The State of Patient-Reported Outcome Measures for Pediatric Patients with Inherited Retinal Disease.

Patient-reported outcome measures (PROMs) are questionnaires that assess health outcomes meaningful to the patient. PROMs have multiple applications, such as supporting clinicians' decision-making for patient care, understanding the impact of disease on patient functioning, and evaluating the efficacy of therapeutics. Though PROMs were developed for various eye conditions, no PROM was tailored to pediatric patients with inherited retinal disease (IRD). Hence, a literature search was conducted using MEDLINE and Embase to identify PROMs potentially relevant to this patient population. This review evaluated selected pediatric PROMs against the US Food and Drug Administration (FDA) guidelines and found restricted use in the context of IRD. As there is a need for PROMs tailored to pediatric patients with IRD, we provide a perspective on applying the International Society for Pharmacoeconomics and Outcomes Research and FDA standards on the development of PROMs specific to IRD.

Inherited retinal diseases refer to a group of genetic conditions that affect the eye's light-sensing cells and lead to vision loss. When a patient undergoes an eye assessment, the measures used are technical (e.g., visual acuity, visual field) and do not routinely address the patient's experience. It is increasingly evident that the technical tools used do not really reflect how patients' vision affects their daily lives. Questionnaires designed to assess how a condition impacts a daily activity are referred to as patient-reported outcome measures. The perspective of the impact of a condition on daily activities differs between adults and children. These tools are being created to evaluate health outcomes important to the patient on the basis of their condition and age. This is especially important when determining the value of therapies from the patient perspective. To date, no such questionnaire has been designed for pediatric patients with inherited retinal disease, an important cause of blindness. We explored the literature to evaluate existing pediatric vision tools and found that those could not be used to fill this gap. Given that we found a need to develop questionnaires tailored to pediatric patients with IRD, we also provide insight into how such a tool can be created for this population.

Short-term effects of intravitreal bevacizumab in contrast sensitivity of patients with diabetic macular edema and optimizing glycemic control.

AIMS: To analyze contrast sensitivity of intravitreal bevacizumab injections with optimizing glycemic control versus optimizing glycemic control (in combination with sham injections) in eyes with Diabetic Macular Edema (DME). DESIGN: Prospective, interventional, masked, randomized controlled trial. METHODS: Forty-one eyes of 34 patients with type 2 diabetes mellitus and DME with glycated hemoglobin (HbA1c) < 11% received either intravitreal bevacizumab injection (Group 1) or sham injection (Group 2) at 0 and 6 weeks along with optimizing glycemic control. Mean change in best-corrected visual acuity (BCVA), contrast sensitivity (CS), optical coherence tomography (OCT)-measured by central macular thickness (CMT) were compared and correlated at baseline, 2, 6 and 12 weeks. RESULTS: The study showed a mean CS improved in group 1 from 1.14 ±â€¯0.36 logCS to 1.32 ±â€¯0.24 logCS and also in group 2 from 1.11 ±â€¯0.29 logCS to 1.18 ±â€¯0.29 logCS at 12 weeks (P = 0.12). CS and CMT promptly decreased in group 1 compared to group 2 at 2 weeks (ΔCS = 0.15 ±â€¯0.25 vs. 0.03 ±â€¯0.15 logCS; P = 0.04; ΔCMT = 116 ±â€¯115 vs. 17 ±â€¯71 µm; P = 0.01). There was a mean reduction of approximately 0.5% in HbA1c levels in both groups at 12 weeks (P = 0.002). CONCLUSION: The use of bevacizumab in combination with optimizing glycemic control results in earlier improvement of contrast sensitivity in type 2 diabetes patients with DME. However, the optimizing glycemic control itself has shown also to be effective at 12 weeks. ClinicalTrials.gov Identifier: NCT02308644.

Subconjunctival 0.1% epinephrine versus placebo in maintenance of mydriasis during vitrectomy: a randomized controlled trial.

BACKGROUND: Pupil dilation and mydriasis maintenance throughout vitreoretinal surgeries are important to allow satisfactory fundus visualization and reduce risk of complications. The purpose of this study is to evaluate the role of subconjunctival epinephrine 0.1% injection in mydriasis maintenance during vitrectomy. METHODS: Ninety-nine consecutive patients undergoing vitrectomy were enrolled. All subjects were preoperatively dilated with tropicamide 1%. Each patient was randomly allocated either in the epinephrine or placebo group. In epinephrine group, patients were submitted to a 0.2 cc subconjunctival injection of a 0.1% epinephrine solution just before first incisions. In placebo group, the same procedure was performed with 0.2 cc of saline 0.9%. Horizontal pupil diameter was measured with calipers before and in the end of the procedure. RESULTS: Patients in the epinephrine group showed a significantly larger mean pupil diameter in the end of the surgery compared to placebo. There was a significant increase of mean pupil diameter from the beginning to the end of the surgery in such patients. Blood pressure was significantly higher in the epinephrine group than in placebo group. No other adverse effects were noted. CONCLUSION: Subconjunctival epinephrine is effective for maintaining and increasing pupil size during vitrectomy, compared to placebo. Caution should be taken regarding intraoperative blood pressure levels. TRIAL REGISTRATION: RBR; RBR-3qzhvg; Registered 8 May 2018-Retrospectively registered, http://www.ensaiosclinicos.gov.br/rg/RBR-3qzhvg/.