Refractory myasthenia gravis (MG)
Conditions
Interventions
Sponsors
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
Eligibility
Sex/Gender
All
Age
No minimum to 35 Years
Inclusion criteria
Inclusion criteria: 1.Patients with MG aged =6, and meeting at least one of the following criteria for refractory MG, while demonstrating good clinical treatment adherence and receiving standardized treatment, are eligible for enrollment:; (1)Post-intervention status (PIS) of unchanged or worse after adequate doses and duration of treatment with at least two conventional immunotherapeutic agents (including corticosteroids and non-steroidal immunosuppressants). (2)PIS of improved after adequate doses and duration of treatment with at least two conventional immunotherapeutic agents, but with an MG-ADL score still >=6 persisting for at least six months. (3)PIS of remission or improvement after adequate doses and duration of treatment with at least two conventional immunotherapeutic agents, but experiencing disease exacerbation (MG-ADL score >=6) at least twice per year during regular tapering of immunotherapy. (4)Patients who, despite receiving multiple immunotherapies including intravenous immunoglobulin (IVIG), plasma exchange, and intravenous high-dose methylprednisolone (IVMP) following a crisis, along with active infection control, remain unable to wean from mechanical ventilation due to respiratory muscle weakness caused by MG for more than 14 days. 2.Informed consent must be obtained from the guardians of all enrolled pediatric patients. For patients older than 8 years, assent must also be obtained from the patient using a pediatric version of the informed consent form, in addition to the guardian's signed consent. Patients over 18 years of age must sign the adult version of the informed consent form. Enrollment and treatment will be conducted in accordance with ethical requirements.
Exclusion criteria
Exclusion criteria: Patients meeting any of the following criteria will be excluded from participation in this study:; 1.Expected survival period < 2 weeks. 2.No suitable veins for peripheral blood lymphocyte collection or insufficient quantity of cells collected. 3.Presence of uncontrolled active infections, such as active hepatitis B or C, HIV, fungal infections, tuberculosis, EBV, or CMV infection. 4.Use of immunomodulatory agents affecting T-cell function (e.g., Rituximab) within the past 3 months. 5.Poor in vitro expansion of lymphocytes after isolation, failing to reach the clinical dosage requirement. 6.Lost to follow-up early after treatment (< 3 months), making near- and long-term prognosis evaluation impossible. 7.Presence of other systemic diseases that could interfere with the study. 8.Any other condition considered by the investigator as grounds for exclusion.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Cytoxicity Assessment;Myasthenia Gravis (MG)-Associated Autoantibody Testing;Electromyography (EMG); | — |
Secondary
| Measure | Time frame |
|---|---|
| CAR-T cell expansion and persistence;Quantitative Myasthenia Gravis Score (QMG) and the Myasthenia Gravis Activities of Daily Living (MG-ADL); | — |
Countries
China
Contacts
Public ContactLi Benshang; Wang Jiwen
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
Outcome results
None listed