Retina
Finding an AMD Holy Grail
Ongoing European study searches for intermediate AMD markers.
Gearoid Tuohy
Published: Wednesday, February 1, 2023
By Dr Gearóid Tuohy
With 700 patients enrolled and well underway, the European MACUSTAR project aims to develop novel clinical endpoints for those with intermediate age-related macular degeneration (iAMD).
There is a significant unmet need for treatment options in both early and intermediate AMD. While there are some valuable treatments for late-stage non-neovascular AMD (i.e., geographic atrophy)—using particular agents to slow the progression of the disease—the “holy grail” for a therapy in the ophthalmic community is a process to intervene earlier in the pathology, preventing the later stages of the disease and, thus, minimising visual loss.
The MACUSTAR project will characterise the functional deficits in iAMD and develop and validate functional, structural, and patient-reported outcome measures. Additional goals include investigating risk factors for progression from iAMD to late AMD, according to Frank Holz MD.
As there may be no universally accepted clinical endpoints for early or intermediate stages of the disease, Professor Holz commented that accomplishing this goal might be challenging. Designing, using, and measuring the “right endpoints,” therefore, will be imperative for MACUSTAR.
“Measuring a patient with intermediate AMD with BCVA would not be helpful, as it may maintain for a long period until it crashes into late-stage disease development,” he explained. “So we need other endpoints accepted by the regulatory authorities to test interventions in these earlier stages.”
MACUSTAR investigators across Europe are assessing methods for structural, functional, and patient-reported outcome tests; reliable test/retest processes; and how the community can differentiate between different neighbouring disease states and how these may change over time.
The project covers this gap in the research work to develop key outcomes supported by expert academic, clinical, and commercial organisations. MACUSTAR will follow more than 700 AMD patients over three years, using state-of-theart imaging techniques, vision testing, and patient-reported outcome measures that capture the disease’s impact on quality of life.
The continent-wide project has 20 clinical sites to date. Each site collects data including mean and point-wise retinal sensitivities on scotopic/mesopic fundus-controlled perimetry, low-luminance visual acuity (LLVA), low luminance deficit, Moorfields Acuity Chart visual acuity, dark adaptation absolute threshold and rod intercept time, and several more assays. Sites also collect patient-reported low-luminance functioning.
One of MACUSTAR’s valuable research outcomes so far is further validation of the “Vision Impairment in Low Luminance (VILL) questionnaire”, which captures visual functioning and vision-related quality of life (VRQoL) under low luminance, low-contrast conditions relevant to AMD.
The relevance of this work only increases as other existing patient-reported outcome instruments do not fulfil regulator development requirements or sufficiently capture AMD patients’ difficulties. The new VILL questionnaire now supports the psychometric performance of patients, including internal consistency, item fit, subscale structure, test/retest reliability, and construct validity in a multinational, multilanguage setting. Prof Holz noted this research could impact clinical research, practice, and policy in the future.
The Innovative Medicines Initiative (IMI), with support from the European Commission and the European Federation of Pharmaceutical Industries and Associations (EFPIA), funds the €16.2M project—scheduled to complete in spring 2024 after running for approximately 6.5 years.
For more information on MACUSTAR, please visit www.macustar.eu
Prof Holz spoke at the 22nd EURETINA Congress in Hamburg.
Frank G Holz MD is Chair of the Department of Ophthalmology at University Hospital Bonn, Germany. frank.holz@ukb.uni-bonn.de
Tags: AMD
Latest Articles
Glaucoma Treatment Under Pressure
New techniques and technologies add to surgeons’ difficult decisions
Outside the Box, Inside the Pipeline
Researchers are tackling glaucoma diagnosis and treatment from all sides.
The EHDS Is Ready for the Green Light
If proposal is approved, Europe could see better access to, and exchange and use of, health data.
ESCRS to Release Guidelines for Cataract and Refractive Surgery
Comprehensive approach to the safest and most effective modern surgery.
Barry Fellowship Opens Up ‘Whole New Field of Thought’
The 2022 recipient combines theoretical and practical to learn new treatments.
Digitalising the OR—Experience and Perspectives
Benefits include saving time and improving outcomes.
Dynamic Measures Needed for Quality of Vision
Functional visual acuity testing and straylight metering may better reflect real-world conditions.
What Is Stopping Digital OR Adoption?
Ophthalmologists know the benefits—now it’s time to construct the right plan.
Time to Move Beyond Monofocal IOLs?
European surgeons appear hesitant to first offer other presbyopia-correcting options to patients.