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Long-term efficacy and safety results of taliglucerase alfa through 5 years in adult treatment-naïve patients with Gaucher disease
| Content Provider | Scilit |
|---|---|
| Author | Zimran, Ari Durán, Gloria Giraldo, Pilar Rosenbaum, Hanna Giona, Fiorina Petakov, Milan Muñoz, Eduardo Terreros Solorio-Meza, Sergio Eduardo Cooper, Peter A. Varughese, Sheeba Alon, Sari Chertkoff, Raul |
| Copyright Year | 2019 |
| Description | Journal: Blood Cells, Molecules, and Diseases Taliglucerase alfa, the first available plant cell-expressed recombinant therapeutic protein, is an enzyme replacement therapy approved for Gaucher disease (GD). PB-06-001, a pivotal phase 3, multicenter, randomized, double-blind, parallel-dose study investigated taliglucerase alfa 30 or 60U/kg every other week through 9months in treatment-naïve adults with GD; 30-month extension study PB-06-003 followed. Patients completing PB-06-001 and PB-06-003 could continue treatment in PB-06-007. Nineteen patients enrolled in PB-06-007 (30U/kg, n=8; 60U/kg, n=9; dose adjusted, n=2); 17 completed 5 total years of treatment. In these 3 groups, respectively, taliglucerase alfa resulted in mean decreases in spleen volume (-8.7, -6.9, -12.4 multiples of normal), liver volume (-0.6, -0.4, -0.5 multiples of normal), chitotriosidase activity (-83.1%, -93.4%, -87.9%), and chemokine (CC motif) ligand 18 concentration (-66.7%, -83.3%, -78.9%), as well as mean increases in hemoglobin concentration (+2.1, +2.1, +1.8mg/dL) and platelet count (+31,871, +106,800, +34,000/mm). The most common adverse events were nasopharyngitis and arthralgia. Most adverse events were mild/moderate; no serious adverse events were considered treatment-related. These results demonstrate continued improvement of disease parameters during 5years of taliglucerase alfa therapy in 17 treatment-naive patients with no new safety concerns, extending the taliglucerase alfa clinical efficacy and safety dataset. This study was registered at www.clinicaltrials.gov as NCT01422187. |
| Related Links | https://core.ac.uk/download/pdf/82691984.pdf |
| Ending Page | 21 |
| Page Count | 8 |
| Starting Page | 14 |
| ISSN | 10799796 |
| e-ISSN | 10960961 |
| DOI | 10.1016/j.bcmd.2016.07.002 |
| Journal | Blood Cells, Molecules, and Diseases |
| Volume Number | 78 |
| Language | English |
| Publisher | Elsevier BV |
| Publisher Date | 2019-09-01 |
| Access Restriction | Open |
| Subject Keyword | Journal: Blood Cells, Molecules, and Diseases Otorhinolaryngology Chemokine (cc Motif) Ligand 18 Enzyme Replacement Therapy Taliglucerase Alfa |
| Content Type | Text |
| Resource Type | Article |
| Subject | Cell Biology Hematology Molecular Biology Molecular Medicine |
