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000169155 037__ $$aDZNE-2023-00034
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000169155 1001_ $$aVignal-ClermoḤ, Catherine$$b0
000169155 245__ $$aSafety of lenadogene nolparvovec gene therapy over 5 years in 189 patients with Leber hereditary optic neuropathy.
000169155 260__ $$aNew York, NY$$bElsevier Science$$c2023
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000169155 520__ $$a To evaluate the safety profile of lenadogene nolparvovec (Lumevoq) in patients with Leber hereditary optic neuropathy.Pooled analysis of safety data from 5 clinical studies.A total of 189 patients received single unilateral or bilateral intravitreal injections of a recombinant adeno-associated virus 2 (rAAV2/2) vector encoding the human wild-type ND4 gene. Adverse events (AEs) were collected throughout the studies, up to 5 years. Intraocular inflammation and increased intraocular pressure (IOP) were ocular AEs of special interest. Other assessments included ocular examinations, vector bio-dissemination, and systemic immune responses against rAAV2/2.Almost all patients (95.2%) received 9 × 1010 viral genomes and 87.8% had at least 2 years of follow-up. Most patients (75.1%) experienced at least one systemic AE, but systemic treatment-related AEs occurred in 3 patients; none were serious. Intraocular inflammation was reported in 75.6% of lenadogene nolparvovec-treated eyes. Almost all intraocular inflammations occurred in the anterior chamber (58.8%) or in the vitreous (40.3%), and were of mild (90.3%) or moderate (8.8%) intensity; most resolved with topical corticosteroids alone. All IOP increases were mild to moderate in intensity. No AE led to study discontinuation. Bio-dissemination of lenadogene nolparvovec and systemic immune response were limited. The safety profile was comparable for patients treated bilaterally and unilaterally.Lenadogene nolparvovec had a good overall safety profile with excellent systemic tolerability, consistent with limited bio-dissemination. The product was well tolerated, with mostly mild ocular side effects responsive to conventional ophthalmologic treatments.
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000169155 650_7 $$2Other$$aLeber hereditary optic neuropathy
000169155 650_7 $$2Other$$aintravitreal gene therapy
000169155 650_7 $$2Other$$asafety
000169155 650_2 $$2MeSH$$aDependovirus
000169155 650_2 $$2MeSH$$aHumans
000169155 650_2 $$2MeSH$$aOptic Atrophy, Hereditary, Leber: drug therapy
000169155 650_2 $$2MeSH$$aOptic Atrophy, Hereditary, Leber: genetics
000169155 650_2 $$2MeSH$$aGenetic Vectors
000169155 650_2 $$2MeSH$$aParvovirinae: genetics
000169155 650_2 $$2MeSH$$aGenetic Therapy
000169155 650_2 $$2MeSH$$aInflammation: etiology
000169155 7001_ $$aYu-Wai-Man, Patrick$$b1
000169155 7001_ $$aNewman, Nancy J$$b2
000169155 7001_ $$aCarelli, Valerio$$b3
000169155 7001_ $$aMoster, Mark L$$b4
000169155 7001_ $$aBiousse, Valerie$$b5
000169155 7001_ $$aSubramanian, Prem S$$b6
000169155 7001_ $$0P:(DE-HGF)0$$aWang, An-Guor$$b7
000169155 7001_ $$aDonahue, Sean P$$b8
000169155 7001_ $$aLeroy, Bart P$$b9
000169155 7001_ $$aSadun, Alfredo A$$b10
000169155 7001_ $$0P:(DE-2719)2810704$$aKlopstock, Thomas$$b11$$udzne
000169155 7001_ $$aSergott, Robert C$$b12
000169155 7001_ $$aFernández, Gema Rebolleda$$b13
000169155 7001_ $$aChwalisz, Bart K$$b14
000169155 7001_ $$aBanik, Rudrani$$b15
000169155 7001_ $$aTaiel, Magali$$b16
000169155 7001_ $$aRoux, Michel$$b17
000169155 7001_ $$aSahel, José-Alain$$b18
000169155 7001_ $$aGroup, LHON Study$$b19$$eCollaboration Author
000169155 773__ $$0PERI:(DE-600)2019600-3$$a10.1016/j.ajo.2022.11.026$$gp. S0002939422004640$$p108 - 125$$tAmerican journal of ophthalmology$$v249$$x0002-9394$$y2023
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