title: Study of enzyme replacement therapy for Gaucher Disease: comparative analysis of clinical and laboratory parameters at diagnosis and after two, five and ten years of treatment.
abstract: OBJECTIVE
  To evaluate the impact of enzyme replacement therapy for Gaucher Disease on clinical and laboratory parameters after two, five and ten years of treatment.
  
  
  METHODS
  Data were collected from patient records and analyzed using BioEstat software (version 5.0). Student's t-test, Analysis of Variance (ANOVA), Wilcoxon test and Kruskal-Wallis test were used for statistical analysis. Hepatomegaly and splenomegaly were analyzed using the Kappa test.
  
  
  RESULTS
  There was a significant increase in hemoglobin levels (p-value <0.01) and platelet counts (p-value=0.01) within two years of therapy. At the same time, the frequencies of splenomegaly (p-value <0.01) and hepatomegaly (p-value <0.05) reduced. These results were similar at five and ten years of enzyme replacement therapy.
  
  
  CONCLUSIONS
  There are substantial and quick (within two years) laboratory and clinical responses to enzyme replacement therapy. These improvements continue as long as enzyme replacement therapy is administered every two weeks, as recommended by the literature.
authors.affiliation: Universidade Federal do Pará (UFPA), Belém, PA, Brazil. Electronic address: anamas@oi.com.br.
authors.forename: Ana Maria Almeida
authors.initials: AM
authors.lastname: Souza
authors.affiliation: Universidade Federal do Pará (UFPA), Belém, PA, Brazil.
authors.forename: Thiago Pimentel
authors.initials: TP
authors.lastname: Muniz
authors.affiliation: Universidade Federal do Pará (UFPA), Belém, PA, Brazil.
authors.forename: Rafael Maciel
authors.initials: RM
authors.lastname: Brito
keywords: Anemia; Enzyme replacement therapy; Gaucher disease; Splenomegaly