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. 2019 Dec;60(6):707-715.
doi: 10.1002/mus.26695. Epub 2019 Oct 9.

Cross-sectional analysis of the Myasthenia Gravis Patient Registry: Disability and treatment

Gary Cutter  1 , Haichang Xin  1 , Inmaculada Aban  1 , Ted M Burns  2 , Phillip H Allman  1 , Ramin Farzaneh-Far  3 , Petra W Duda  3 , Henry J Kaminski  4
Affiliations

Cross-sectional analysis of the Myasthenia Gravis Patient Registry: Disability and treatment

Gary Cutter et al. Muscle Nerve. 2019 Dec.

Abstract

Introduction: The Myasthenia Gravis Patient Registry (MGR) is a voluntary, patient-submitted database dedicated to improve understanding of care/burden of myasthenia gravis (MG).

Methods: In this study we present analyses of baseline records through July 2017 (n = 1140) containing data on the MG-Activities of Daily Living (MG-ADL) and the MG 15-item Quality of Life (MG-QOL15) instruments, two validated scales assessing quality of life in MG patients at sign-up into the MGR.

Results: Most registrants reported moderate to severe impairment of health-related quality of life, with a median MG-ADL score of 6 and a median MG-QOL15 score of 21. Seventy-one percent of the patients had received pyridostigmine. Corticosteroids, mycophenolate mofetil, and azathioprine were the most common immunomodulators/immunosuppressants, with 85% of participants having ever using one of these agents. Forty-seven registrants reported receiving intravenous immunoglobulin, and 30% received plasma exchange. Twelve percent reported other treatments, and 40% were unsure whether they received less common therapies. Forty percent had undergone thymectomy.

Discussion: The MGR data correlate well with other MG cohorts. Many MG patients remain negatively impacted despite treatment.

Keywords: ADL; MG; MGFA; QOL; myasthenia.

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Conflict of interest statement

R.F.F. is an employee of Ra Pharmaceuticals, Inc, which funded the study. P.W.D. is an employee of Ra Pharmaceuticals. G.C. is employed by the University of Alabama at Birmingham and president of Pythagoras, Inc, a private consulting company in Birmingham, Alabama. G.C. is also a member of the data safety and monitoring boards for AMO Pharmaceuticals, Biolinerx, Horizon Pharmaceuticals, Hisun Pharmaceuticals, Merck, Merck/Pfizer, Opko Biologics, Neurim, Novartis, Ophazyme, Sanofi‐Aventis, Reata Pharmaceuticals, Receptos/Celgene, Teva Pharmaceuticals, the NHLBI (protocol review committee), and the NICHD (OPRU oversight committee), and is on the consulting or advisory boards of Atara Biotherapeutics, Axon, Biogen, Argenix, Brainstorm Cell Therapeutics, Charleston Labs, Click Therapeutics, Genzyme, Genentech, GW Pharma, Klein‐Buendel, Medimmune, Medday, Novartis, Roche, Scifluor, Somahlution, Teva Pharmaceuticals, TG Therapeutics, and University of Texas at Houston. H.J.K. is a member of the data safety and monitoring boards for Novartis and the National Institutes of Health; receives consulting fees from Alnylam Pharmaceuticals, UCB, Biocatalyst, RA Pharmaceuticals, and Momenta Pharmaceuticals; receives grant support from Akari Pharmaceuticals and the Muscular Dystrophy Association; and holds a patent related to technology to focus a complement inhibitor on the neuromuscular junction for the treatment of myasthenia gravis (US Patent No. 8,961,981). The remaining authors have no potential conflicts of interest to disclose related to this report.

Figures

Figure 1
Figure 1
Age at disease onset. Although incidence was similar from the second to seventh decade overall, the peak age at disease onset was in the fourth decade in female patients and in the sixth decade in male patients
Figure 2
Figure 2
Distribution of (A) MG‐ADL and (B) MG‐QOL15 at sign‐up. The majority of patients reported moderate to severe impairment in their activities of daily living, as measured by MG‐ADL (N = 1140) and MG‐QOL15 (N = 1138) scale scores. MG‐ADL, Myasthenia Gravis—Activities of Daily Living; MG‐QOL15, 15‐item Myasthenia Gravis Quality of Life
Figure 3
Figure 3
Immunotherapies in MG Patient Registry at sign‐up. Current and past medication use as reported by patients is generally consistent with current treatment guidelines for MG standard of care. MG, myathenia gravis; PLEX, plasmapheresis
Figure 4
Figure 4
Analysis of most frequently used immunomodulatory therapies at sign‐up. Patients using corticosteroids, azathioprine, and/or mycophenolate mofetil (A), at present (n = 723), and (B) at any time (n = 966), past or present, for treatment of myasthenia of the 1140 MGR patients assessed. The Venn diagrams with areas proportional to the number of patients in each group have been generated using EulerAPE (http://www.eulerdiagrams.org/eulerAPE/). MGR, Myasthenia Gravis Patient Registry
Figure 5
Figure 5
MG‐ADL and MG‐QOL15 in the immunotherapy subgroups at sign‐up. Mean and 95% confidence intervals for MG‐ADL and MG‐QOL15 measured at entry into the MGR for immunotherapy subgroups based on their self‐reported current or past medication use. Black vertical lines indicate overall means and 95% confidence intervals for the entire study sample, irrespective of therapy. MG‐ADL, Myasthenia Gravis—Activities of Daily Living; MG‐QOL15, 15‐item Myasthenia Gravis Quality of Life; MGR, Myasthenia Gravis Patient Registry
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