TY - JOUR
T1 - Efficacy and Safety of Taliglucerase Alfa for the Treatment of Gaucher Disease
T2 - A 9-Year Experience
AU - Paskulin, Livia D’Avila
AU - Starosta, Rodrigo Tzovenos
AU - E Vairo, Filippo Pinto
AU - Krug, Bárbara C.
AU - Picon, Paulo
AU - Schwartz, Ida Vanessa Doederlein
N1 - Funding Information:
Dr. Ida Schwartz received research grants from Sanofi-Genzyme, Takeda, and Protalix.
Funding Information:
Financial support for this study was provided by Coordenação de Aperfeiçoamento de Pessoal de Nível Superior (CAPES).
Publisher Copyright:
© 2022, Latin American Society Inborn Errors and Neonatal Screening. All rights reserved.
PY - 2022
Y1 - 2022
N2 - Gaucher disease (GD) is one of the most common lysosomal disorders, occurring in approximately 1 in 40,000 live births worldwide. Since 2014 enzyme replacement therapy (ERT) with taliglucerase alfa has been the treatment of choice for adult patients with GD in Brazil. The aim of this study was to evaluate the long-term efficacy and safety of taliglucerase alfa in a cohort of Brazilian patients treated at a referral center for inborn errors of metabolism. All patients who received at least one infusion of the enzyme at the study center were considered eligible to participate. Patients were followed for adverse reactions and events throughout the study period. Platelets, hemoglobin, chitotriosidase activity, bone marrow burden (BMB) score, bone mineral density, and the severity score index (SSI) were analyzed. For patients who were switched to taliglucerase alfa from imiglucerase, the same variables were compared before and after the switch. At 9-year follow-up, all parameters of interest had remained stable or improved. The overall rate of adverse events was lower than in other studies that evaluated long-term ERT with taliglucerase, and no serious adverse events were considered related to treatment. Based on our findings, ERT with taliglucerase alfa is an effective and safe approach for treatment of patients with GD.
AB - Gaucher disease (GD) is one of the most common lysosomal disorders, occurring in approximately 1 in 40,000 live births worldwide. Since 2014 enzyme replacement therapy (ERT) with taliglucerase alfa has been the treatment of choice for adult patients with GD in Brazil. The aim of this study was to evaluate the long-term efficacy and safety of taliglucerase alfa in a cohort of Brazilian patients treated at a referral center for inborn errors of metabolism. All patients who received at least one infusion of the enzyme at the study center were considered eligible to participate. Patients were followed for adverse reactions and events throughout the study period. Platelets, hemoglobin, chitotriosidase activity, bone marrow burden (BMB) score, bone mineral density, and the severity score index (SSI) were analyzed. For patients who were switched to taliglucerase alfa from imiglucerase, the same variables were compared before and after the switch. At 9-year follow-up, all parameters of interest had remained stable or improved. The overall rate of adverse events was lower than in other studies that evaluated long-term ERT with taliglucerase, and no serious adverse events were considered related to treatment. Based on our findings, ERT with taliglucerase alfa is an effective and safe approach for treatment of patients with GD.
KW - GBA
KW - Gaucher disease
KW - enzyme replacement therapy
KW - taliglucerase alfa
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U2 - 10.1590/2326-4594-JIEMS-2021-0031
DO - 10.1590/2326-4594-JIEMS-2021-0031
M3 - Article
AN - SCOPUS:85137874054
SN - 2326-4098
VL - 10
JO - Journal of Inborn Errors of Metabolism and Screening
JF - Journal of Inborn Errors of Metabolism and Screening
M1 - e20210031
ER -