Statistical approaches in published ophthalmic clinical science papers: a comparison to statistical practice two decades ago.

The aim of this study is to evaluate the current practice of statistical analysis of eye data in clinical science papers published in () and to determine whether the practice of statistical analysis has improved in the past two decades. All clinical science papers (n=125) published in in January-June 2017 were reviewed for their statistical analysis approaches for analysing primary ocular measure. We compared our findings to the results from a previous paper that reviewed papers in 1995.

Survey of Ophthalmologists Regarding Practice Patterns for Dry Eye and Sjogren Syndrome.

Objective: To survey ophthalmologists about current practice patterns regarding the evaluation of dry eye patients and referrals for a Sjogren syndrome (SS) workup.

Methods: An online survey was sent to ophthalmologists affiliated with the Scheie Eye Institute or Wills Eye Hospital using REDCap in August 2015. Descriptive statistics were used to summarize the data.

Single grading vs double grading with adjudication in the telemedicine approaches to evaluating acute-phase retinopathy of prematurity (e-ROP) study.

Purpose: To evaluate the sensitivity and specificity of single, independent, nonphysician trained reader (TR) gradings in the Telemedicine Approaches to Evaluating Acute-phase Retinopathy of Prematurity (e-ROP) study.

Methods: Secondary analyses of image grading results from 1,235 infants of birth weights <1251 g. Two of three TRs independently graded image sets; discrepancies were adjudicated by the reading center director (an ophthalmologist) to reach final grading.

[Evolution and transition of quality evaluation of Platycodon grandiflorum].

Platycodon grandiflorum, a traditional Chinese medicine commonly used for coughing and eliminating phlegm to relieve asthma, has gained great attention to its quality evaluation in ancient and modern herbal books. This paper investigated the methods of quality evaluation in the ancient herbal books systematically, and the results showed that there were bitter and sweet P. grandiflorum, south and north P.

Is gastroesophageal reflux disease associated with primary acquired nasolacrimal duct obstruction?

Purpose: To determine if patients with primary acquired nasolacrimal duct obstruction (PANDO) have an increased prevalence of gastroesophageal reflux disease (GERD) compared to the general population.

Methods: Cross-sectional case-control study. The Mayo Clinic Reflux Disease Questionnaire (RDQ) is used to help physicians diagnose GERD.

Intereye Agreement of Retinopathy of Prematurity from Image Evaluation in the Telemedicine Approaches to Evaluating of Acute-Phase ROP (e-ROP) Study.

Purpose: To determine the symmetry on retinal image grading of fellow eyes for retinopathy of prematurity (ROP) features (stage, zone and plus disease) and severity, to provide the basis for the within subject comparison for ROP trials.

Design: Secondary analyses of data from the Telemedicine Approaches to Evaluating of Acute-Phase ROP (e-ROP) Study.

Subjects: Infants with birth weight less than 1251g.

Attention and Visual Motor Integration in Young Children with Uncorrected Hyperopia.

Significance: Among 4- and 5-year-old children, deficits in measures of attention, visual-motor integration (VMI) and visual perception (VP) are associated with moderate, uncorrected hyperopia (3 to 6 diopters [D]) accompanied by reduced near visual function (near visual acuity worse than 20/40 or stereoacuity worse than 240 seconds of arc).

Purpose: To compare attention, visual motor, and visual perceptual skills in uncorrected hyperopes and emmetropes attending preschool or kindergarten and evaluate their associations with visual function.

Methods: Participants were 4 and 5 years of age with either hyperopia (≥3 to ≤6 D, astigmatism ≤1.

Evaluation of Approaches to Analyzing Continuous Correlated Eye Data When Sample Size Is Small.

Purpose: To evaluate the performance of commonly used statistical methods for analyzing continuous correlated eye data when sample size is small.

Methods: We simulated correlated continuous data from two designs: (1) two eyes of a subject in two comparison groups; (2) two eyes of a subject in the same comparison group, under various sample size (5-50), inter-eye correlation (0-0.75) and effect size (0-0.

Optical coherence tomography identifies outer retina thinning in frontotemporal degeneration.

Objective: Whereas Alzheimer disease (AD) is associated with inner retina thinning visualized by spectral-domain optical coherence tomography (SD-OCT), we sought to determine if the retina has a distinguishing biomarker for frontotemporal degeneration (FTD).

Methods: Using a cross-sectional design, we examined retinal structure in 38 consecutively enrolled patients with FTD and 44 controls using a standard SD-OCT protocol. Retinal layers were segmented with the Iowa Reference Algorithm.

Detection of Potentially Severe Retinopathy of Prematurity by Remote Image Grading.

Importance: Telemedicine in retinopathy of prematurity (ROP) has the potential for delivering timely care to premature infants at risk for serious ROP.

Objective: To describe the characteristics of eyes at risk for ROP to provide insights into what types of ROP are most easily detected early by image grading.

Design, Setting, And Participants: Secondary analysis of eyes with referral-warranted (RW) ROP (stage 3 ROP, zone I ROP, plus disease) on diagnostic examination from the Telemedicine Approaches to Evaluating Acute-Phase Retinopathy of Prematurity (e-ROP) study was conducted from May 1, 2011, to October 31, 2013, in 1257 premature infants with birth weights less than 1251 g in 13 neonatal units in North America.

Validation of the Children's Hospital of Philadelphia Retinopathy of Prematurity (CHOP ROP) Model.

Importance: The Children's Hospital of Philadelphia Retinopathy of Prematurity (CHOP ROP) model uses birth weight (BW), gestational age at birth (GA), and weight gain rate to predict the risk of severe retinopathy of prematurity (ROP). In a model development study, it predicted all infants requiring treatment, while greatly reducing the number of examinations compared with current screening guidelines.

