71 research outputs found
Real-world treatment outcomes of neovascular Age-related Macular Degeneration in the Netherlands
Purpose: To compare treatment outcomes of treatment-naïve eyes with neovascular age-related macular degeneration (nAMD) with bevacizumab as the first-line treatment, according to the guidelines of the Dutch Ophthalmological Society, with those treated first with either ranibizumab or aflibercept, as used in many other countries, all treated using a treat-and-extend strategy. Methods: Data were obtained from the prospectively designed Fight Re
Defining a Minimum Set of Standardized Patient-centered Outcome Measures for Macular Degeneration
Purpose To define a minimum set of outcome measures for tracking, comparing, and improving macular degeneration care. Design Recommendations from a working group of international experts in macular degeneration outcomes registry development and patient advocates, facilitated by the International Consortium for Health Outcomes Measurement (ICHOM). Methods Modified Delphi technique, supported by structured teleconferences, followed by online surveys to drive consensus decisions. Potential outcomes were identified through literature review of outcomes collected in existing registries and reported in major clinical trials. Outcomes were refined by the working group and selected based on impact on patients, relationship to good clinical care, and feasibility of measurement in routine clinical practice. Results Standardized measurement of the following outcomes is recommended: visual functioning and quality of life (distance visual acuity, mobility and independence, emotional well-being, reading and accessing information); number of treatments; complications of treatment; and disease control. Proposed data collection sources include administrative data, clinical data during routine clinical visits, and patient-reported sources annually. Recording the following clinical characteristics is recommended to enable risk adjustment: age; sex; ethnicity; smoking status; baseline visual acuity in both eyes; type of macular degeneration; presence of geographic atrophy, subretinal fibrosis, or pigment epithelial detachment; previous macular degeneration treatment; ocular comorbidities. Conclusions The recommended minimum outcomes and pragmatic reporting standards should enable standardized, meaningful assessments and comparisons of macular degeneration treatment outcomes. Adoption could accelerate global improvements in standardized data gathering and reporting of patient-centered outcomes. This can facilitate informed decisions by patients and health care providers, plus allow long-term monitoring of aggregate data, ultimately improving understanding of disease progression and treatment responses
Nitric oxide releasing-dendrimers: an overview
Platforms able to storage, release or scavenge NO in a controlled and specific manner is interesting for biological applications. Among the possible matrices for these purposes, dendrimers are excellent candidates for that. These molecules have been used as drug delivery systems and exhibit interesting properties, like the possibility to perform chemical modifications on dendrimers surface, the capacity of storage high concentrations of compounds of interest in the same molecule and the ability to improve the solubility and the biocompatibility of the compounds bonded to it. This review emphasizes the recent progress in the development and in the biological applications of different NO-releasing dendrimers and the nitric oxide release pathways in these compounds
ALPK1 missense pathogenic variant in five families leads to ROSAH syndrome, an ocular multisystem autosomal dominant disorder
Purpose: To identify the molecular cause in five unrelated families
with a distinct autosomal dominant ocular systemic disorder we
called ROSAH syndrome due to clinical features of retinal dystrophy,
optic nerve edema, splenomegaly, anhidrosis, and migraine headache.
Methods: Independent discovery exome and genome sequencing
in families 1, 2, and 3, and confirmation in families 4 and 5.
Expression of wild-type messenger RNA and protein in human and
mouse tissues and cell lines. Ciliary assays in fibroblasts from
affected and unaffected family members.
Results: We found the heterozygous missense variant in the ɑkinase gene, ALPK1, (c.710C>T, [p.Thr237Met]), segregated with
disease in all five families. All patients shared the ROSAH
phenotype with additional low-grade ocular inflammation, pancytopenia, recurrent infections, and mild renal impairment in some.
ALPK1 was notably expressed in retina, retinal pigment epithelium,
and optic nerve, with immunofluorescence indicating localization
to the basal body of the connecting cilium of the photoreceptors,
and presence in the sweat glands. Immunocytofluorescence
revealed expression at the centrioles and spindle poles during
metaphase, and at the base of the primary cilium. Affected family
member fibroblasts demonstrated defective ciliogenesis.
Conclusion: Heterozygosity for ALPK1, p.Thr237Met leads to
ROSAH syndrome, an autosomal dominant ocular systemic
disorder
Management of diabetic retinopathy: A systematic review
10.1001/jama.298.8.902Journal of the American Medical Association2988902-916JAMA
Switching between ranibizumab and aflibercept for the treatment of neovascular age-related macular degeneration.
The introduction of antivascular endothelial growth factor agents such as ranibizumab and aflibercept has revolutionized the management of neovascular age-related macular degeneration. A number of randomized clinical trials have shown that ranibizumab and aflibercept produce similar efficacy and safety outcomes. Most of the switching studies published to date show that efficacy benefits are uncontrolled, retrospective trials with limitations in terms of their selection, monitoring, numbers, and assessment criteria. Based on the published literature to date, we propose arguments for and against switching antivascular endothelial growth factor agents, provide our own perspective on this topic, and suggest a focus for future research
Retinal vascular caliber changes after intravitreal triamcinolone treatment for diabetic macular edema
10.1167/iovs.08-1678Investigative Ophthalmology and Visual Science49114707-4711IOVS
Retinal Vascular Caliber and Macular Telangiectasia Type 2
10.1016/j.ophtha.2008.09.009Ophthalmology1162319-323OPHT
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