This study is designed to address the evidence gaps in a real-world setting and help patients with MG choose treatments that are best suited to them. It is a prospective, multicenter observational cohort study of comparative effectiveness of MG treatments, with a patient-centered primary outcome measure, to guide clinicians, patients and payers regarding the choice of treatment options for this chronic and serious disease. Primary: To compare the effectiveness of azathioprine (AZT) and mycophenolate mofetil (MMF). Secondary: To compare the outcomes in patients receiving an adequate dose and duration of AZT or MMF over the 2-3 year study period, vs. patients not receiving adequate doses and duration of these agents
Design \& procedures - This is an observational study in the real world clinical setting to evaluate immunosuppressive treatment (IS) of myasthenia gravis (MG). Patients with acquired autoimmune MG ≥ 18 years of age who are not on IS agents, and have not been on corticosteroids for at least 30 days will be enrolled at 20 sites in the US and Canada. These patients will be treated according to the physician's judgment and patient preferences as in routine clinical practice. Patients will be followed prospectively, with the frequency of clinical visits and laboratory monitoring determined by the treating physician, which may differ among patients. Standard outcome measures measuring efficacy and adverse effects that are used in clinical practice will be collected, with emphasis on patient reported outcomes. Informed consent will be obtained using an approved consent form. Patient identifiable / clinical information from the medical record, including the study outcome measures will be uploaded to a centralized REDCap database. The investigators plan to recruit 220 patients, adjusting for a 10% drop out rate, with a final sample of 200 patients for analysis.
Study Type
OBSERVATIONAL
Enrollment
167
oral tablets
oral tablet
Stanford University
Palo Alto, California, United States
Unversity of Miami
Miami, Florida, United States
Rush University Medical Center
Chicago, Illinois, United States
Ochsner Clinic Foundation
New Orleans, Louisiana, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States
St. Elizabeth's Medical Center
Brighton, Massachusetts, United States
University at Buffalo, SUNY
Buffalo, New York, United States
University of Rochester Medical Center
Rochester, New York, United States
...and 9 more locations
Change in Patient-Reported Myasthenia Gravis Quality of Life, 15, revised ( MG-QOL15r)
Measures MG symptoms, physical, social and emotional functioning related to MG, with 15 items, 3 response option, 0-2 for each item, Total score range 0-30, higher scores indicating worse function
Time frame: Baseline, 24-36 months
Change in composite outcome of clinical improvement and adverse effects
measured by a composite of clinical improvement and adverse effects of treatments. Clinical improvement: achievement of MGFA Post-Intervention Status (PIS) Minimal Manifestation Status (MM) or better, defined below. Adverse effects end point: no more than Grade 1 CTCAE (Common Terminology Criteria for Adverse Events) medication side-effects, defined below. MGFA PIS- MM: the patient has no symptoms or functional limitations from MG but has some weakness on examination of some muscles CTCAE: list of adverse event (AE) terms commonly encountered in oncology but is useful to monitor the side effects of any intervention Each AE term is defined and graded on a 1 to 5 scale indicating the severity of the AE, 1 representing the mildest side effect and 5 representing death Grade 1 CTCAE side-effects: "asymptomatic or only mild symptoms; intervention not indicated"
Time frame: Baseline, 24-36 months
Change in Myasthenia gravis composite (MGC) scores
10 item scale of patient-reported functions and clinician-reported examination findings. Scores range from 0-50 (0- normal and 50- most severe)
Time frame: Baseline, 24-36 months
Change in Myasthenia Gravis Activities of Daily Living Scale (MG-ADL)
Patient-reported 8- item questionnaire evaluating commonly reported symptoms in MG on a 4 response scale from 0-3 (0 - normal, 3- highest disability) Range 0-24, higher score is worse
Time frame: Baseline, 24-36 months
Change in Myasthenia Gravis Manual Muscle Test scores (MG-MMT)
Clinician-assessed scale of 18 muscle functions commonly affected by MG, each graded from 0 (normal) to 4 (paralyzed/unable to perform), Range 0-120, higher score reflects worse function
Time frame: Baseline, 24-36 months
Change in Visual Analogue Scale (VAS) for Disease Severity
Patient perception of disease severity measured in millimeters on a 100 mm line. Higher number indicates more severe disease
Time frame: Baseline, 24-36 months
Change in Visual Analogue Scale (VAS) for Treatment Side effects
patient perception of side effects of treatment measured in millimeters on a 100 mm line. Higher number indicates worse side effects
Time frame: Baseline, 24-36 months
Change in number of hospitalizations for MG
counts of hospitalizations for MG- higher count is worse
Time frame: Baseline, 24-36 months
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