Juvenile Autoimmune Necrotizing Myopathies

Central Hospital, Nancy, France10 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Immune-Mediated Necrotizing Myopathy

Eligibility

Sex: ALL

Medical Language ↔ Plain English

Inclusion Criteria: * 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 Exclusion Criteria: * Seronegative necrotizing myopathies * Forms compatible with another type of myositis (e.g. dermatomyositis)

Locations (1)

Hopital d'enfants CHRU Nancy

Vandœuvre-lès-Nancy, France

Outcomes

Primary Outcomes

Type of muscle involvment

Proximal or distal or axial muscle involvement by muscle testing

Time frame: At diagnosis

Type of cutaneous involvment

Rash or Gottron lesions

Time frame: At diagnosis

Other clinical manifestations

Dysphagia or dyspnea or fever or interstitial lung involvement or articular involvement or gastrointestinal involvment

Time frame: At diagnosis

Blood CK level

Time frame: At diagnosis

Histology

Results of muscle biopsy : necrosis

Time frame: At diagnosis

Histology

Results of muscle biopsy : inflammation

Time frame: At diagnosis

MMT score

Time frame: At diagnosis

CMAS score

Time frame: At diagnosis

Secondary Outcomes

Type of drugs used

Drugs ever used : name and duration

Time frame: At last visit

Iatrogenic complications

Central Contacts

Irène LEMELLE

CONTACT

03 83 15 45 96 i.lemelle@chru-nancy.fr

Data from ClinicalTrials.gov

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