Gaucher Disease Clinical Trial
Official title:
Prevalence of Autoantibodies in the Gaucher Disease and the Role of CD1 Molecules in Immune Manifestations of This Disease
Hypergammaglobulinaemia is frequently observed in type 1 Gaucher disease (GD1), being either
polyclonal or monoclonal gammopathies. Polyclonal hypergammaglobulinemia may be related to
the presence of autoantibodies. The clinical significance of such antibodies is questioned
in Gaucher disease (GD), as some cases of immunologic thrombocytopenia and autoimmune
hemolytic anemia have also been reported.
Objectives:
To evaluate the prevalence of autoantibodies and autoimmune diseases in GD1 patients, we
conducted a multicenter national study. The investigators investigated whether there was a
link between splenectomy, genotype, therapeutic options and the presence of these
autoantibodies.They also investigated whether there was a correlation with some clinical
manifestations of GD1
n/a
Observational Model: Case Control, Time Perspective: Prospective
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