Dataset Information

The Patient Experience: Symptoms and Impact of Dry Age-Related Macular Degeneration.

ABSTRACT:

Introduction

No published literature systematically explores the dry age-related macular degeneration (AMD) patient experience. To inform the development of patient-reported outcome measures (PROMs), the important and relevant signs, symptoms, and impacts for patients with dry AMD were identified.

Methods

A holistic approach was used to capture, define, and organize the signs, symptoms, and impacts that are important to patients with dry AMD. Qualitative evidence was identified through a targeted literature review and clinician (N?=?5) and patient (N?=?20) interviews. The targeted review was expanded to include patients with AMD, as few studies specific to dry AMD were identified. The qualitative evidence was incorporated into a conceptual model that included the signs, symptoms, and impacts of dry AMD affecting the patient experience.

Results

Twenty-nine articles (dry AMD, N?=?5; general AMD, N?=?24) exploring health-related quality-of-life evidence in patients with AMD were identified. Concepts identified and included in the preliminary, literature-based model included signs and symptoms related to general vision loss and general impacts (e.g., dependency on others, poor spatial perception/mobility, difficulty reading, emotional affects). No concepts unique to dry AMD were identified. Interviewed clinicians refined the literature-based model. Across all visual acuity severities, ??80% of patients reported difficulty driving, reading, and completing activities of daily living, along with frustration and dependency on others; all patients reported blurred vision. The final model included 35 signs, symptoms, and impacts, with 19 considered salient.

Conclusions

To better understand the patient experience, we captured, defined, and organized signs, symptoms, and impacts into a dry AMD conceptual model. This model can aid in the development of PROMs reflecting the experience of patients with dry AMD.

SUBMITTER: Schultz NM

PROVIDER: S-EPMC7886930 | biostudies-literature | 2021 Mar

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

The Patient Experience: Symptoms and Impact of Dry Age-Related Macular Degeneration.

Schultz Neil M NM Braunack-Mayer Lydia L Schwartz Jason J Gaspar Luis L

Ophthalmology and therapy 20210129 1

<h4>Introduction</h4>No published literature systematically explores the dry age-related macular degeneration (AMD) patient experience. To inform the development of patient-reported outcome measures (PROMs), the important and relevant signs, symptoms, and impacts for patients with dry AMD were identified.<h4>Methods</h4>A holistic approach was used to capture, define, and organize the signs, symptoms, and impacts that are important to patients with dry AMD. Qualitative evidence was identified th ...[more]

PMID: 33512689

Similar Datasets

Project description:To describe baseline night vision symptoms and their association with >/=3-lines loss in visual acuity (VA), choroidal neovascularization (CNV), and geographic atrophy (GA).Cohort study within a multicenter randomized clinical trial.A total of 1052 participants with >/=10 large (>125 mu) drusen and VA >/=20/40 in each eye.At baseline, participants self-administered a 10-item Night Vision Questionnaire (NVQ-10). VA testing was performed at baseline, 6 months, and annually. One eye of each participant was randomly assigned to laser treatment, and the contralateral eye was assigned to observation. During follow-up, trained readers identified CNV on the basis of fluorescein angiograms and end point GA, defined as >1 disc area of new GA, based on color photographs. Evaluation was performed by repeated-measures logistic regression for NVQ-10 score as a risk factor for >/=3-lines loss in VA and by survival analysis for CNV and GA, with and without adjustment for participant and ocular characteristics. Evaluations were based on observed eyes and treated eyes, considered separately and combined.A >/=3-lines loss in VA, development of CNV and end point GA.At baseline, NVQ-10 scores ranged from 3 to 100 with a mean of 70 (100 corresponds to no night vision symptoms). Compared with participants with the best night vision (fourth quartile of scores), participants with the worst night vision (first quartile of scores) were at increased risk of >/=3-lines loss in VA in both observed and treated eyes; odds ratios (95% confidence interval) were 2.85 (1.85-4.39) and 2.00 (1.27-3.14), respectively. The relative risk for the first quartile versus the fourth quartile for development of GA was 4.18 (1.80-9.68) in observed eyes and 2.59 (1.13-5.95) in treated eyes. The relative risk for CNV incidence was 1.99 (1.12-3.54) in observed eyes and 1.33 (0.81-2.19) in treated eyes. These relationships were maintained after adjustment for baseline participant and ocular characteristics.Participants who perceived the most problems in their night vision at baseline had an increased risk of >/=3-lines loss in VA, CNV, and GA. These associations are independent of established risk factors.

