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| 024 | 7 | _ | |a 10.1007/s40123-022-00611-x |2 doi |
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| 100 | 1 | _ | |a Carelli, Valerio |0 0000-0003-4923-6404 |b 0 |
| 245 | _ | _ | |a Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation |
| 260 | _ | _ | |a Heidelberg |c 2023 |b SpringerOpen |
| 336 | 7 | _ | |a article |2 DRIVER |
| 336 | 7 | _ | |a Output Types/Journal article |2 DataCite |
| 336 | 7 | _ | |a Journal Article |b journal |m journal |0 PUB:(DE-HGF)16 |s 1692958341_28577 |2 PUB:(DE-HGF) |
| 336 | 7 | _ | |a ARTICLE |2 BibTeX |
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| 336 | 7 | _ | |a Journal Article |0 0 |2 EndNote |
| 520 | _ | _ | |a Lenadogene nolparvovec is a promising novel gene therapy for patients with Leber hereditary optic neuropathy (LHON) carrying the m.11778G>A ND4 mutation (MT-ND4). A previous pooled analysis of phase 3 studies showed an improvement in visual acuity of patients injected with lenadogene nolparvovec compared to natural history. Here, we report updated results by incorporating data from the latest phase 3 trial REFLECT in the pool, increasing the number of treated patients from 76 to 174.The visual acuity of 174 MT-ND4-carrying patients with LHON injected in one or both eyes with lenadogene nolparvovec from four pooled phase 3 studies (REVERSE, RESCUE and their long-term extension trial RESTORE; and REFLECT trial) was compared to the spontaneous evolution of an external control group of 208 matched patients from 11 natural history studies.Treated patients showed a clinically relevant and sustained improvement in their visual acuity when compared to natural history. Mean improvement versus natural history was - 0.30 logMAR (+ 15 ETDRS letters equivalent) at last observation (P < 0.01) with a maximal follow-up of 3.9 years after injection. Most treated eyes were on-chart as compared to less than half of natural history eyes at 48 months after vision loss (89.6% versus 48.1%; P < 0.01) and at last observation (76.1% versus 44.4%; P < 0.01). When we adjusted for covariates of interest (gender, age of onset, ethnicity, and duration of follow-up), the estimated mean gain was - 0.43 logMAR (+ 21.5 ETDRS letters equivalent) versus natural history at last observation (P < 0.0001). Treatment effect was consistent across all phase 3 clinical trials. Analyses from REFLECT suggest a larger treatment effect in patients receiving bilateral injection compared to unilateral injection.The efficacy of lenadogene nolparvovec in improving visual acuity in MT-ND4 LHON was confirmed in a large cohort of patients, compared to the spontaneous natural history decline. Bilateral injection of gene therapy may offer added benefits over unilateral injection.NCT02652780 (REVERSE); NCT02652767 (RESCUE); NCT03406104 (RESTORE); NCT03293524 (REFLECT); NCT03295071 (REALITY). |
| 536 | _ | _ | |a 353 - Clinical and Health Care Research (POF4-353) |0 G:(DE-HGF)POF4-353 |c POF4-353 |f POF IV |x 0 |
