A Prospective Cohort Study of Myasthenia Gravis in China

Peking Union Medical College Hospital202 enrolled

Overview

The goal of this prospective cohort study is to investigate long-term therapeutic strategies for myasthenia gravis (MG) and identify potential biomarkers. The main questions it aims to answer are: 1. Whether low-dose oral steroids may lead to a reduction in the recurrence rate among patients with MG. 2. To identify potential biomarkers that can predict disease progression and prognosis. This study recruits well-controlled patients with MG. Based on patient preferences and considerations such as coexisting conditions (e.g., uncontrolled hypertension, diabetes, severe osteoporosis, obesity), the participants will be non-randomly divided into two groups: a maintenance steroid therapy group and a withdrawal group (withdraw all immunosuppresants). Subsequently, these groups of patients will undergo long-term follow-up assessments.

Study Type

OBSERVATIONAL

Enrollment

202

Conditions

Myasthenia Gravis

Interventions

Steroid DrugDRUG

Maintaining low-dose oral steroids

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * 1: Attended the outpatient department of neurology of PUMCH from 9/30/2022 to 9/30/2024. * 2: Diagnosed as myasthenia gravis. * 3: Follow-up time at PUMCH\>6 months. * 4: The patient understood and signed the informed consent form. Exclusion Criteria: * 1: Comorbidities with other conditions that cause skeletal muscle weakness make the clinical symptoms difficult to assess * 2: Records related to comorbidities and medications were not available at baseline and during follow-up.

Outcomes

Primary Outcomes

Relapse of myasthenia gravis

One of the following three is met: (1) MGFA Postintervention Status: failure to maintain MMS (minimal manifestations status) or better. (2) Myasthenia gravis activities of daily living (MG-ADL) score ≥ 3; (3) Re-administration of cholinesterase inhibitors was required after achieving Complete Stable Remission (CSR) on the MGFA Postintervention Status.

Time frame: Within two years after recruitment

Secondary Outcomes

Time of relapse

The time between recruitment and relapse of MG.

Time frame: Within two years after recruitment

Change of MG assessment scales at relapse

Change of MG assessment scales at relapse, including MGFA PIS (Postintervention Status), MG-ADL (myasthenia gravis activities of daily living), QMG (Quantitative Myasthenia gravis) and MGC (Myasthenia gravis composite).

Time frame: Within two years after recruitment

Adverse events of steroids

Using CTCAE (Common Terminology Criteria for Adverse Events) to assess Adverse events.

Time frame: Within two years after recruitment

A Prospective Cohort Study of Myasthenia Gravis in China

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts