Publication: Sporadic late-onset nemaline myopathy with monoclonal gammopathy of undetermined significance (SLONM-MGUS): An alternative treatment using cyclophosphamide-thalidomide-dexamethasone (CTD) regimen
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Issued Date
2018-07-01
Resource Type
ISSN
18732364
09608966
09608966
Other identifier(s)
2-s2.0-85048477674
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Mahidol University
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SCOPUS
Bibliographic Citation
Neuromuscular Disorders. Vol.28, No.7 (2018), 610-613
Suggested Citation
Theerawat Kumutpongpanich, Weerapat Owattanapanich, Jantima Tanboon, Ichizo Nishino, Kanokwan Boonyapisit Sporadic late-onset nemaline myopathy with monoclonal gammopathy of undetermined significance (SLONM-MGUS): An alternative treatment using cyclophosphamide-thalidomide-dexamethasone (CTD) regimen. Neuromuscular Disorders. Vol.28, No.7 (2018), 610-613. doi:10.1016/j.nmd.2018.04.011 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/46567
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Title
Sporadic late-onset nemaline myopathy with monoclonal gammopathy of undetermined significance (SLONM-MGUS): An alternative treatment using cyclophosphamide-thalidomide-dexamethasone (CTD) regimen
Abstract
© 2018 Elsevier B.V. Sporadic late-onset nemaline myopathy with monoclonal gammopathy of undetermined significance is a rare subacute adult-onset myopathy. Without appropriate treatment, the prognosis is unfavorable and can be fatal. Various efficacious treatment options have been reported. High dose melphalan followed by autologous stem cell transplantation is the most used option with favorable outcome. Nevertheless, potentially safer alternative regimens await exploration. Here, we report the case of sporadic late-onset nemaline myopathy with monoclonal gammopathy of undetermined significance in a 33-year-old man with significant clinical improvement and complete remission of monoclonal gammopathy after 5 cycles of cyclophosphamide, thalidomide, and dexamethasone regimen. The regimen may be considered as an alternative option for patients with sporadic late-onset nemaline myopathy with monoclonal gammopathy of undetermined significance that are ineligible for upfront high-dose melphalan with autologous stem cell transplantation or that are being treated in resource-limited settings. Longer-term follow-up is needed to determine the long-term effectiveness of the cyclophosphamide, thalidomide, and dexamethasone regimen.