Summary

Neuromuscul Disord. 2018 Jul;28(7):610-613. doi: 10.1016/j.nmd.2018.04.011. Epub 2018 May 16.

Sporadic late-onset nemaline myopathy with monoclonal gammopathy of undetermined significance (SLONM-MGUS): An alternative treatment using cyclophosphamide-thalidomide-dexamethasone (CTD) regimen.

Abstract:

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.

日本語要旨:

M蛋白血症を伴う孤発性成人発症型ネマリンミオパチー(SLONM-MGUS)は進行性の筋力低下を呈する稀な疾患であり、未治療では予後不良で死に至る可能性がある。高容量メルファラン療法に続く自己末梢血幹細胞移植が最も治療効果が高い選択肢とされているが、より安全性の高い治療法が期待されている。我々はシクロフォスファミド・サリドマイド・デキサメサゾン療法(5サイクル)で完全寛解に至った33歳男性を経験した。高容量メルファラン療法に続く自己末梢血幹細胞移植に適応がない、もしくは、なんらかの制限を要する症例に対する新たな選択肢になる可能性がある。

PMID:  29910095

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