Article
Taliglucerase alfa in Gaucher disease: Description of a Brazilian experience
Registro en:
CRAVO, Renata et al. Taliglucerase alfa in Gaucher disease: Description of a Brazilian experience. Blood Cells, Molecules & Diseases, La Jolla, v. 68, p. 160-162, 2018.
1096-0961
10.1016/j.bcmd.2017.01.005
Autor
Cravo, Renata
Rotman, Vivian
Oliveira, Patricia Mouta
Defendi, Hugo
Conceição, D. Araujo
Xavier, Janaina Xavier
Chertkoff, Raul
Noronha, Tatiana Guimarães
Maia, Maria De Lourdes Sousa
Resumen
We evaluated retrospectively, efficacy and safety of taliglucerase alfa for Gaucher disease in a Brazilian population. Thirteen patients were included for efficacy analysis only one of them naïve to enzyme replacement therapy. All the parameters evaluated remained stable throughout treatment (mean duration 3,5 years). Only three patients (out of 35) had to discontinue treatment due to a serious adverse event. In conclusion, treatment with taliglucerase alfa was found to be safe and efficient.