Objective: To validate the CHOP ROP model in a multicenter cohort that is large enough to obtain a precise estimate of the model's sensitivity for treatment-requiring ROP.

Factors Associated with Occurrence of Radiation-induced Optic Neuropathy at "Safe" Radiation Dosage.

Background: Radiation-induced optic neuropathy (RION) is a rare, and often visually devastating, complication of radiation therapy (RT) near the anterior visual pathways.

Methods: A retrospective case series of patients who developed RION at a tertiary medical center, followed by a case-control study comparing RION cases with matched controls who received RT.

Results: Thirteen patients (18 eyes) with RION were identified.

Visual and Morphologic Outcomes in Eyes with Hard Exudate in the Comparison of Age-Related Macular Degeneration Treatments Trials.

Purpose: To compare baseline characteristics, visual acuity (VA) and morphological outcomes between eyes with baseline hard exudates (HE) and all other eyes among patients with neovascular age-related macular degeneration (NVAMD) treated with anti-vascular endothelial growth factors (anti-VEGF).

Design: Prospective cohort study within the Comparison of Age-Related Macular Degeneration Treatments Trials (CATT).

Participants: Patients with NVAMD.

Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations.

X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene is an early onset and severe cause of blindness. Successful proof-of-concept studies in a canine model have recently shown that development of a corrective gene therapy for RPGR-XLRP may now be an attainable goal. In preparation for a future clinical trial, we have here optimized the therapeutic AAV vector construct by showing that GRK1 (rather than IRBP) is a more efficient promoter for targeting gene expression to both rods and cones in non-human primates.

Cure modeling in real-time prediction: How much does it help?

Various parametric and nonparametric modeling approaches exist for real-time prediction in time-to-event clinical trials. Recently, Chen (2016 BMC Biomedical Research Methodology 16) proposed a prediction method based on parametric cure-mixture modeling, intending to cover those situations where it appears that a non-negligible fraction of subjects is cured. In this article we apply a Weibull cure-mixture model to create predictions, demonstrating the approach in RTOG 0129, a randomized trial in head-and-neck cancer.

Tutorial on Biostatistics: Statistical Analysis for Correlated Binary Eye Data.

Purpose: To describe and demonstrate methods for analyzing correlated binary eye data.

Methods: We describe non-model based (McNemar's test, Cochran-Mantel-Haenszel test) and model-based methods (generalized linear mixed effects model, marginal model) for analyses involving both eyes. These methods were applied to: (1) CAPT (Complications of Age-related Macular Degeneration Prevention Trial) where one eye was treated and the other observed (paired design); (2) ETROP (Early Treatment for Retinopathy of Prematurity) where bilaterally affected infants had one eye treated conventionally and the other treated early and unilaterally affected infants had treatment assigned randomly; and (3) AREDS (Age-Related Eye Disease Study) where treatment was systemic and outcome was eye-specific (both eyes in the same treatment group).

Comparison of cycloplegic refraction between Grand Seiko autorefractor and Retinomax autorefractor in the Vision in Preschoolers-Hyperopia in Preschoolers (VIP-HIP) Study.

Purpose: To evaluate the agreement of cycloplegic refractive error measures between the Grand Seiko and Retinomax autorefractors in 4- and 5-year-old children.

Methods: Cycloplegic refractive error of children was measured using the Grand Seiko and Retinomax during a comprehensive eye examination. Accommodative error was measured using the Grand Seiko.

Inflammatory Mediators in Xanthelasma Palpebrarum: Histopathologic and Immunohistochemical Study.

Purpose: To evaluate the expression of inflammatory mediators in xanthelasma palpebrarum.

Methods: In this retrospective histopathologic case-control study, xanthelasma specimens obtained from the private practice and pathology archives of 1 author (R.Z.

Evaluation of Optical Coherence Tomography to Detect Elevated Intracranial Pressure in Children.

Importance: Detecting elevated intracranial pressure in children with subacute conditions, such as craniosynostosis or tumor, may enable timely intervention and prevent neurocognitive impairment, but conventional techniques are invasive and often equivocal. Elevated intracranial pressure leads to structural changes in the peripapillary retina. Spectral-domain (SD) optical coherence tomography (OCT) can noninvasively quantify retinal layers to a micron-level resolution.

The Role of the Human Visual Cortex in Assessment of the Long-Term Durability of Retinal Gene Therapy in Follow-on RPE65 Clinical Trial Patients.

Purpose: Gene therapy (GT) has offered immense hope to individuals who are visually impaired because of RPE65 mutations. Although GT has shown great success in clinical trials enrolling these individuals, evidence for stability and durability of this treatment over time is still unknown. Herein we explored the value of functional magnetic resonance imaging (fMRI) as an objective measure to assess independently the longevity of retinal GT.

Tutorial on Biostatistics: Linear Regression Analysis of Continuous Correlated Eye Data.

Purpose: To describe and demonstrate appropriate linear regression methods for analyzing correlated continuous eye data.

Methods: We describe several approaches to regression analysis involving both eyes, including mixed effects and marginal models under various covariance structures to account for inter-eye correlation. We demonstrate, with SAS statistical software, applications in a study comparing baseline refractive error between one eye with choroidal neovascularization (CNV) and the unaffected fellow eye, and in a study determining factors associated with visual field in the elderly.

Incidence and Growth of Geographic Atrophy during 5 Years of Comparison of Age-Related Macular Degeneration Treatments Trials.

Purpose: To estimate the incidence, size, and growth rate of geographic atrophy (GA) during 5 years of follow-up among participants in the Comparison of Age-Related Macular Degeneration Treatments Trials (CATT).

Design: Cohort within a clinical trial.

Participants: Participants included in CATT.