Tags

Type your tag names separated by a space and hit enter

Switch from agalsidase beta to agalsidase alfa in the enzyme replacement therapy of patients with Fabry disease in Latin America.
Medicina (B Aires). 2017; 77(3):173-179.M

Abstract

There are currently two available enzyme replacement therapies for Fabry disease and little information regarding efficacy and safety of switching therapies. Between 2009 and 2012 there was a worldwide shortage of agalsidase beta and patients on that enzyme were switched to agalsidase alfa. This retrospective observational study assessed a 2-year period of efficacy and safety in a population of Fabry patients, in Argentina (30 patients) and Venezuela (3 patients), who switched therapies from algasidase beta to agalsidase alfa. Thirty-three patients completed 24-months follow-up after the switch (age 32.4 ± 2.0, range 10.0-55.9 years; male: female 23:10). Measures of renal function such as estimated glomerular filtration rate remained almost unchanged in 31 patients without end stage renal disease over the 2 years after switching and urine protein excretion continued stable. Cardiac functional parameters: left ventricular mass index, interventricular septum, left ventricular posterior wall showed no significant change from baseline in the 33 patients. Quality of life, pain and disease severity scores were mostly unchanged after 24-months and agalsidase alfa was generally well tolerated. Our findings showed there is no significant change in the efficacy measured through the renal or cardiac function, quality of life, pain, disease severity scoring and safety for at least 2 years after switching from agalsidase beta to agalsidase alfa.

Authors+Show Affiliations

Centro de Estudio de Enfermedades Lisosomales, Hospital Nacional Prof. Dr. Alejandro Posadas, Haedo, Argentina.Fundación Investigar, Buenos Aires, Argentina.Hospital Regional ángela Llano, Corrientes, Argentina.Hospital Regional ángela Llano, Corrientes, Argentina.Hospital de San José de Buga, Guadalajara, Colombia.Nefrología, Hospital General del Sur Dr. Pedro Iturbe, Maracaibo, Venezuela.Hospital Militar Central, Servicio de Genética, Buenos Aires, Argentina.Instituto de Nefrología Pergamino, Pergamino, Argentina.Centro de Estudio de Enfermedades Lisosomales, Hospital Nacional Prof. Dr. Alejandro Posadas, Haedo, Argentina. E-mail: franmasllorens@yahoo.com.ar.

Pub Type(s)

Journal Article
Multicenter Study
Observational Study
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

28643672

Citation

Ripeau, Diego, et al. "Switch From Agalsidase Beta to Agalsidase Alfa in the Enzyme Replacement Therapy of Patients With Fabry Disease in Latin America." Medicina, vol. 77, no. 3, 2017, pp. 173-179.
Ripeau D, Amartino H, Cedrolla M, et al. Switch from agalsidase beta to agalsidase alfa in the enzyme replacement therapy of patients with Fabry disease in Latin America. Medicina (B Aires). 2017;77(3):173-179.
Ripeau, D., Amartino, H., Cedrolla, M., Urtiaga, L., Urdaneta, B., Cano, M., Valdez, R., Antongiovanni, N., & Masllorens, F. (2017). Switch from agalsidase beta to agalsidase alfa in the enzyme replacement therapy of patients with Fabry disease in Latin America. Medicina, 77(3), 173-179.
Ripeau D, et al. Switch From Agalsidase Beta to Agalsidase Alfa in the Enzyme Replacement Therapy of Patients With Fabry Disease in Latin America. Medicina (B Aires). 2017;77(3):173-179. PubMed PMID: 28643672.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Switch from agalsidase beta to agalsidase alfa in the enzyme replacement therapy of patients with Fabry disease in Latin America. AU - Ripeau,Diego, AU - Amartino,Hernán, AU - Cedrolla,Martín, AU - Urtiaga,Luis, AU - Urdaneta,Bella, AU - Cano,Marilis, AU - Valdez,Rita, AU - Antongiovanni,Norberto, AU - Masllorens,Francisca, PY - 2017/6/24/entrez PY - 2017/6/24/pubmed PY - 2018/8/8/medline KW - Fabry disease KW - agalsidase alfa KW - agalsidase beta KW - switch SP - 173 EP - 179 JF - Medicina JO - Medicina (B Aires) VL - 77 IS - 3 N2 - There are currently two available enzyme replacement therapies for Fabry disease and little information regarding efficacy and safety of switching therapies. Between 2009 and 2012 there was a worldwide shortage of agalsidase beta and patients on that enzyme were switched to agalsidase alfa. This retrospective observational study assessed a 2-year period of efficacy and safety in a population of Fabry patients, in Argentina (30 patients) and Venezuela (3 patients), who switched therapies from algasidase beta to agalsidase alfa. Thirty-three patients completed 24-months follow-up after the switch (age 32.4 ± 2.0, range 10.0-55.9 years; male: female 23:10). Measures of renal function such as estimated glomerular filtration rate remained almost unchanged in 31 patients without end stage renal disease over the 2 years after switching and urine protein excretion continued stable. Cardiac functional parameters: left ventricular mass index, interventricular septum, left ventricular posterior wall showed no significant change from baseline in the 33 patients. Quality of life, pain and disease severity scores were mostly unchanged after 24-months and agalsidase alfa was generally well tolerated. Our findings showed there is no significant change in the efficacy measured through the renal or cardiac function, quality of life, pain, disease severity scoring and safety for at least 2 years after switching from agalsidase beta to agalsidase alfa. SN - 0025-7680 UR - https://www.unboundmedicine.com/medline/citation/28643672/Switch_from_agalsidase_beta_to_agalsidase_alfa_in_the_enzyme_replacement_therapy_of_patients_with_Fabry_disease_in_Latin_America_ L2 - http://www.medicinabuenosaires.com/PMID/28643672.pdf DB - PRIME DP - Unbound Medicine ER -