Clinical, Paraclinical, Functional Characteristics and Evolution of Juvenile Autoimmune Necrotizing Myopathies in a National Retrospective Cohort
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Histology
研究概览
简要总结
Autoimmune necrotizing myopathies (AINM) in adult patients are characterized by severity of muscle damage, presence of necrosis with little inflammation on muscle biopsy and anti-HMGCR or anti-SRP auto-antibodies. Data on AINM in children are currently lacking. The purpose of this study is to specify the characteristics at AINM diagnosis, treatments and evolution of juvenile AINM with anti-HMGCR or anti-SRP antibodies.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Retrospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •AINM histologically proved by predominant muscle fibers necrosis lesions associated with minimal or absent inflammatory lesions
- •Either anti-SRP or anti-HMGCR antibodies positivity
- •Disease onset before age 18
排除标准
- •Seronegative necrotizing myopathies
- •Forms compatible with another type of myositis (e.g. dermatomyositis)
结局指标
主要结局
Histology
时间窗: At diagnosis
Results of muscle biopsy : inflammation
Type of cutaneous involvment
时间窗: At diagnosis
Rash or Gottron lesions
Type of muscle involvment
时间窗: At diagnosis
Proximal or distal or axial muscle involvement by muscle testing
MMT score
时间窗: At diagnosis
MMT score
Blood CK level
时间窗: At diagnosis
Blood CK level
Other clinical manifestations
时间窗: At diagnosis
Dysphagia or dyspnea or fever or interstitial lung involvement or articular involvement or gastrointestinal involvment
CMAS score
时间窗: At diagnosis
CMAS score
次要结局
- CMAS score(At last visit)
- Iatrogenic complications(At last visit)
- Number of relapses(At last visit)
- Type of muscle involvment(At last visit)
- MMT score(At last visit)
- Status of the disease at last visit(At last visit)
- Type of cutaneous involvment(At last visit)
- Other clinical manifestations(At last visit)
- Type of drugs used(At last visit)
- Blood CK level(At last visit)