No abstract

Background Hereditary transthyretin (ATTRv) amyloidosis is a rare, inherited, progressive disease caused by mutations in the transthyretin (TTR) gene. We aimed to assess the efficacy and safety of long-term treatment with patisiran, an RNA interference therapeutic that inhibits TTR production, in patients with ATTRv amyloidosis with polyneuropathy. MethodsThis multi-country, multi-centre, open-label extension (OLE) trial enrolled patients at 43 sites in 19 countries as of 24 September 2018. Patients were eligible if they had completed the phase 3 APOLLO (randomised, double-blind, placebo-controlled [2:1], 18-month study) or phase 2 OLE (single-arm, 24-month study) parent studies and tolerated the study drug. Eligible patients from APOLLO (APOLLO-patisiran [received patisiran during APOLLO] and APOLLO-placebo [received placebo during APOLLO] groups) and the phase 2 OLE (phase 2 OLE patisiran group) studies enrolled in this Global OLE trial and receive patisiran 0•3 mg/kg by intravenous infusion every 3 weeks for up to 5 years. Efficacy assessments include measures of polyneuropathy (modified Neuropathy Impairment Score +7 [mNIS+7]), quality of life, autonomic symptoms, nutritional status, disability, ambulation status, motor function, and cardiac stress. Patients included in the current efficacy analyses are those who had completed 12-month efficacy assessments as of the data cut-off. Safety analyses included all patients who received ≥1 dose of patisiran up to the data cut-off. The Global OLE is ongoing with no new enrolment, and current findings are based on the 12-month interim analysis. The study is registered with ClinicalTrials.gov, NCT02510261.

The aim of the study presented here was to evaluate retinal and optic nerve head (ONH) perfusion in patients with severe asymptomatic carotid artery stenosis (CAS) compared with healthy controls and to analyze the impact of carotid endarterectomy using optical coherence tomography angiography (OCT-A). 25 eyes of 25 patients with CAS (study group) and 25 eyes of 25 healthy controls (control group) were prospectively included in this study. OCT-A was performed using RTVue XR Avanti (Optovue, Inc, Fremont, California, USA). The flow density data in the superficial and deep retinal OCT-angiogram of the macula and in the radial peripapillary capillary network (RPC) of the ONH were extracted and analyzed. The flow density in the superficial retinal OCT angiogram of the macula and in the ONH were significantly lower in the study group compared with the control group (macula: p = 0.003) (ONH: p = 0.013). The flow density in the ONH improved significantly after carotid endarterectomy (p = 0.004). A reduced flow density was observed in patients with CAS when compared with healthy controls. The flow density also improved after carotid endarterectomy. Quantitative changes in the microvascular density, as measured using OCT-A, could well be useful in the diagnosis of CAS and the evaluation of therapy success.

PurposeTo evaluate the repeatability, the reproducibility and the agreement of foveal avascular zone (FAZ) measurements using three different optical coherence tomography angiography (OCT-A) devices.ProceduresThis prospective study included 24 eyes of 24 healthy volunteers. OCT-A imaging was performed using RTVue XR Avanti, Canon OCT-HS100 and Spectralis OCT-A. Repeated measurements were performed under the same conditions on two separate days, and the area of the FAZ was determined and analyzed using the above devices.ResultsAll three devices showed a high ICC and there was no significant difference between the ICCs (pairwise comparison) of the three devices (Optovue–Canon (p = 0.66); Canon–Heidelberg (p = 0.21); Heidelberg–Optovue (p = 0.37). Agreement analysis of the three devices revealed a significant elevation of FAZ area values with the Heidelberg device and a slight underestimation of the FAZ area with the Canon device. Nevertheless, overall we found a high level of agreement between all of the three devices (ICC ≥ 0.958 (0.905–0.982)).ConclusionsGood reproducibility and repeatability were observed for all three devices. However, the agreement analysis revealed slight, but significant differences, which might limit alternating use of these devices for clinical research and follow-up examinations.

Purpose: To evaluate the correlation between the flow density measured by optical coherence tomography angiography and functional parameters in patients with retinitis pigmentosa. Methods: Twenty eyes of 20 patients with retinitis pigmentosa and 21 eyes of 21 healthy subjects were prospectively included in this study. Optical coherence tomography angiography was performed using RTVue XR Avanti with AngioVue (Optovue Inc). The macula was imaged with a 6 × 6-mm2 scan, whereas for the optic nerve head a 4.5 × 4.5-mm2 scan was taken. Visual acuity, visual field parameters (mean deviation and visual field index), full-field electroretinography, and multifocal electroretinography were tested for correlation with flow density data. Results: The flow density (whole en face) in the superficial/deep retinal OCT angiograms and in the optical coherence tomography angiography of the optic nerve head was significantly lower in the retinitis pigmentosa group when compared with the control group (P < 0.001). The flow density in the superficial retinal OCT angiogram (fovea) correlated significantly with the visual acuity (rSpearman = −0.77, P < 0.001) and the visual field parameters (visual field index: rSpearman = 0.56, P = 0.01; mean deviation: rSpearman = 0.54, P = 0.01). Conclusion: Patients with retinitis pigmentosa show a decreased macular and optic nerve head perfusion compared with healthy subjects. The flow density measured using optical coherence tomography angiography correlated with subjective and objective functional parameters.