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This study is designed to explore the efficacy and safety of Telitacicept combined with low-dose steroids for the treatment of refractory MG, and to investigate related biomarkers such as immunoglobulins, BlyS/APRIL, and AChR-Ab titers, in order to clarify whether Telitacicept can rapidly and effectively help achieve MG treatment goals and assist in steroid reduction.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patients with refractory MG treated with Telitacicept combined with low-dose steroids | Experimental | This is an open-label, single-arm exploratory study of Telitacicept (240mg weekly, then every two weeks after achieving MMS or QMG reduction of ≥ 6 points) combined with a gradual reduction of steroids and other immunosuppressants. When the steroid dose is reduced to 5mg/day or 10mg/every other day, Telitacicept can be reduced to 160mg, administered subcutaneously every two weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Telitacicept | Drug | Patients with MG who fulfill the inclusion criteria will receive Telitacicept 240mg weekly as an adjunct to their medication. Upon reaching MMS, significant symptom improvement, or a QMG score reduction of at least 6 points, Telitacicept is reduced to biweekly doses. Pyridostigmine and NSISTs are tapered based on patient response. Following this, Prednisone is tapered, starting with rapid reduction early and slowing later. If a patient on 60mg Prednisone daily achieves treatment goals within 6-8 weeks of Telitacicept initiation, the tapering sequence is 60mg every other day for 4 weeks, then 30mg daily for 2-4 weeks, and so on, until reaching 5mg daily or 10mg every other day. At this point, Telitacicept may be reduced to 160mg biweekly. |
| Measure | Description | Time Frame |
|---|---|---|
| The change in patients' ADL scores | The variation in ADL scores at 52 weeks compared to baseline in patients. Total MG -ADL scores range from 0 (normal) to 24 (severe). | from baseline to week 52 |
| The change in patients' QMG scores | The variation in QMG scores at 52 weeks compared to baseline in patients. Total QMG scores range from 0 (none) to 39 (severe). | from baseline to week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| MGFA-PIS | Change in MGFA-PIS grading from baseline to week 52 | from baseline to week 52 |
| the average daily corticosteroid usage | Change in the average daily corticosteroid usage from baseline to week 24 during follow-up；Change in the average daily corticosteroid usage from baseline to week 52 during follow-up |
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Inclusion Criteria:
Age between 18-85 years, both genders included；
Meet the diagnostic criteria of the 2020 Chinese MG guidelines, with positive serological testing for AChR-Ab;
Clinical classification of Type I to Type IVa according to the MGFA;
Meet the criteria for refractory MG in the 2022 Japanese MG guidelines: poor response to standard treatment, intolerance to standard treatment drugs due to adverse reactions, frequent relapses/exacerbations after reduction of standard treatment drugs, frequent need for rescue treatment due to disease fluctuations, frequent myasthenic crises, and comorbidities that limit the use of standard treatment;
Patients with unstable symptoms (MG-ADL score ≥6 or QMG ≥8) despite treatment with standard therapeutic regimens before enrollment, defined as follows:
Patients must provide written informed consent.
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Jia Li, Master's Degree | Contact | 19016577562 | lijia@wzhospital.cn |
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| ID | Term |
|---|---|
| D009157 | Myasthenia Gravis |
| D001327 | Autoimmune Diseases |
| ID | Term |
|---|---|
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
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| ID | Term |
|---|---|
| C000722462 | telitacicept |
| D011729 | Pyridostigmine Bromide |
| D011241 | Prednisone |
| ID | Term |
|---|---|
| D011726 | Pyridinium Compounds |
| D011725 | Pyridines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
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|
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| from baseline to week 24；from baseline to week 52 |
| the usage of traditional non-steroidal immunosuppressants | Change in the usage of traditional non-steroidal immunosuppressants from baseline to week 24 during follow-up; Change in the usage of traditional non-steroidal immunosuppressants from baseline to week 52 during follow-up. Medication usage logs or medical record reviews to document the changes in the use of traditional non-steroidal immunosuppressants (such as Azathioprine, Mycophenolate Mofetil, Tacrolimus, Methotrexate, Cyclosporine, etc.) from baseline to week 52. The change in the use of traditional non-steroidal immunosuppressants will be assessed by comparing the medication usage at baseline and at weeks 24 and 52. This includes details on the type of medication, dosage, frequency, and any adjustments or discontinuations. | from baseline at weeks 24 and 52 |
| Number of relapses | Number of relapses in both groups by the end of treatment (clinical relapse is defined as an increase in QMG score of ≥3 points); | week 52 |
| AUDTC | Area under the corticosteroid dose-time curve (AUDTC) at week 52 | week 52 |
| Proportion of patients with a corticosteroid dose ≤10mg/d | Proportion of patients with a corticosteroid dose ≤10mg/d at weeks 24 and 52 during follow-up; | at weeks 24 and 52 |
| Proportion of patients achieving MMS with a corticosteroid dose ≤ 5mg/d | Proportion of patients achieving MMS with a corticosteroid dose ≤5mg/d at week 24; | week 24 |
| MG QoL15r scores | Change in MG QoL15r scores from baseline at weeks 24 and 52 during follow-up | from baseline at weeks 24 and 52 |
| safety（Incidence, severity, and outcome of adverse events） | Incidence, severity, and outcome of adverse events | 52 weeks |
| relevant biomarkers | relevant biomarkers, such as immunoglobulins,lymphocyte subpopulation changes, BlyS/APRIL, and AChR-Ab titers. | from baseline at week 52 |
| D010257 | Paraneoplastic Syndromes |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D007154 | Immune System Diseases |
| D011244 |
| Pregnadienediols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |