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Long‐term efficacy and safety results of taliglucerase alfa up to 36 months in adult treatment‐naïve patients with Gaucher disease
| Content Provider | Semantic Scholar |
|---|---|
| Author | Zimran, Ari Durán, Gloria Mehta, Atul Giraldo, Pilar Rosenbaum, Hanna Giona, Fiorina Amato, Dominick J. Petakov, Milan S. Muñoz, Eduardo Terreros Solorio-Meza, Sergio Cooper, Peter A. Varughese, Sheeba Chertkoff, Raul Almon, Einat Brill‐ |
| Copyright Year | 2016 |
| Abstract | Taliglucerase alfa is an intravenous enzyme replacement therapy approved for treatment of type 1 Gaucher disease (GD), and is the first available plant cell-expressed recombinant therapeutic protein. Herein, we report long-term safety and efficacy results of taliglucerase alfa in treatment-naïve adult patients with GD. Patients were randomized to receive taliglucerase alfa 30 or 60 U/kg every other week, and 23 patients completed 36 months of treatment. Taliglucerase alfa (30 U/kg; 60 U/kg, respectively) resulted in mean decreases in spleen volume (50.1%; 64.6%) and liver volume (25.6%; 24.4%) with mean increases in hemoglobin concentration (16.0%; 35.8%) and platelet count (45.7%; 114.0%), and mean decreases in chitotriosidase activity (71.5%; 82.2%). All treatment-related adverse events were mild to moderate in intensity and transient. The most common adverse events were nasopharyngitis, arthralgia, upper respiratory tract infection, headache, pain in extremity, and hypertension. These 36-month results of taliglucerase alfa in treatment-naïve adult patients with GD demonstrate continued improvement in disease parameters with no new safety concerns. These findings extend the taliglucerase alfa clinical safety and efficacy dataset. www.clinicaltrials.gov identifier NCT00705939. Am. J. Hematol. 91:656-660, 2016. © 2016 Wiley Periodicals, Inc. |
| Starting Page | 656 |
| Ending Page | 660 |
| Page Count | 5 |
| File Format | PDF HTM / HTML |
| DOI | 10.1002/ajh.24369 |
| PubMed reference number | 27174694 |
| Journal | Medline |
| Volume Number | 91 |
| Alternate Webpage(s) | https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/70/86/AJH-91-656.PMC5074246.pdf |
| Alternate Webpage(s) | https://doi.org/10.1002/ajh.24369 |
| Journal | American journal of hematology |
| Language | English |
| Access Restriction | Open |
| Content Type | Text |
| Resource Type | Article |
