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An In Vitro Model for the Study of Cellular Pathophysiology in Globoid Cell Leukodystrophy
Published on: October 21, 2014
IgM monoclonal component associated with type I Gaucher disease resolved after enzyme replacement therapy: a case
C Martinez-Redondo1, F J Ortuño, M L Lozano
1Hematology and Clinical Oncology Unit, Hospital Universitario J.M. Morales Meseguer, Marqués de los Vélez s/n, 30008, Murcia, Spain.
Abstract:
The frequency of monoclonal gammopathy of undetermined significance (MGUS) is higher in patients with type I Gaucher disease (GD I) than in the general population. Although enzyme replacement therapy is effective in the control of the disease, its effect on MGUS is still controversial. We present the case of a 65-year-old woman with extensive GD I associated with IgM MGUS, in whom enzyme replacement therapy succeeded in eradicating the monoclonal component. This observation further supports the idea that enzyme replacement therapy decreases the chronic antigenic stimulation responsible for gammopathies in Gaucher disease.
