Abstract:
PURPOSE:To assess the safety of rAAV.sFlt-1 subretinal injection in neovascular age-related macular degeneration (wet AMD) over 36 months. DESIGN:Phase 1 dose escalation trial. METHODS:Eight subjects with advanced, treatment-experienced wet AMD were randomly assigned (3:1) to treatment and non-gene therapy control groups. Eligible subjects were ≥65 years, had wet AMD, and had best-corrected visual acuity (BCVA) 10/200 to 20/80 in the study eye and 20/200 or better in the other eye. Three of the treatment group subjects received low-dose (1 × 1010 vector genomes) and 3 high-dose (1 × 1011 vector genomes) rAAV.sFLT-1 via subretinal injection. Study monitoring was monthly to the primary endpoint at month 12 and then protocol-driven follow-up study visits were conducted at months 18 and 36. All subjects received intravitreal ranibizumab at baseline and at week 4, and retreatment injections at subsequent visits based on prespecified criteria for active wet AMD. The primary endpoint was ocular and systemic safety, but exploratory data including BCVA, retinal center point thickness, and the number of ranibizumab retreatments at and between study visits were also analyzed. RESULTS:Six of the 8 subjects completed the 36-month study. Subretinal injection with pars plana vitrectomy was well tolerated in this cohort. No ocular or systemic safety signals were observed during the long-term follow-up period. Exploratory data analysis suggests stability of wet AMD over the 36-month period. CONCLUSIONS:Subretinal delivery of rAAV.sFLT-1 was well tolerated and demonstrated a favourable safety profile through month 36. Thus, rAAV.sFLT-1 could be safely considered for future evaluation in the treatment of wet AMD.
journal_name
Am J Ophthalmoljournal_title
American journal of ophthalmologyauthors
Constable IJ,Lai CM,Magno AL,French MA,Barone SB,Schwartz SD,Blumenkranz MS,Degli-Esposti MA,Rakoczy EPdoi
10.1016/j.ajo.2017.02.018subject
Has Abstractpub_date
2017-05-01 00:00:00pages
150-158eissn
0002-9394issn
1879-1891pii
S0002-9394(17)30085-5journal_volume
177pub_type
杂志文章,随机对照试验abstract:PURPOSE:To investigate the characteristics, mergers, and risk factors of different types of myopic maculopathy (MM) in a highly myopic population. DESIGN:Population-based, cross-sectional study. METHODS:A total of 1086 eyes (762 patients) were enrolled. Each participant underwent detailed ocular examinations. Combini...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/j.ajo.2019.07.010
更新日期:2019-12-01 00:00:00
abstract:PURPOSE:To demonstrate techniques of iris reconstruction (pupilloplasty) at the time of penetrating keratoplasty (PKP) for a variety of pupil abnormalities, and to evaluate the effect of pupilloplasty on graft survival and visual outcome. DESIGN:Case-control study. METHODS:Thirty-five cases (combined PKP and pupillop...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/j.ajo.2007.10.006
更新日期:2008-02-01 00:00:00
abstract:PURPOSE:To determine whether alterations of p53, a tumor suppressor gene, were present in uveal melanoma, and to characterize further the nature of those changes. METHODS:Immunohistochemical analysis with a monoclonal antibody was used to determine whether alterations of p53 were present in 35 enucleated archival uvea...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/s0002-9394(14)75431-5
更新日期:1996-05-01 00:00:00
abstract:PURPOSE:To present a case of cancer-associated retinopathy (CAR) in a patient with small cell carcinoma of the lung and antiretinal antibodies who experienced visual symptoms before diagnosis of cancer. DESIGN:Case report. METHODS:A 61-year-old man with a sudden loss of vision and photophobia was referred to the opht...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/j.ajo.2004.10.044
更新日期:2005-04-01 00:00:00
abstract:PURPOSE:To report a young girl with spontaneous separation of an idiopathic epiretinal membrane and notable visual recovery. METHOD:Case report. RESULT:A 12-year-old girl had spontaneous improvement in visual acuity of the left eye from 20/100 to 20/40 attributable to spontaneous peeling of an idiopathic epiretinal m...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/s0002-9394(99)00021-5
更新日期:1999-06-01 00:00:00
abstract:PURPOSE:To study the prevalence and risk factors of intraocular pressure (IOP) elevation and refractory IOP elevation after a single 4 mg intravitreal triamcinolone acetonide (IVTA) injection in a Chinese population. DESIGN:Retrospective, observational case series. METHODS:Retrospective chart review of all the patien...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/j.ajo.2008.05.034
更新日期:2008-10-01 00:00:00
abstract::Patients with chronic forms of blepharochalasis often develop eyelid deformities characterized by blepharoptosis and prolapse of the orbital fat and lacrimal gland. Some individuals have an acquired form of blepharophimosis, secondary to the dehiscence of the canthal tendons. In this late stage of the condition, the t...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/0002-9394(85)90009-1
更新日期:1985-04-15 00:00:00
abstract:PURPOSE:To analyze the long-term growth patterns of type 1 neovascularization (NV) in eyes with age-related macular degeneration (AMD) receiving anti-vascular endothelial growth factor (VEGF) therapy. DESIGN:Retrospective cohort study. METHODS:Patients were enrolled from 2 eye centers and underwent optical coherence ...
