Sporadic late‐onset nemaline myopathy as a rare cause of slowly progressive muscle weakness with young adult onset |
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| Authors: | Meiko Hashimoto Maeda MD PhD Hikari Ohta MD Koji Izutsu MD PhD Jun Shimizu MD PhD Yoshikazu Uesaka MD PhD |
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| Affiliation: | 1. Department of Neurology, Federation of National Public Service Personnel Mutual Aid Associations Toranomon Hospital, Tokyo, Japan;2. Department of Neurology, University of Tokyo, Graduate School of Medicine, Tokyo, Japan;3. Okinaka Memorial Institute for Medical Research, Tokyo, Japan;4. Department of Haematology, Federation of National Public Service Personnel Mutual Aid Associations, Toranomon Hospital, Tokyo, Japan |
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| Abstract: | Introduction: Sporadic late‐onset nemaline myopathy (SLONM) is a rare intractable acquired myopathy characterized by progressive muscle weakness and atrophy, usually with middle to late adult onset. Autologous peripheral blood stem cell transplantation (auto‐PBSCT) has been reported to be a promising treatment for SLONM. Methods: In this study we performed clinical characterization, muscle histopathological analysis, and muscle power monitoring after auto‐PBSCT in a 27‐year‐old HIV‐negative man with monoclonal gammopathy. Results: He showed improved muscle strength after treatment with high‐dose melphalan and auto‐PBSCT. Conclusions: Considering the recent reports of successful treatment of SLONM, early and correct diagnosis of this condition in association with monoclonal gammopathy is important. SLONM should be added to the list of diseases to consider in the differential diagnosis of progressive muscle weakness with young adult onset. Muscle Nerve 51 :772–774, 2015 |
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| Keywords: | auto‐PBSCT HIV‐negative patients monoclonal gammopathy sporadic late‐onset nemaline myopathy young adult onset |
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