Update on Clinical Trial Endpoints in Gene Therapy Trials for Inherited Retinal Diseases.

Inherited retinal diseases (IRDs) encompass a wide spectrum of rare conditions characterized by diverse phenotypes associated with hundreds of genetic variations, often leading to progressive visual impairment and profound vision loss. Multiple natural history studies and clinical trials exploring gene therapy for various IRDs are ongoing. Outcomes for ophthalmic trials measure visual changes in three main categories-structural, functional, and patient-focused outcomes.

Self-Inflicted Laser-Induced Retinopathy.

This photo essay details a patient with self-inflicted laser-induced retinal injury progressing to full-thickness macular holes in both eyes. A 40-year-old patient presented after a self-inflicted injury by a handheld class 3 blue laser (450 nm) he purchased on the internet. The patient reported shining the laser through a window, which reflected the beam back into his eyes.

Updates on Emerging Interventions for Autosomal Recessive -Associated Stargardt Disease.

Autosomal recessive Stargardt disease (STGD1) is an inherited retinal degenerative disease associated with a mutated ATP-binding cassette, subfamily A, member 4 () gene. STGD1 is the most common form of juvenile macular degeneration with onset in late childhood to early or middle adulthood and causes progressive, irreversible visual impairment and blindness. No effective treatment is currently available.

Surgical Management of Full-Thickness Macular Holes in Macular Telangiectasia Type 2: A Global Multicenter Study.

Purpose: To report on macular hole repair in macular telangiectasia type 2 (MacTel2).

Design: Global, multicenter, retrospective case series.

Participants: Patients undergoing surgery for MacTel2-associated full-thickness macular hole (MTMH).

Current Assistive Devices Usage and Recommendations for a Future Artificial Vision Prosthesis among Patients with Severe Visual Impairment Due to Inherited Retinal Diseases.

Patients with inherited retinal diseases (IRDs) utilize various adaptive techniques and devices designed to assist them with activities of daily living (ADLs). The purpose of this study was to assess the assistive devices used by patients with IRDs, the difficulties they face despite these devices, and their recommendations for a future visual prosthesis. In collaboration with blind patients, an online survey was developed and administered to adults with IRDs and visual acuities of 20/400 to no light perception in the better-seeing eye.

Needle aspiration for management of acute intraoperative fluid misdirection during phacoemulsification.

Purpose: Acute intraoperative fluid misdirection is a serious complication that may occur during phacoemulsification. We provide a detailed description and a video of our preferred technique for prompt management of acute intraoperative fluid misdirection.

Observations: A 79-year-old male developed sudden shallowing of the anterior chamber and marked elevation of intraocular pressure at hydrodissection during phacoemulsification surgery.

Visual Acuity, Retinal Morphology, and Patients' Perceptions after Voretigene Neparovec-rzyl Therapy for RPE65-Associated Retinal Disease.

Objective: To explore the effect of patients' age, baseline visual acuity (VA), and intraoperative foveal detachment on outcomes of subretinal voretigene neparvovec-rzyl (Luxturna) therapy and to assess patients' perceptions of the treatment effect.

Design: Multicenter, retrospective, consecutive case series, and cross-sectional prospective survey.

Participants: All 41 consecutive patients treated with voretigene neparvovec-rzyl after Food and Drug Administration approval at 3 institutions between January 2018 and May 2020.

Surgical Observations From the First 120 Cases of Subretinal Gene Therapy for Inherited Retinal Diseases.

Purpose: To report surgical observations formulated during the first 120 cases of subretinal gene therapy in patients with inherited retinal degenerations (IRDs).

Methods: A two-surgeon team compiled surgical observations and formulated surgical pearls based on the consecutive cases of subretinal viral vector injection in patients enrolled in clinical trials focusing on choroideremia, achromatopsia, and RP GTPase regulator associated retinitis pigmentosa, as well as patients with retinal pigment epithelium-specific-65-kDa (RPE65) associated Leber congenital amaurosis receiving Food and Drug Administration-approved voretigene neparvovec-rzyl therapy.

Results: One hundred twenty subretinal surgeries were performed by a two-surgeon team.

