myasthenia gravis
Conditions
Interventions
Efgartigimod therapy group:None
Sponsors
Affiliated Hospital of Zunyi Medical University
Eligibility
Sex/Gender
All
Age
18 Years to 80 Years
Inclusion criteria
Inclusion criteria: (1) Patients aged 18-80 years, diagnosed with generalized myasthenia gravis (gMG) according to the 2020 edition of the "Chinese Guidelines for the Diagnosis and Treatment of Myasthenia Gravis," and treated with intravenous infusion of efgartigimod at 10 mg/kg; (2) Maintaining any of the following stable standard treatment regimens (used alone or in combination):1). Cholinesterase inhibitors;2).Glucocorticoids;3). Immunosuppressants; (3) Serological testing positive for AChR-Ab/MuSK-Ab/LRP4-Ab/titin-Ab/RyR-Ab; (4) Myasthenia Gravis Foundation of America (MGFA) clinical classification type II-IV; (5) Myasthenia Gravis Activities of Daily Living (MG-ADL) score >= 6, with ocular-related scores accounting for less than 50% of the total score; Quantitative Myasthenia Gravis (QMG) score >= 8, with at least 4 items scoring 2 points or higher; (6) Duration from exposure to efgartigimod to the last visit is at least 6 months.
Exclusion criteria
Exclusion criteria: (1) Active hepatitis B, positive for hepatitis C antibody, positive for HIV antibody, or active tuberculosis; (2) Female patients who are pregnant, lactating, or planning to conceive during the trial period; (3) Patients deemed by the investigator to be unsuitable for trial participation (e.g., those with severe psychiatric disorders); (4) Patients with incomplete clinical data at baseline.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The proportion of patients achieving Minimal Symptom Expression (MSE) during the first treatment cycle; | — |
Secondary
| Measure | Time frame |
|---|---|
| Proportion of patients with MG-ADL responses in the first treatment cycle.;Changes in MG-ADL scores from baseline at 4, 8, 12, 16, 20, and 24 weeks after initiation of Efgartigimod.;Change in prednisone dose from baseline at weeks 4, 8, 12, 16, 20, and 24 following Efgartigimod initiation.;Change in pyridostigmine dose from baseline at weeks 4, 8, 12, 16, 20, and 24 after Efgartigimod initiation.;Proportion of rehospitalized patients due to disease worsening (new symptoms or MG-ADL score increase =2) within 24 weeks.; | — |
Countries
China
Contacts
Public ContactZhong Luo
Affiliated Hospital of Zunyi Medical University
Outcome results
None listed