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Progressive Camptocormia With Head Drop and Dysphagia

Educational Objective
Based on this clinical scenario and the accompanying image, understand how to arrive at a correct diagnosis.
1 Credit CME

A 72-year-old man presented with mild proximal weakness developing into progressive camptocormia, head drop, and prominent dysphagia over a 2-year period. He reported a 20-kg weight loss and numbness to the ankles. His medical history was significant for thyroid cancer treated with total thyroidectomy resulting in a chronic left accessory nerve injury, type 2 diabetes, hypertension, and gout. His medications were metformin, allopurinol, perindopril, thyroxine, and rabeprazole. He had no history of smoking and drank 3 glasses of wine per week. His family history was unremarkable.

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D. Sporadic late-onset nemaline rod myopathy

Electron microscopy of the vastus lateralis muscle biopsy identified nemaline rods within several muscle fibers (Figure 2). A diagnosis was made of sporadic late-onset nemaline rod myopathy (SLONM) associated with monoclonal gammopathy. The patient received a 6-month trial of intravenous immunoglobulin without clinical improvement. He has subsequently been referred for hematological evaluation and potential autologous hematopoietic stem cell transplant.

SLONM is a rare, acquired, adult-onset progressive myopathy first described by A. G. Engel in 1966.1 The typical clinical phenotype is characterized by weakness and atrophy of predominantly proximal and axial muscles, which may lead to camptocormia or head drop. Dysphagia and dyspnea are common, occurring in approximately 50% of cases,2 with respiratory failure a common cause of death.3 Cardiac involvement is reported, manifesting as conduction abnormalities and/or cardiomyopathy.2,4

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Article Information

Corresponding Author: Shadi El-Wahsh, MD, Concord Repatriation General Hospital, Hospital Rd, Sydney 2137, Australia (shadimedhat.elwahsh@health.nsw.gov.au).

Published Online: December 12, 2022. doi:10.1001/jamaneurol.2022.4441

Conflict of Interest Disclosures: None reported.

Additional Contributions: We thank the patient for granting permission to publish this information. We also thank Roger Pamphlett, MBChB, MD, University of Sydney, for providing the light microscopy image and Renee Chan, MD, and Stephanie Sampedro, Concord Repatriation General Hospital, for providing the electron microscopy image.

References
1.
Engel  AG .  Late-onset rod myopathy: a new syndrome?   Mayo Clin Proc. 1966;41(11):713-741.PubMedGoogle Scholar
2.
Schnitzler  LJ , Schreckenbach  T , Nadaj-Pakleza  A ,  et al.  Sporadic late-onset nemaline myopathy: review of 76 cases.   Orphanet J Rare Dis. 2017;12(1):86. doi:10.1186/s13023-017-0640-2PubMedGoogle ScholarCrossref
3.
Chahin  N , Selcen  D , Engel  AG .  Sporadic late onset nemaline myopathy.   Neurology. 2005;65(8):1158-1164. doi:10.1212/01.wnl.0000180362.90078.dcPubMedGoogle ScholarCrossref
4.
Monforte  M , Primiano  G , Silvestri  G ,  et al.  Sporadic late-onset nemaline myopathy: clinical, pathology and imaging findings.   J Neurol. 2018;265(3):542-551. doi:10.1007/s00415-018-8741-yPubMedGoogle ScholarCrossref
5.
Naddaf  E , Milone  M , Kansagra  A , Buadi  F , Kourelis  T .  Sporadic late-onset nemaline myopathy: clinical spectrum, survival, and treatment outcomes.   Neurology. 2019;93(3):e298-e305. doi:10.1212/WNL.0000000000007777PubMedGoogle ScholarCrossref
6.
Agrawal  PB , Strickland  CD , Midgett  C ,  et al.  Heterogeneity of nemaline myopathy cases with skeletal muscle α-actin gene mutations.   Ann Neurol. 2004;56(1):86-96. doi:10.1002/ana.20157PubMedGoogle ScholarCrossref
7.
Uruha  A , Benveniste  O .  Sporadic late-onset nemaline myopathy with monoclonal gammopathy of undetermined significance.   Curr Opin Neurol. 2017;30(5):457-463. doi:10.1097/WCO.0000000000000477PubMedGoogle ScholarCrossref
8.
Kotchetkov  R , Susman  D , Bhutani  D , Broch  K , Dispenzieri  A , Buadi  FK .  Chemotherapy-based approach is the preferred treatment for sporadic late-onset nemaline myopathy with a monoclonal protein.   Int J Cancer. 2021;148(11):2807-2814. doi:10.1002/ijc.33483PubMedGoogle ScholarCrossref
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