Sporadic late onset nemaline myopathy responsive to IVIg and immunotherapy

Margherita Milone; Amiram Katz; Anthony A. Amato; Carl A. Soderland; Miruna Segarceanu; Nathan P. Young; H. Royden Jones

doi:10.1002/mus.21504

Sporadic late onset nemaline myopathy responsive to IVIg and immunotherapy

Margherita Milone, Amiram Katz, Anthony A. Amato, Carl A. Soderland, Miruna Segarceanu, Nathan P. Young, H. Royden Jones

Neurology

Research output: Contribution to journal › Article › peer-review

30 Scopus citations

Abstract

Sporadic late onset nemaline myopathy (SLONM) is a progressive myopathy of indeterminate etiology and poor outcome. If associated with a monoclonal gammopathy, SLONM carries a more unfavorable prognosis. Immunotherapy was unsuccessful. We report two HIV-negative SLONM/ monoclonal gammopathy patients who improved following intravenous immunoglobulin (IVIg) treatment alone or in combination with immunosuppressant agents. This favorable response to treatment suggests that a dysimmune mechanism is operative in some SLONM individuals. We suggest that IVIg deserves initial consideration for SLONM therapy.

Original language	English (US)
Pages (from-to)	272-276
Number of pages	5
Journal	Muscle and Nerve
Volume	41
Issue number	2
DOIs	https://doi.org/10.1002/mus.21504
State	Published - Feb 2010

Keywords

IVIg
Monoclonal gammopathy
Nemaline myopathy

ASJC Scopus subject areas

Physiology
Clinical Neurology
Cellular and Molecular Neuroscience
Physiology (medical)

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10.1002/mus.21504

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abstract = "Sporadic late onset nemaline myopathy (SLONM) is a progressive myopathy of indeterminate etiology and poor outcome. If associated with a monoclonal gammopathy, SLONM carries a more unfavorable prognosis. Immunotherapy was unsuccessful. We report two HIV-negative SLONM/ monoclonal gammopathy patients who improved following intravenous immunoglobulin (IVIg) treatment alone or in combination with immunosuppressant agents. This favorable response to treatment suggests that a dysimmune mechanism is operative in some SLONM individuals. We suggest that IVIg deserves initial consideration for SLONM therapy.",

keywords = "IVIg, Monoclonal gammopathy, Nemaline myopathy",

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AU - Milone, Margherita

AU - Katz, Amiram

AU - Amato, Anthony A.

AU - Soderland, Carl A.

AU - Segarceanu, Miruna

AU - Young, Nathan P.

AU - Jones, H. Royden

PY - 2010/2

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N2 - Sporadic late onset nemaline myopathy (SLONM) is a progressive myopathy of indeterminate etiology and poor outcome. If associated with a monoclonal gammopathy, SLONM carries a more unfavorable prognosis. Immunotherapy was unsuccessful. We report two HIV-negative SLONM/ monoclonal gammopathy patients who improved following intravenous immunoglobulin (IVIg) treatment alone or in combination with immunosuppressant agents. This favorable response to treatment suggests that a dysimmune mechanism is operative in some SLONM individuals. We suggest that IVIg deserves initial consideration for SLONM therapy.

AB - Sporadic late onset nemaline myopathy (SLONM) is a progressive myopathy of indeterminate etiology and poor outcome. If associated with a monoclonal gammopathy, SLONM carries a more unfavorable prognosis. Immunotherapy was unsuccessful. We report two HIV-negative SLONM/ monoclonal gammopathy patients who improved following intravenous immunoglobulin (IVIg) treatment alone or in combination with immunosuppressant agents. This favorable response to treatment suggests that a dysimmune mechanism is operative in some SLONM individuals. We suggest that IVIg deserves initial consideration for SLONM therapy.

KW - IVIg

KW - Monoclonal gammopathy

KW - Nemaline myopathy

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Sporadic late onset nemaline myopathy responsive to IVIg and immunotherapy

Abstract

Keywords

ASJC Scopus subject areas

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