journal_title:American journal of ophthalmology
pub_type: 杂志文章,多中心研究
doi:10.1016/j.ajo.2017.12.005
更新日期:2018-03-01 00:00:00
abstract:PURPOSE:To determine the incidence and possible causes of a late recurrence of a retinal detachment (RD) in eyes with stages 4B and 5 retinopathy of prematurity (ROP), in which the retina was once reattached by lensectomy and vitrectomy. DESIGN:Retrospective, comparative case series. METHODS:The medical records of 12...
journal_title:American journal of ophthalmology
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更新日期:2009-04-01 00:00:00
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journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/j.ajo.2008.08.031
更新日期:2009-02-01 00:00:00
abstract:PURPOSE:To describe the incidence rates of visual loss and ocular complications in patients with retinal vasculitis (RV). DESIGN:Retrospective cohort study. METHODS:Clinical data were collected for 96 patients (175 eyes) diagnosed with RV from 2003 to 2013. Main outcome measures included rates of visual loss and ocul...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/j.ajo.2017.05.007
更新日期:2017-07-01 00:00:00
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journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/j.ajo.2010.05.035
更新日期:2010-11-01 00:00:00
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journal_title:American journal of ophthalmology
pub_type: 杂志文章,多中心研究
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更新日期:2020-10-01 00:00:00
abstract:PURPOSE:To evaluate the involvement of the anterior ocular segment in Harada disease. METHODS:In a prospective study, ultrasound biomicroscopy was performed in 18 consecutive eyes of nine patients with Harada disease in its acute phase before and after onset of systemic corticosteroid therapy. Examination was repeated...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/s0002-9394(98)00033-6
更新日期:1998-05-01 00:00:00
abstract:PURPOSE:To report the features of idiopathic intracranial hypertension in prepubertal children, with emphasis on presentation, treatment, and outcome. METHODS:We retrospectively reviewed the charts of all patients 11 years and younger diagnosed with idiopathic intracranial hypertension at two university-affiliated med...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/s0002-9394(98)00386-9
更新日期:1999-02-01 00:00:00
abstract:OBJECTIVE:To report clinicopathologic features, radiologic findings, and treatment outcomes of isolated, orbital aspergillosis. DESIGN:Multicenter, retrospective case series. METHODS:setting: Multicenter. PARTICIPANTS:There were 8 lesions in 8 eyes of 8 patients with isolated, orbital aspergillosis. PROCEDURE:Revie...
journal_title:American journal of ophthalmology
pub_type: 杂志文章,多中心研究
doi:10.1016/j.ajo.2016.03.007
更新日期:2016-05-01 00:00:00
abstract::We reviewed the histories of three patients with sebaceous carcinoma (two of the eyelid and one of the caruncle) who had had their tumors excised by surgeons experienced in the Mohs' technique. In each patient the tumor was considered totally excised. In one patient, residual intraepithelial pagetoid sebaceous carcino...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/s0002-9394(14)74400-9
更新日期:1987-06-15 00:00:00
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journal_title:American journal of ophthalmology
pub_type: 杂志文章
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abstract:PURPOSE:To apply in vivo corneal confocal microscopy (IVCM) to study the pathogenesis of keratitis (keratoendotheliitis) fugax hereditaria, an autosomal dominant cryopyrin-associated periodic keratitis, associated with the c.61G>C pathogenic variant in the NLRP3 gene, in its acute and chronic phase, and to report histo...
