Visual Function Decline Resulting from Geographic Atrophy: Results from the Chroma and Spectri Phase 3 Trials.

PURPOSE: To assess visual function outcomes to 48 weeks in patients with bilateral geographic atrophy (GA) secondary to age-related macular degeneration included in 2 interventional clinical trials: relationship to baseline lesion size, outcomes by baseline lesion characteristic subgroups, and correlation of visual function outcomes with GA area. DESIGN: The Chroma and Spectri studies (ClinicalTrials.gov identifiers, NCT02247479 and NCT02247531, respectively) were identically designed phase 3, double-masked, multicenter, randomized, sham injection-controlled clinical trials that evaluated intravitreal lampalizumab in GA. PARTICIPANTS: Eligible patients were 50 years of age or older with well-demarcated bilateral GA (lesion size, 1-7 disc areas) without evidence of or previous treatment for choroidal neovascularization in either eye and best-corrected visual acuity (BCVA) letter score of 49 letters or more (≥1 GA lesion within 250 µm of foveal center if BCVA ≥79 letters). METHODS: Patients (pooled n = 1881) were randomized 2:1:2:1 to lampalizumab every 4 weeks, sham every 4 weeks, lampalizumab every 6 weeks, or sham every 6 weeks. Sham arms were pooled for analysis. MAIN OUTCOME MEASURES: Functional end points included change in BCVA from baseline to week 48, low-luminance visual acuity, mesopic microperimetry (number of absolute scotomatous points, mean macular sensitivity), binocular and monocular maximum reading speed, and 2 validated patient-reported outcome measures: Functional Reading Independence Index and 25-item National Eye Institute Visual Function Questionnaire. RESULTS: Enlargement of GA area, approximately 2 mm2/year on average across all treatment groups in each study, was accompanied by overall deterioration in all functional end points. No statistically significant differences were found between lampalizumab or sham arms for changes from baseline in functional assessment scores. Of visual function tests, only microperimetry outcomes were correlated moderately with GA lesion area when assessed cross-sectionally at baseline and week 48. CONCLUSIONS: Chroma and Spectri provide a unique data set of functional end points in GA that are relevant for future clinical trials. Patients with bilateral GA experienced a consistent decline in visual function over 48 weeks, but measures of visual function were not correlated strongly with GA lesion area. It is not possible to predict visual function outcomes from GA lesion size.

Burden of Illness in Geographic Atrophy: A Study of Vision-Related Quality of Life and Health Care Resource Use.

PURPOSE: To gain comprehensive information on the burden of illness due to geographic atrophy (GA). METHODS: This cross-sectional study with a retrospective chart review involved patients aged ≥70 years with physician-confirmed bilateral symptomatic GA due to age-related macular degeneration (GA group), as well as patients of similar age with no ophthalmic condition that in the opinion of the investigator affected visual function (non-GA group). Data relating to patients' current disease status and sociodemographics were self-reported on patient questionnaires at study entry and extracted from patient charts. Historical data on health care resource utilization (HCRU) were also collected via patient questionnaires and retrospective chart review (GA group only). Overall vision-related functioning and quality of life (QoL) were compared between the GA and non-GA groups using the National Eye Institute Visual Function Questionnaire-25 (NEI-VFQ-25) composite and subscales, and change in vision over the past year was assessed using the Global Rating of Change Scale. RESULTS: Vision-related functioning and QoL were poorer in patients with vs without GA (n=137 vs 52), as demonstrated by significantly lower NEI-VFQ-25 composite score (mean, 53.1 vs 84.5 points, respectively; P<0.001), as well as lower subscale scores for near activities, distance activities, dependency, driving, social functioning, mental health, role difficulties, color vision, and peripheral vision. Substantially more patients with GA than without GA reported worsening in vision over the past year (82% vs 25%, respectively; odds ratio, 13.55; P<0.001). In the GA group, associated mean annual costs for direct ophthalmological resource use per patient amounted to €1772 (mostly for tests/procedures), and for indirect ophthalmological resource use, €410 (mostly for general practitioner visits). CONCLUSION: Patients with GA experience a poorer level of vision-related function and QoL than their peers, especially in relation to driving. GA is also associated with notable HCRU/associated costs, mostly direct costs attributed to diagnostic tests/procedures.

Living with Geographic Atrophy: An Ethnographic Study.

