Efficacy and Safety of Taliglucerase Alfa for the Treatment of Gaucher Disease: A 9-Year Experience
| العنوان: | Efficacy and Safety of Taliglucerase Alfa for the Treatment of Gaucher Disease: A 9-Year Experience |
|---|---|
| المؤلفون: | Livia d’Avila Paskulin, Rodrigo Tzovenos Starosta, Filippo Pinto e Vairo, Bárbara C. Krug, Paulo Picon, Ida Vanessa Doederlein Schwartz |
| المصدر: | Journal of Inborn Errors of Metabolism and Screening, Volume: 10, Article number: e20210031, Published: 13 MAY 2022 Journal of Inborn Errors of Metabolism and Screening v.10 2022 Journal of Inborn Errors of Metabolism and Screening Instituto Genética para Todos (IGPT) instacron:IGPT |
| بيانات النشر: | Latin American Society Inborn Errors and Neonatal Screening (SLEIMPN); Instituto Genética para Todos (IGPT), 2022. |
| سنة النشر: | 2022 |
| مصطلحات موضوعية: | Endocrinology, Diabetes and Metabolism, Pediatrics, Perinatology and Child Health, GBA, Gaucher disease, taliglucerase alfa, Genetics (clinical), enzyme replacement therapy |
| الوصف: | 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. |
| وصف الملف: | text/html |
| اللغة: | English |
| URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0823a4aa74caf184497e0e6c1e7eda8b http://www.scielo.br/scielo.php?script=sci_arttext&pid=S2326-45942022000100303&lng=en&tlng=en |
| حقوق: | OPEN |
| رقم الأكسشن: | edsair.doi.dedup.....0823a4aa74caf184497e0e6c1e7eda8b |
| قاعدة البيانات: | OpenAIRE |
| الوصف غير متاح. |