
Physician-Reported Perspectives on Myasthenia Gravis in the United States: A Real-World Survey
Authors
Milada Mahic
Ali M. Bozorg
Jonathan J. DeCourcy
Keisha J. Golden
Gregor A. Gibson
Christian F. Taylor
Angela Ting
Tyler J. Story
Anna Scowcroft
Publication date
20-07-2022
Publisher
Springer Healthcare
Published in
Neurology and Therapy / Issue 4/2022
Print ISSN: 2193-8253
Electronic ISSN: 2193-6536
DOI
https://doi.org/10.1007/s40120-022-00383-3

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