Sporadic late-onset nemaline myopathy with MGUS: Long-term follow-up after melphalan and SCT.

Details

Serval ID
serval:BIB_EA4FC3472A3B
Type
Article: article from journal or magazin.
Collection
Publications
Institution
Title
Sporadic late-onset nemaline myopathy with MGUS: Long-term follow-up after melphalan and SCT.
Journal
Neurology
Author(s)
Voermans N.C., Benveniste O., Minnema M.C., Lokhorst H., Lammens M., Meersseman W., Delforge M., Kuntzer T., Novy J., Pabst T., Bouhour F., Romero N., Leblond V., Bergh P.v., Vekemans M.C., van Engelen B.G., Eymard B.
ISSN
1526-632X (Electronic)
ISSN-L
0028-3878
Publication state
Published
Issued date
2014
Peer-reviewed
Oui
Volume
83
Number
23
Pages
2133-2139
Language
english
Notes
Publication types: Journal Article
Abstract
OBJECTIVE: Sporadic late-onset nemaline myopathy (SLONM) is a rare, late-onset myopathy that progresses subacutely. If associated with a monoclonal gammopathy of unknown significance (MGUS), the outcome is unfavorable: the majority of these patients die within 1 to 5 years of respiratory failure. This study aims to qualitatively assess the long-term treatment effect of high-dose melphalan (HDM) followed by autologous stem cell transplantation (SCT) in a series of 8 patients with SLONM-MGUS.
METHODS: We performed a retrospective case series study (n = 8) on the long-term (1-8 years) treatment effect of HDM followed by autologous SCT (HDM-SCT) on survival, muscle strength, and functional capacities.
RESULTS: Seven patients showed a lasting moderate-good clinical response, 2 of them after the second HDM-SCT. All of them had a complete, a very good partial, or a partial hematologic response. One patient showed no clinical or hematologic response and died.
CONCLUSIONS: This case series shows the positive effect of HDM-SCT in this rare disorder. Factors that may portend an unfavorable outcome are a long disease course before the hematologic treatment and a poor hematologic response. Age at onset, level and type of M protein (κ vs λ), and severity of muscle weakness were not associated with a specific outcome.
CLASSIFICATION OF EVIDENCE: This study provides Class IV evidence that for patients with SLONM-MGUS, HDM-SCT increases the probability of survival and functional improvement.
Pubmed
Web of science
Create date
02/01/2015 9:29
Last modification date
20/08/2019 16:12
Usage data