A Prospective, Observational, Non-interventional Clinical Study of Participants With Choroideremia: The NIGHT Study.

Autor: Maclaren RE; From the Oxford Eye Hospital (R.E.M.), Oxford University Hospitals NHS Foundation Trust, Oxford, UK. Electronic address: maclaren@eye.ox.ac.uk., Lam BL; Bascom Palmer Eye Institute (B.L.L.), University of Miami, Miami, Florida, USA., Fischer MD; University Eye Hospital, Centre for Ophthalmology (M.D.F.), University Hospital Tübingen, Tübingen, Germany., Holz FG; Department of Ophthalmology (F.-G.H.), University of Bonn, Bonn, Germany., Pennesi ME; Department of Ophthalmology, Casey Eye Institute (M.E.P.), Oregon Health & Science University, Portland, Oregon, USA., Birch DG; Retina Foundation of the Southwest (D.G.B.), Dallas, Texas, USA., Sankila EM; Department of Ophthalmology (E.-M.S.), University of Helsinki and Helsinki University Hospital, Helsinki, Finland., Meunier IA; National Reference Centre for Inherited Sensory Diseases (I.A.M.), University of Montpellier, Montpellier University Hospital, Montpellier, France., Stepien KE; Department of Ophthalmology and Visual Sciences (K.E.S.), University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin, USA., Sallum JMF; Department of Ophthalmology and Visual Sciences (J.M.F.S.), Federal University of São Paulo, São Paulo, São Paulo, Brazil., Li J; Biogen Inc. (J.L., D.Y., S.P., J.A.G.), Cambridge, Massachusetts, USA., Yoon D; Biogen Inc. (J.L., D.Y., S.P., J.A.G.), Cambridge, Massachusetts, USA., Panda S; Biogen Inc. (J.L., D.Y., S.P., J.A.G.), Cambridge, Massachusetts, USA., Gow JA; Biogen Inc. (J.L., D.Y., S.P., J.A.G.), Cambridge, Massachusetts, USA.
Jazyk: angličtina
Zdroj: American journal of ophthalmology [Am J Ophthalmol] 2024 Jul; Vol. 263, pp. 35-49. Date of Electronic Publication: 2024 Feb 03.
DOI: 10.1016/j.ajo.2024.01.022
Abstrakt: Purpose: The NIGHT study aimed to assess the natural history of choroideremia (CHM), an X-linked inherited chorioretinal degenerative disease leading to blindness, and determine which outcomes would be the most sensitive for monitoring disease progression.
Design: A prospective, observational, multicenter cohort study.
Methods: Males aged ≥18 years with genetically confirmed CHM, visible active disease within the macular region, and best-corrected visual acuity (BCVA) ≥34 Early Treatment Diabetic Retinopathy Study (ETDRS) letters at baseline were assessed for 20 months. The primary outcome was the change in BCVA over time at Months 4, 8, 12, 16, and 20. A range of functional and anatomical secondary outcome measures were assessed up to Month 12, including retinal sensitivity, central ellipsoid zone (EZ) area, and total area of fundus autofluorescence (FAF). Additional ocular assessments for safety were performed.
Results: A total of 220 participants completed the study. The mean BCVA was stable over 20 months. Most participants (81.4% in the worse eye and 77.8% in the better eye) had change from baseline > -5 ETDRS letters at Month 20. Interocular symmetry was low overall. Reductions from baseline to Month 12 were observed (worse eye, better eye) for retinal sensitivity (functional outcome; -0.68 dB, -0.48 dB), central EZ area (anatomical outcome; -0.276 mm 2 , -0.290 mm 2 ), and total area of FAF (anatomical outcome; -0.605 mm 2 , -0.533 mm 2 ). No assessment-related serious adverse events occurred.
Conclusions: Retinal sensitivity, central EZ area, and total area of FAF are more sensitive than BCVA in measuring the natural progression of CHM.
(Copyright © 2024 Elsevier Inc. All rights reserved.)
Databáze: MEDLINE