Real-World Safety and Effectiveness of Voretigene Neparvovec Results up to 2 Years from the Prospective, Registry-Based PERCEIVE Study /
Voretigene neparvovec (VN) is the first available gene therapy for patients with biallelic RPE65-mediated inherited retinal dystrophy who have sufficient viable retinal cells. PERCEIVE is an ongoing, post-authorization, prospective, multicenter, registry-based observational study and is the largest...
Elmentve itt :
| Szerzők: | |
|---|---|
| Dokumentumtípus: | Cikk |
| Megjelent: |
2024
|
| Sorozat: | BIOMOLECULES
14 No. 1 |
| Tárgyszavak: | |
| doi: | 10.3390/biom14010122 |
| mtmt: | 35727058 |
| Online Access: | http://publicatio.bibl.u-szeged.hu/36896 |
| LEADER | 02757nab a2200397 i 4500 | ||
|---|---|---|---|
| 001 | publ36896 | ||
| 005 | 20250529104739.0 | ||
| 008 | 250529s2024 hu o 000 eng d | ||
| 022 | |a 2218-273X | ||
| 024 | 7 | |a 10.3390/biom14010122 |2 doi | |
| 024 | 7 | |a 35727058 |2 mtmt | |
| 040 | |a SZTE Publicatio Repozitórium |b hun | ||
| 041 | |a eng | ||
| 100 | 1 | |a Fischer M. Dominik | |
| 245 | 1 | 0 | |a Real-World Safety and Effectiveness of Voretigene Neparvovec |h [elektronikus dokumentum] : |b Results up to 2 Years from the Prospective, Registry-Based PERCEIVE Study / |c Fischer M. Dominik |
| 260 | |c 2024 | ||
| 300 | |a 15 | ||
| 490 | 0 | |a BIOMOLECULES |v 14 No. 1 | |
| 520 | 3 | |a Voretigene neparvovec (VN) is the first available gene therapy for patients with biallelic RPE65-mediated inherited retinal dystrophy who have sufficient viable retinal cells. PERCEIVE is an ongoing, post-authorization, prospective, multicenter, registry-based observational study and is the largest study assessing the real-world, long-term safety and effectiveness of VN. Here, we present the outcomes of 103 patients treated with VN according to local prescribing information. The mean (SD) age was 19.5 (10.85) years, 52 (50.5%) were female, and the mean (SD) duration of the follow up was 0.8 (0.64) years (maximum: 2.3 years). Thirty-five patients (34%) experienced ocular treatment-emergent adverse events (TEAEs), most frequently related to chorioretinal atrophy (n = 13 [12.6%]). Eighteen patients (17.5%; 24 eyes [13.1%]) experienced ocular TEAEs of special interest, including intraocular inflammation and/or infection related to the procedure (n = 7). The mean (SD) changes from baseline in full-field light-sensitivity threshold testing (white light) at month 1, month 6, year 1, and year 2 were -16.59 (13.48) dB (51 eyes), -18.24 (14.62) dB (42 eyes), -15.84 (14.10) dB (10 eyes), and -13.67 (22.62) dB (13 eyes), respectively. The change in visual acuity from baseline was not clinically significant. Overall, the outcomes of the PERCEIVE study are consistent with the findings of VN pivotal clinical trials. | |
| 650 | 4 | |a Klinikai orvostan | |
| 700 | 0 | 1 | |a Simonelli Francesca |e aut |
| 700 | 0 | 1 | |a Sahni Jayashree |e aut |
| 700 | 0 | 1 | |a Holz Frank G. |e aut |
| 700 | 0 | 1 | |a Maier Rainer |e aut |
| 700 | 0 | 1 | |a Fasser Christina |e aut |
| 700 | 0 | 1 | |a Suhner Andrea |e aut |
| 700 | 0 | 1 | |a Stiehl Daniel P. |e aut |
| 700 | 0 | 1 | |a Chen Bee |e aut |
| 700 | 0 | 1 | |a Audo Isabelle |e aut |
| 700 | 0 | 1 | |a Leroy Bart P. |e aut |
| 700 | 0 | 1 | |a Khan Arif O. |e aut |
| 700 | 0 | 2 | |a Tóth-Molnár Edit |e aut |
| 700 | 0 | 2 | |a Nagy Zoltán Zsolt |e aut |
| 700 | 0 | 2 | |a Kollaborációs szervezet: PERCEIVE Study Group |e aut |
| 700 | 0 | 2 | |a et al. |e aut |
| 856 | 4 | 0 | |u http://publicatio.bibl.u-szeged.hu/36896/1/35727058.pdf |z Dokumentum-elérés |