%0 Journal Article
%A Carelli, Valerio
%A Newman, Nancy J.
%A Yu-Wai-Man, Patrick
%A Biousse, Valerie
%A Moster, Mark L.
%A Subramanian, Prem S.
%A Vignal-Clermont, Catherine
%A Wang, An-Guor
%A Donahue, Sean P.
%A Leroy, Bart P.
%A Sergott, Robert C.
%A Klopstock, Thomas
%A Sadun, Alfredo A.
%A Rebolleda Fernández, Gema
%A Chwalisz, Bart K.
%A Banik, Rudrani
%A Girmens, Jean François
%A La Morgia, Chiara
%A DeBusk, Adam A.
%A Jurkute, Neringa
%A Priglinger, Claudia
%A Karanjia, Rustum
%A Josse, Constant
%A Salzmann, Julie
%A Montestruc, François
%A Roux, Michel
%A Taiel, Magali
%A Sahel, José-Alain
%A Carelli, Valerio
%A Barboni, Piero
%A Carbonelli, Michele
%A Di Vito, Lidia
%A Amore, Giulia
%A Contin, Manuela
%A Mohamed, Susan
%A La Morgia, Chiara
%A Silvestri, Sara
%A D’Agati, Pietro
%A Newman, Nancy J
%A Biousse, Valérie
%A Hubbard, George Baker
%A O’Keefe, Ghazala
%A Hendrick, Andrew M.
%A Dattilo, Michael
%A Peragallo, Jason H.
%A Hawy, Eman
%A DuBois, Lindreth
%A Gibbs, Deborah
%A Filho, Alcides Fernandes
%A Dobbs, Jannah
%A Yu-Wai-Man, Patrick
%A Acheson, James
%A Boston, Hayley
%A Eleftheriadou, Maria
%A Esposti, Simona
%A Gemenetzi, Maria
%A Leitch-Devlin, Lauren
%A Tucker, William R.
%A Jurkute, Neringa
%A Burale, Asma
%A Anand, Shweta
%A Memon, Muhammad A.
%A Hussain, Rima
%A Jorany, Rasha
%A Sheel, Priyansha
%A Moster, Mark L.
%A Sergott, Robert C.
%A SantaMaria, Melissa
%A Tollis, Heather
%A DeBusk, Adam A
%A Haller, Julia A.
%A Massini, Maria
%A Subramanian, Prem S.
%A Pecen, Paula
%A Mathias, Marc
%A Preston, Mary
%A Cho, Steve
%A Sahel, José A.
%A Vignal-Clermont, Catherine
%A Girmens, Jean François
%A Hage, Rabih
%A Plaine, Lise
%A Khemliche, Wahiba
%A Wang, An-Guor
%A Cheng, Hui-Chen
%A Chen, Celia
%A Hwang, Jeong-Min
%A Sun, Chuanbin
%A Donahue, Sean
%A Patel, Shriji
%A Gangaputra, Sapna
%A Barrett, Megan
%A Ruark, Scott
%A Wilkins, Saige
%A Leroy, Bart P.
%A De Zaeytijd, Julie
%A Van Cauwenbergh, Caroline
%A Verhauwen, Hilde
%A Klopstock, Thomas
%A Catarino, Claudia B.
%A Priglinger, Claudia
%A Priglinger, Siegfried
%A Rudolph, Günther
%A von Livonius Bettina, Stephan Thurau
%A Muth, Daniel
%A Wolf, Armin
%A Al-Tamami, Jasmina
%A Pressler, Angelika
%A Schertler, Cosima
%A Hildebrandt, Martin
%A Neuenhahn, Michael
%A Sadun, Alfredo A.
%A Heilweil, Gad
%A Karanjia, Rustum
%A Tsui, Irena
%A Rebolleda Fernández, Gema
%A Urquijo, Laia Jaumendreu
%A Negrete Muñoz, Francisco J.
%A Fortin, Elizabeth
%A Chwalisz, Bart K.
%A Cestari, Dean
%A Banik, Rudrani
%A Tai, Katy
%A Castillo, Lorena
%A Garcia, Virginia
%A Bandello, Antonio Morilla Francesco
%A Barboni, Piero
%A Cascavilla, Maria Lucia
%A Battista, Marco
%A Calcagno, Francesca
%A Pina, Adelaide
%A Leruez, Stéphanie
%A Forooza, Rod
%T Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G</td><td width="150">
%T gt;A MT-ND4 Mutation
%J Ophthalmology and therapy
%V 12
%N 1
%@ 2193-6528
%C Heidelberg
%I SpringerOpen
%M DZNE-2023-00777
%P 401 - 429
%D 2023
%X 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
%K Gene therapy (Other)
%K LHON (Other)
%K Leber hereditary optic neuropathy (Other)
%K MT-ND4 (Other)
%K Natural history (Other)
%K Visual acuity (Other)
%F PUB:(DE-HGF)16
%9 Journal Article
%$ PMID:36449262
%2 PMC:PMC9834474
%R 10.1007/s40123-022-00611-x
%U https://pub.dzne.de/record/259705