Project description:Age-related macular degeneration (AMD) is the most prevalent form of irreversible blindness worldwide in the elderly population. The pathology of dry AMD consists of macular degeneration of photoreceptors and the RPE, lipofuscin (A2E) accumulation, and drusen formation. Mice have been widely used for generating models that simulate human AMD features for investigating the pathogenesis, treatment and prevention of the disease. Although the mouse has no macula, focal atrophy of photoreceptors and RPE, lipofuscin accumulation, and increased A2E can develop in aged mouse eyes. However, drusen are rarely seen in mice because of their simpler Bruch's membrane and different process of lipofuscin extrusion compared with humans. Thus, analyzing basal deposits at the ultrastructural level and understanding the ultrastructural pathologic differences between various mouse AMD models are critical to comprehending the significance of research findings and response to possible therapeutic options for dry AMD. Based on the multifactorial pathogenesis of AMD, murine dry AMD models can be classified into three groups. First, genetically engineered mice that target genes related to juvenile macular dystrophies are the most common models, and they include abcr(-/-) (Stargardt disease), transgenic ELOVL4 (Stargardt-3 dominant inheritary disease), Efemp1(R345W/R345W) (Doyne honeycomb retinal dystrophy), and Timp3(S156C/S156C) (Sorsby fundus dystrophy) mice. Other murine models target genes relevant to AMD, including inflammatory genes such as Cfh(-/-), Ccl2(-/-), Ccr2(-/-), Cx3cr1(-/-), and Ccl2(-/-)/cx3cr1(-/-), oxidative stress associated genes such as Sod1(-/-) and Sod2 knockdown, metabolic pathway genes such as neprilysin(-/-) (amyloid beta), transgenic mcd/mcd (cathepsin D), Cp(-/-)/Heph(-/Y) (ferroxidase ceruloplasmin/hepaestin, iron metabolism), and transgenic ApoE4 on high fat and high cholesterol diet (lipid metabolism). Second, mice have also been immunologically manipulated by immunization with carboxyethylpyrrole (CEP), an oxidative fragment of DHA found in drusen, and found to present with dry AMD features. Third, natural mouse strains such as arrd2/arrd2 (Mdm gene mutation) and the senescence accelerated mice (SAM) spontaneously develop features of dry AMD like photoreceptor atrophy and thickening of Bruch's membrane. All the aforementioned models develop retinal lesions with various features that simulate dry AMD lesions: focal photoreceptor degeneration, abnormal RPE with increased lipofuscin, basal infolding, decreased melanosomes and degeneration. However, Bruch's membrane changes are less common. Most mice develop retinal lesions at an older age (6-24 months, depending on the models), while the Ccl2(-/-)/cx3cr1(-/-) mice develop lesions by 4-6 weeks. Although murine models present various degrees of retinal and/or RPE degeneration, classical drusen is extremely rare. Using electron microscopy, small drusenoid deposits are found between RPE and Bruch's membrane in a few models including Efemp1(R345W/R345W), Ccl2(-/-)/cx3cr1(-/-), neprilysin(-/-), transgenic mcd/mcd, and ApoE4 transgenic mice on a high fat diet. High A2E levels are measured in the retinas of abcr(-/-), transgenic ELOVL4, and Ccl2(-/-)/cx3cr1(-/-) mice. In summary, murine models provide useful tools for studying AMD pathogenesis and evaluating novel therapies for this disease. This review compares the major dry AMD murine models and discusses retinal pathology at the ultrastructural level.

Dataset Information

The Patient Experience: Symptoms and Impact of Dry Age-Related Macular Degeneration.

Introduction

Methods

Results

Conclusions

Publications

The Patient Experience: Symptoms and Impact of Dry Age-Related Macular Degeneration.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets