Key result
Daratumumab-based induction with autologous stem cell transplantation yields 5-year relapse-free survival in refractory SLONM.
Why the study?
Sporadic late-onset nemaline myopathy (SLONM) is a rare acquired myopathy often associated with monoclonal gammopathy, with limited data on effective treatments.
Does daratumumab-based induction followed by autologous stem cell transplantation improve outcomes in a patient with sporadic late-onset nemaline myopathy associated with smoldering myeloma?
Case Report (n=1)
No
Does daratumumab-based induction followed by autologous stem cell transplantation improve outcomes in a patient with sporadic late-onset nemaline myopathy associated with smoldering myeloma?
Daratumumab-based induction followed by autologous stem cell transplantation may be an effective treatment for sporadic late-onset nemaline myopathy associated with plasma cell dyscrasia.
Answers are grounded in this paper and cite their sources.
Daratumumab-based induction plus ASCT may benefit select SLONM cases with myeloma; leaves open confirmation in prospective trials.
Sporadic late-onset nemaline myopathy (SLONM) is a rare, acquired myopathy often associated with monoclonal gammopathy. We report a 48-year-old man presenting with progressive proximal and truncal muscle weakness in whom SLONM associated with smoldering myeloma was highly suspected. He received daratumumab, lenalidomide, and dexamethasone followed by high-dose melphalan with autologous stem cell transplantation and posttransplant therapy. Treatment resulted in marked improvement in muscle strength and performance status. The patient achieved minimal residual disease negativity and has remained relapse-free for 5 years. This case highlights the potential efficacy of daratumumab-based therapy followed by transplantation in SLONM associated with plasma cell dyscrasia. Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission.
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Moriwaki et al. (2026) conducted a case report in Sporadic late-onset nemaline myopathy (SLONM) associated with smoldering myeloma (n=1). Daratumumab, lenalidomide, and dexamethasone (DLd) followed by autologous stem cell transplantation was evaluated on Clinical response and minimal residual disease (MRD) status. Daratumumab-based induction followed by autologous stem cell transplantation led to marked improvement in muscle strength, minimal residual disease negativity, and 5 years of relapse-free survival in a patient with SLONM and smoldering myeloma.
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