INTRODUCTION: The specific impact from the patient's perspective of geographic atrophy (GA), an advanced form of age-related macular degeneration (AMD), is not well understood. METHODS: An ethnographic study was conducted to understand the impact of bilateral GA secondary to AMD on daily functioning by observing regular activities performed at home and through semi-structured interviews. Eligible subjects had a definitive GA diagnosis, including presence of drusen, GA lesion size of at least one disc area in the better-seeing eye, and no other confounding ophthalmologic diagnosis. Data were collected via video recordings and field notes, and analyzed by coding video transcripts. RESULTS: Functional impact domains affecting more than two of the 16 subjects from the United Kingdom, United States, or Germany were activities of daily living (difficulty reading, n = 16; driving, n = 12; and watching movies, television, or theater, n = 11), emotional (frustration, and fear of blindness, n = 7 each), social/leisure (interference with hobbies, n = 8, and diminished social activities, n = 4), physical (n = 4), and financial (n = 10). Subjects with a best-corrected visual acuity (BCVA) of 20/100 or better in the better-seeing eye (n = 10) reported similar functional impacts to those with a BCVA of worse than 20/100 in their better-seeing eye (n = 5). CONCLUSION: This study helps address gaps in patient-focused research into GA, which negatively impacts the day-to-day functioning of patients. Larger qualitative and quantitative studies are needed to quantify patient experiences and assess the correlation between BCVA score and impact of GA. FUNDING: F. Hoffmann-La Roche Ltd.

Adaptation and validation of the Urticaria Patient Daily Diary for adolescents.

The Urticaria Patient Daily Diary, including the Urticaria Activity Score, has recently been validated in adults with chronic idiopathic urticaria (CIU), but its validity in adolescents is unknown. This study was designed to (1) assess the content validity of the Adolescent Urticaria Patient Daily Diary and, (2) collect exploratory data on symptom experiences, sleep interference, and health-related quality of life (HRQOL) of adolescents with CIU. The Urticaria Patient Daily Diary was modified to increase its relevance with an adolescent population. A qualitative, cross-sectional, multicenter study was then conducted in the United States so that adolescent subjects could provide information on the impact of urticaria on their lives and comment on the diary. Data were collected via in-person semistructured interviews with subjects 12-17 years of age with moderate-to-severe CIU. The most bothersome symptom was itching (44%). The impact of CIU on HRQOL varied. The majority of subjects (78%) reported waking up at least once a night. Overall, subjects found the diary to be clear, easy to comprehend, and easy to complete. Revisions were made to the diary based on feedback from subjects. After nine interviews, no new information was received. The symptoms of CIU are bothersome to adolescents, particularly itch, and urticaria has a negative impact on the sleep of adolescent patients. The final Adolescent Urticaria Patient Daily Diary has evidence of content validity in patients with CIU ranging from 12 to 17 years of age.

Gaining efficiencies: resources and demand for dialysis around the globe.

OBJECTIVES: End-stage renal disease (ESRD) is a debilitating condition resulting in death unless treated. Treatment options are transplantation and dialysis. Alternative dialysis modalities are peritoneal dialysis (PD) and hemodialysis (HD), each of which has been shown to produce similar outcomes and survival. Nevertheless, the financial implications of each modality are different and these differences vary by country, especially in the developing world. Changes in clinically appropriate dialysis delivery leading to more efficient use of resources would increase the resources available to treat ESRD or other disabling conditions. This article outlines the relative advantages of HD and PD and uses budget impact analysis to estimate the country-specific, 5-year financial implications on total dialysis costs assuming utilization shifts from HD to PD in two high-income (UK, Singapore), three upper-middle-income (Mexico, Chile, Romania), and three lower-middle-income (Thailand, China, Colombia) countries. RESULTS: Peritoneal dialysis is a clinically effective dialysis option that can be significantly cost-saving compared to HD, even in developing countries. CONCLUSIONS: The magnitude of costs associated with treating ESRD patients globally is large and growing. PD is a clinically effective dialysis option that can be used by a majority of ESRD patients and can also be significantly cost-saving compared to HD therapy. Increasing clinically appropriate PD use would substantially reduce health-care costs and help health-care systems meet ever-tightening budget constraints.

Reimbursement and economic factors influencing dialysis modality choice around the world.

The worldwide incidence of kidney failure is on the rise and treatment is costly; thus, the global burden of illness is growing. Kidney failure patients require either a kidney transplant or dialysis to maintain life. This review focuses on the economics of dialysis. Alternative dialysis modalities are haemodialysis (HD) and peritoneal dialysis (PD). Important economic factors influencing dialysis modality selection include financing, reimbursement and resource availability. In general, where there is little or no facility or physician reimbursement or payment for PD, the share of PD is very low. Regarding resource availability, when centre HD capacity is high, there is an incentive to use that capacity rather than place patients on home dialysis. In certain countries, there is interest in revising the reimbursement structure to favour home-based therapies, including PD and home HD. Modality selection is influenced by employment status, with an association between being employed and PD as the modality choice. Cost drivers differ for PD and HD. PD is driven mainly by variable costs such as solutions and tubing, while HD is driven mainly by fixed costs of facility space and staff. Many cost comparisons of dialysis modalities have been conducted. A key factor to consider in reviewing cost comparisons is the perspective of the analysis because different costs are relevant for different perspectives. In developed countries, HD is generally more expensive than PD to the payer. Additional research is needed in the developing world before conclusive statements may be made regarding the relative costs of HD and PD.