| 588 | _ | _ | |a Dataset connected to CrossRef, Journals: pub.dzne.de |
| 650 | _ | 7 | |a Gene therapy |2 Other |
| 650 | _ | 7 | |a LHON |2 Other |
| 650 | _ | 7 | |a Leber hereditary optic neuropathy |2 Other |
| 650 | _ | 7 | |a MT-ND4 |2 Other |
| 650 | _ | 7 | |a Natural history |2 Other |
| 650 | _ | 7 | |a Visual acuity |2 Other |
| 700 | 1 | _ | |a Newman, Nancy J. |b 1 |
| 700 | 1 | _ | |a Yu-Wai-Man, Patrick |b 2 |
| 700 | 1 | _ | |a Biousse, Valerie |b 3 |
| 700 | 1 | _ | |a Moster, Mark L. |b 4 |
| 700 | 1 | _ | |a Subramanian, Prem S. |b 5 |
| 700 | 1 | _ | |a Vignal-Clermont, Catherine |b 6 |
| 700 | 1 | _ | |a Wang, An-Guor |0 P:(DE-HGF)0 |b 7 |
| 700 | 1 | _ | |a Donahue, Sean P. |b 8 |
| 700 | 1 | _ | |a Leroy, Bart P. |b 9 |
| 700 | 1 | _ | |a Sergott, Robert C. |b 10 |
| 700 | 1 | _ | |a Klopstock, Thomas |0 P:(DE-2719)2810704 |b 11 |u dzne |
| 700 | 1 | _ | |a Sadun, Alfredo A. |b 12 |
| 700 | 1 | _ | |a Rebolleda Fernández, Gema |b 13 |
| 700 | 1 | _ | |a Chwalisz, Bart K. |b 14 |
| 700 | 1 | _ | |a Banik, Rudrani |b 15 |
| 700 | 1 | _ | |a Girmens, Jean François |b 16 |
| 700 | 1 | _ | |a La Morgia, Chiara |b 17 |
| 700 | 1 | _ | |a DeBusk, Adam A. |b 18 |
| 700 | 1 | _ | |a Jurkute, Neringa |b 19 |
| 700 | 1 | _ | |a Priglinger, Claudia |b 20 |
| 700 | 1 | _ | |a Karanjia, Rustum |b 21 |
| 700 | 1 | _ | |a Josse, Constant |b 22 |
| 700 | 1 | _ | |a Salzmann, Julie |b 23 |
| 700 | 1 | _ | |a Montestruc, François |b 24 |
| 700 | 1 | _ | |a Roux, Michel |b 25 |
| 700 | 1 | _ | |a Taiel, Magali |b 26 |
| 700 | 1 | _ | |a Sahel, José-Alain |b 27 |
| 700 | 1 | _ | |a Carelli, Valerio |b 28 |
| 700 | 1 | _ | |a Barboni, Piero |b 29 |
| 700 | 1 | _ | |a Carbonelli, Michele |b 30 |
| 700 | 1 | _ | |a Di Vito, Lidia |b 31 |
| 700 | 1 | _ | |a Amore, Giulia |b 32 |
| 700 | 1 | _ | |a Contin, Manuela |b 33 |
| 700 | 1 | _ | |a Mohamed, Susan |b 34 |
| 700 | 1 | _ | |a La Morgia, Chiara |b 35 |
| 700 | 1 | _ | |a Silvestri, Sara |b 36 |
| 700 | 1 | _ | |a D’Agati, Pietro |b 37 |
| 700 | 1 | _ | |a Newman, Nancy J |b 38 |
| 700 | 1 | _ | |a Biousse, Valérie |b 39 |
| 700 | 1 | _ | |a Hubbard, George Baker |b 40 |
| 700 | 1 | _ | |a O’Keefe, Ghazala |b 41 |
| 700 | 1 | _ | |a Hendrick, Andrew M. |b 42 |
| 700 | 1 | _ | |a Dattilo, Michael |b 43 |
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| 700 | 1 | _ | |a SantaMaria, Melissa |b 67 |
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| 700 | 1 | _ | |a Subramanian, Prem S. |b 72 |
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| 700 | 1 | _ | |a Sun, Chuanbin |b 87 |
| 700 | 1 | _ | |a Donahue, Sean |b 88 |
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| 700 | 1 | _ | |a Forooza, Rod |b 132 |
| 773 | _ | _ | |a 10.1007/s40123-022-00611-x |g Vol. 12, no. 1, p. 401 - 429 |0 PERI:(DE-600)2682230-1 |n 1 |p 401 - 429 |t Ophthalmology and therapy |v 12 |y 2023 |x 2193-6528 |
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