Autologous Retinal Transplantation for Primary and Refractory Macular Holes and Macular Hole Retinal Detachments: The Global Consortium.

Purpose: To report the anatomic and functional outcomes of autologous retinal transplantation (ART).

Design: Multicenter, retrospective, interventional, consecutive case series.

Participants: One hundred thirty eyes of 130 patients undergoing ART for the repair of primary and refractory macular holes (MHs), as well as combined MH-rhegmatogenous retinal detachment (MH-RRD), between January 2017 and December 2019.

Outcomes of Pars Plana Vitrectomy Alone versus Combined Scleral Buckling plus Pars Plana Vitrectomy for Primary Retinal Detachment.

Purpose: We compared the outcomes of primary uncomplicated rhegmatogenous retinal detachment (RRD) repair using pars plana vitrectomy (PPV) alone versus combined scleral buckling plus PPV (SB+PPV).

Design: Retrospective, observational study.

Participants: Patients with primary RRD who underwent PPV or SB+PPV from June 1, 2014, through December 31, 2017.

Rhegmatogenous Retinal Detachment after Intravitreal Injection.

Purpose: To describe the clinical features and outcomes in patients with rhegmatogenous retinal detachment (RRD) after intravitreal injection of pharmacologic agents.

Design: Retrospecitve case series.

Participants: Patients diagnosed with rhegmatogenous retinal detachment within 3 months of receiving an intravitreal injection of a pharmacologic agent for treatment of macular disease.

Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR.

Retinal gene therapy has shown great promise in treating retinitis pigmentosa (RP), a primary photoreceptor degeneration that leads to severe sight loss in young people. In the present study, we report the first-in-human phase 1/2, dose-escalation clinical trial for X-linked RP caused by mutations in the RP GTPase regulator (RPGR) gene in 18 patients over up to 6 months of follow-up (https://clinicaltrials.gov/: NCT03116113).

ADVERSE EVENTS OF THE ARGUS II RETINAL PROSTHESIS: Incidence, Causes, and Best Practices for Managing and Preventing Conjunctival Erosion.

Purpose: To analyze and provide an overview of the incidence, management, and prevention of conjunctival erosion in Argus II clinical trial subjects and postapproval patients.

Methods: This retrospective analysis followed the results of 274 patients treated with the Argus II Retinal Prosthesis System between June 2007 and November 2017, including 30 subjects from the US and European clinical trials, and 244 patients in the postapproval phase. Results were gathered for incidence of a serious adverse event, incidence of conjunctival erosion, occurrence sites, rates of erosion, and erosion timing.

The Influence of Surgical Timing on Clinical Outcomes in Primary Extramacular Retinal Detachment in a Tertiary Referral Center.

Purpose: This article reports the influence of timing on the clinical outcomes in primary extramacular rhegmatogenous retinal detachment (RRD) at a tertiary referral center.

Methods: A retrospective case series was conducted of all patients presenting between January 1, 2014 and December 31, 2016, with primary extramacular RRD. Retinal detachments with grade C proliferative vitreoretinopathy, combined tractional and RRD, eyes with inflammatory disease, and prior retinal surgery were excluded.

Visual and anatomic outcomes of cataract surgery with intraoperative or postoperative complications in a teaching institution.

Objective: To review visual and anatomic outcomes after cataract surgery with complications in a teaching institution.

Methods: Consecutive case series. A chart review was conducted of patients who underwent phacoemulsification with intraoperative or postoperative complications, performed by ophthalmology residents under direct supervision of experienced ophthalmology attending physicians.

Opacification of Akreos Hydrophilic Acrylic Lens After Retinal Detachment Repair with Silicone Oil Tamponade: A Case Report.

We report localized opacification of a sclera-fixated Akreos hydrophilic acrylic intraocular lens after successful repair of rhegmatogenous retinal detachment with silicone oil tamponade in a nondiabetic patient. No intraoperative lens opacification during air-fluid exchange or lens dislocation was encountered. Granular opacities within the optic were noted at 5 months after surgery, and did not relent to scrubbing with a vitrector during oil removal.