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Clinical Features and Treatment Outcomes of Necrotizing Autoimmune Myopathy.
JAMA Neurol. 2015 Sep; 72(9):996-1003.JN

Abstract

IMPORTANCE

Necrotizing autoimmune myopathy (NAM) is characterized pathologically by necrotic muscle fibers with absent or minimal inflammation. It is often accompanied by statin therapy, connective tissue diseases, cancer, and autoantibodies specific for signal recognition particle (SRP) or 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR). Data are limited concerning differences among etiologic subgroups and treatment outcomes in NAM.

OBJECTIVES

To describe the clinical, serologic, and electrophysiologic characteristics of NAM, compare patient subgroups, and determine clinical outcome predictors.

DESIGN, SETTING, AND PARTICIPANTS

We conducted a retrospective review of medical records for 63 adult Mayo Clinic patients assigned the clinical and histopathologic diagnosis of NAM from January 1, 2004, through December 31, 2013. Patients were stratified by presumed cause and autoantibody status.

MAIN OUTCOMES AND MEASURES

Clinical, electrophysiologic, and pathologic characteristics were collected and compared among patient subgroups. Predictors of response to treatment were identified by univariate logistic regression.

RESULTS

Lower extremity weakness predominated (46 [73%]). Distal weakness (26 [41%]), dysphagia (22 [35%]), and dyspnea (23 [37%]) were common. Twenty-two patients (35%) were receiving a statin medication at onset, 6 had cancer, and 3 had a connective tissue disease. The median creatine kinase level was 5326 U/L. In 13 patients (24%), SRP-IgG was detected, and in 17 patients (34%), HMGCR-IgG was detected (one-third of whom had not received statin medication). One patient was dual seropositive. Facial weakness was more common in SRP-IgG-positive patients. Myotonic discharges were more common in statin-associated NAM. Prednisone monotherapy was insufficient to control disease in most patients; 30 (90%) of 32 patients required 2 or more immunotherapeutic agents. Relapse occurred in 16 (55%) of 29 patients during immunosuppressant taper or discontinuation. Predictors of favorable outcome were male sex and use of 2 or more immunotherapeutic agents within 3 months of onset.

CONCLUSIONS AND RELEVANCE

Necrotizing autoimmune myopathy was idiopathic in half of this cohort with clinical and histopathologically defined disease. In the remainder, NAM was associated with statin medication, cancer, or connective tissue disease. One in 4 patients was SRP-IgG positive, and 1 in 3 was HMGCR-IgG positive. The disease was usually not controlled by corticosteroid monotherapy. Presentation, course, and outcomes did not differ significantly in seropositive, seronegative, and statin-associated cases. Early aggressive immunosuppressant therapy improved outcomes, and risk of relapse was high during medication dose reduction or withdrawal.

Authors+Show Affiliations

Department of Neurology, Mayo Clinic, Rochester Minnesota.Department of Neurology, Mayo Clinic, Rochester Minnesota2Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, Minnesota3Department of Immunology, Mayo Clinic, Rochester, Minnesota.Department of Laboratory Medicine and Pathology, Mayo Clinic, Rochester, Minnesota.Inova Diagnostics, San Diego, California.Department of Neurology, Mayo Clinic, Rochester Minnesota.

Pub Type(s)

Journal Article

Language

eng

PubMed ID

26192196

Citation

Kassardjian, Charles D., et al. "Clinical Features and Treatment Outcomes of Necrotizing Autoimmune Myopathy." JAMA Neurology, vol. 72, no. 9, 2015, pp. 996-1003.
Kassardjian CD, Lennon VA, Alfugham NB, et al. Clinical Features and Treatment Outcomes of Necrotizing Autoimmune Myopathy. JAMA Neurol. 2015;72(9):996-1003.
Kassardjian, C. D., Lennon, V. A., Alfugham, N. B., Mahler, M., & Milone, M. (2015). Clinical Features and Treatment Outcomes of Necrotizing Autoimmune Myopathy. JAMA Neurology, 72(9), 996-1003. https://doi.org/10.1001/jamaneurol.2015.1207
Kassardjian CD, et al. Clinical Features and Treatment Outcomes of Necrotizing Autoimmune Myopathy. JAMA Neurol. 2015;72(9):996-1003. PubMed PMID: 26192196.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Clinical Features and Treatment Outcomes of Necrotizing Autoimmune Myopathy. AU - Kassardjian,Charles D, AU - Lennon,Vanda A, AU - Alfugham,Nora B, AU - Mahler,Michael, AU - Milone,Margherita, PY - 2015/7/21/entrez PY - 2015/7/21/pubmed PY - 2015/12/15/medline SP - 996 EP - 1003 JF - JAMA neurology JO - JAMA Neurol VL - 72 IS - 9 N2 - IMPORTANCE: Necrotizing autoimmune myopathy (NAM) is characterized pathologically by necrotic muscle fibers with absent or minimal inflammation. It is often accompanied by statin therapy, connective tissue diseases, cancer, and autoantibodies specific for signal recognition particle (SRP) or 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR). Data are limited concerning differences among etiologic subgroups and treatment outcomes in NAM. OBJECTIVES: To describe the clinical, serologic, and electrophysiologic characteristics of NAM, compare patient subgroups, and determine clinical outcome predictors. DESIGN, SETTING, AND PARTICIPANTS: We conducted a retrospective review of medical records for 63 adult Mayo Clinic patients assigned the clinical and histopathologic diagnosis of NAM from January 1, 2004, through December 31, 2013. Patients were stratified by presumed cause and autoantibody status. MAIN OUTCOMES AND MEASURES: Clinical, electrophysiologic, and pathologic characteristics were collected and compared among patient subgroups. Predictors of response to treatment were identified by univariate logistic regression. RESULTS: Lower extremity weakness predominated (46 [73%]). Distal weakness (26 [41%]), dysphagia (22 [35%]), and dyspnea (23 [37%]) were common. Twenty-two patients (35%) were receiving a statin medication at onset, 6 had cancer, and 3 had a connective tissue disease. The median creatine kinase level was 5326 U/L. In 13 patients (24%), SRP-IgG was detected, and in 17 patients (34%), HMGCR-IgG was detected (one-third of whom had not received statin medication). One patient was dual seropositive. Facial weakness was more common in SRP-IgG-positive patients. Myotonic discharges were more common in statin-associated NAM. Prednisone monotherapy was insufficient to control disease in most patients; 30 (90%) of 32 patients required 2 or more immunotherapeutic agents. Relapse occurred in 16 (55%) of 29 patients during immunosuppressant taper or discontinuation. Predictors of favorable outcome were male sex and use of 2 or more immunotherapeutic agents within 3 months of onset. CONCLUSIONS AND RELEVANCE: Necrotizing autoimmune myopathy was idiopathic in half of this cohort with clinical and histopathologically defined disease. In the remainder, NAM was associated with statin medication, cancer, or connective tissue disease. One in 4 patients was SRP-IgG positive, and 1 in 3 was HMGCR-IgG positive. The disease was usually not controlled by corticosteroid monotherapy. Presentation, course, and outcomes did not differ significantly in seropositive, seronegative, and statin-associated cases. Early aggressive immunosuppressant therapy improved outcomes, and risk of relapse was high during medication dose reduction or withdrawal. SN - 2168-6157 UR - https://www.unboundmedicine.com/medline/citation/26192196/Clinical_Features_and_Treatment_Outcomes_of_Necrotizing_Autoimmune_Myopathy_ L2 - https://jamanetwork.com/journals/jamaneurology/fullarticle/10.1001/jamaneurol.2015.1207 DB - PRIME DP - Unbound Medicine ER -