Chemotherapy‐based approach is the preferred treatment for sporadic late‐onset nemaline myopathy with a monoclonal protein

Sporadic late‐onset nemaline myopathy (SLONM) associated with monoclonal protein (MP) is a rare disease with an aggressive, and often fatal course. Whether SLONM + MP represents a malignancy or dysimmune disease remains unclear. Currently, two main approaches are used to treat SLONM + MP: nonchemoth...

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Bibliographic Details
Published in:International journal of cancer 2021-06, Vol.148 (11), p.2807-2814
Main Authors: Kotchetkov, Rouslan, Susman, David, Bhutani, Divaya, Broch, Kaspar, Dispenzieri, Angela, Buadi, Francis K.
Format: Article
Language:English
Subjects:
Autografts
autologous stem cell transplantation
Cancer
Chemotherapy
gammopathy
Hematology
Immunoglobulins
Immunosuppression
immunotherapy
Intravenous administration
Malignancy
Medical research
monoclonal protein
Myopathy
Nemaline myopathy
Patients
Plasmapheresis
Rare diseases
Remission
SLONM
Stem cell transplantation
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Summary:Sporadic late‐onset nemaline myopathy (SLONM) associated with monoclonal protein (MP) is a rare disease with an aggressive, and often fatal course. Whether SLONM + MP represents a malignancy or dysimmune disease remains unclear. Currently, two main approaches are used to treat SLONM + MP: nonchemotherapy‐based treatment (immunosuppression, intravenous immunoglobulins, plasmapheresis and plasma exchange) or chemotherapy with or without autologous stem cell transplantation. Due to the rare occurrence of the disease, the best treatment modality is unknown. We analyzed treatment and outcomes in a large cohort of 53 patients with SLONM + MP: four our own patients and 49 cases from published literature. Neurological improvement in the nonchemotherapy group (N = 25) was observed in 52% of patients: 8% reached marked improvement, 8% moderate response, 36% mild response; none reached complete remission (CR). In the chemotherapy group (N = 28), neurological improvement was seen in 86% of patients: 46% reached CR, 25% marked response, 11% moderate response and 4% mild response. The best neurological improvement correlated with deep hematological remission. Mean time to best response in the chemotherapy group was 8 months versus 21 months in the nonchemotherapy group (P 
ISSN:0020-7136
1097-0215
DOI:10.1002/ijc.33483