journal_title:American journal of ophthalmology
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更新日期:2020-05-01 00:00:00
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journal_title:American journal of ophthalmology
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更新日期:1982-09-01 00:00:00
abstract:PURPOSE:To report a case of bilateral optic disk edema in a patient taking oprelvekin for radioimmunotherapy-induced thrombocytopenia. DESIGN:Observational case report. METHODS:A 38-year-old man with a history of relapsed non-Hodgkins follicular lymphoma complained of bilateral loss of vision following oprelvekin the...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/j.ajo.2004.07.055
更新日期:2005-02-01 00:00:00
abstract:PURPOSE:To assess trends in prevalence of diagnosed ocular disease and use of eye care services in the Veterans Affairs (VA) health care system. DESIGN:Prevalence study. METHODS:We performed a retrospective study of all eligible veterans in the VA Capitol Health Care Network from 2007 to 2011. The VA database was use...
journal_title:American journal of ophthalmology
pub_type: 杂志文章,多中心研究
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更新日期:2017-01-01 00:00:00
abstract:PURPOSE:To report a patient with unilateral vision loss and neovascular glaucoma after attempted superior ophthalmic vein embolization in the treatment of a carotid-cavernous sinus fistula. METHODS:A 69-year-old man with a history of a left dural carotid-cavernous sinus fistula underwent attempted treatment with super...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
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更新日期:1997-12-01 00:00:00
abstract:PURPOSE:To characterize the molecular defect in the TGFBI gene in a Chinese family affected with an atypical lattice corneal dystrophy. DESIGN:Case report and experimental study. METHODS:Molecular genetic analysis was performed on the DNA extracted from peripheral leucocytes from a Chinese family with atypical lattic...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/j.ajo.2007.04.015
更新日期:2007-09-01 00:00:00
abstract::The dimensions of the Westheimer function are stable across populations and in time and vary with the eccentricity of fixation. The function is thus useful to assess whether an individual is fixing centrally and if not to estimate the degree of eccentricity of fixation. We identified both summation and inhibition-like...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/0002-9394(77)90554-2
更新日期:1977-04-01 00:00:00
abstract:PURPOSE:To compare the effects of extracapsular cataract extraction with posterior chamber intraocular lens (ECCE/PC-IOL) vs intracapsular cataract extraction with aphakic glasses (ICCE-AG) on everyday visual functioning and quality of life. METHODS:In a nonmasked randomized controlled clinical trial, 3,400 bilateral ...
journal_title:American journal of ophthalmology
pub_type: 临床试验,杂志文章,随机对照试验
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更新日期:1998-01-01 00:00:00
abstract::Seventeen patients (ten women and seven men, 23 to 72 years old) with stromal herpetic disease were treated with topical and oral acyclovir for 14 days. Of the 12 patients with disciform edema, five showed minimal improvement, four showed no change, and three showed worsening of their disease. Of the five patients wit...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
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更新日期:1984-11-01 00:00:00
abstract::A study of ten patients (six girls and four boys ranging in age from 17 months to 9 1/2 years) with congenital third nerve palsies showed that not all cases are benign without other signs of focal neurologic damage. Six of the ten children showed some degree of pupillary involvement, five had signs of oculomotor synki...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
doi:10.1016/0002-9394(84)90629-9
更新日期:1984-03-01 00:00:00
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journal_title:American journal of ophthalmology
pub_type: 杂志文章,多中心研究
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更新日期:2004-04-01 00:00:00
abstract::A 1:2000 solution of a radiomimetic alkylating agent-triethylene thiophosphoramide (thio-tepa, Lederle Laboratories) with a proven inhibitory action on proliferating vascular endothelium, was used topically as an adjunct to pterygium surgery in an attempt to decrease the incidence of recurrence. A bare-sclera techniqu...
journal_title:American journal of ophthalmology
pub_type: 杂志文章
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更新日期:1966-05-01 00:00:00