Document Detail

Effect of enzyme replacement therapy on gammopathies in Gaucher disease.
MedLine Citation:
PMID:  14757437     Owner:  NLM     Status:  MEDLINE    
Chronic antigenic stimulation by the abnormal lipid storage has been postulated to be the mechanism underlying anecdotal reports of monoclonal and polyclonal gammopathies as well as an increased incidence of multiple myeloma in patients with Gaucher disease of all ages. With the advent of specific enzyme therapy, it has been possible to ascertain whether signs and symptoms associated with Gaucher disease are true features of the disorder by virtue of their responsiveness to treatment. The purpose of this study was to assess the incidence of polyclonal and monoclonal gammopathies in a large cohort of patients and the effect of enzyme treatment. All adult patients whose records of immunoglobulin levels were available at presentation or at the advent of enzyme replacement therapy (ERT), and who had been followed for 2 years or receiving ERT for at least 2 years, respectively, and for whom there were also immunoglobulin levels at their most recent follow-up, were included in the study. The incidence of polyclonal gammopathies ranged between 14% and 25% among treated and untreated patients. There were statistically significant percentage decreases per year of enzyme therapy in polyclonal but not monoclonal (1% of all patients) gammopathies. Among enzyme-treated patients, there was no statistically significant difference among patients with regard to spleen status or relative to other parameters of disease severity, hepatitis status, age or gender. This study represents the largest database of gammopathies among patients with Gaucher disease from a large referral clinic. Because there was no correlation of abnormal immunoglobulin levels with disease severity, etiology may not be related to lipid accumulation per se but perhaps reflects a secondary, enzyme-sensitive process, whereas monoclonal gammopathies remain unaffected.
Ariel Brautbar; Deborah Elstein; Guy Pines; Aya Abrahamov; Ari Zimran
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Publication Detail:
Type:  Journal Article    
Journal Detail:
Title:  Blood cells, molecules & diseases     Volume:  32     ISSN:  1079-9796     ISO Abbreviation:  Blood Cells Mol. Dis.     Publication Date:    2004 Jan-Feb
Date Detail:
Created Date:  2004-02-03     Completed Date:  2004-09-30     Revised Date:  2009-11-19    
Medline Journal Info:
Nlm Unique ID:  9509932     Medline TA:  Blood Cells Mol Dis     Country:  United States    
Other Details:
Languages:  eng     Pagination:  214-7     Citation Subset:  IM    
Gaucher Clinic, Shaare Zedek Medical Center, Jerusalem 91031, Israel.
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MeSH Terms
Aged, 80 and over
Enzymes / pharmacology,  therapeutic use*
Follow-Up Studies
Gaucher Disease / drug therapy*
Immunoglobulins / blood,  drug effects
Middle Aged
Monoclonal Gammopathy of Undetermined Significance / drug therapy*
Treatment Outcome
Reg. No./Substance:
0/Enzymes; 0/Immunoglobulins

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