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Mark E Pennesi, Byron L Lam, M Dominik Fischer, Eeva-Marja Kaarina Sankila, Frank G. Holz, Robert E MacLaren, David G Birch, Ian M MacDonald, Graeme Black, Carel C B Hoyng, Neil M Bressler, Stephen H. Tsang, Kimberly E Stepien, Michael S Ip, Vedran Pavlovic, Aniz Girach; The Natural History of the Progression of Choroideremia (NIGHT) Study: Longitudinal Changes in Visual Acuity over 12 Months. Invest. Ophthalmol. Vis. Sci. 2018;59(9):3898. doi: https://doi.org/.
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© ARVO (1962-2015); The Authors (2016-present)
There are very few prospective natural history studies in Choroideremia (CHM). The NIGHT study is a prospective, multi-center, observational study in CHM (NCT03359551).
Adult participants attended 6 visits over a 20-month period. Best-corrected visual acuity (BCVA) letter score was measured using an Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Degree of visual impairment was defined as (1) >73 (>20/40), (2) 73-34 (20/40-20/200) and (3) <34 ETDRS (<20/200) letters. In this analysis, VA data from 346 eyes over a 12-month period was included.
At baseline, mean (±SD) BCVA letter score was 64 (±21.1; ~20/50), and 39%, 53%, and 8% of eyes had letter scores of >73, 73-34 and <34 ETDRS letters, respectively. Similar distribution of VA data was observed at Month 12, with mean BCVA letter score of 65 (±21.6, ~20/50), and 43%, 48% and 9% of eyes meeting aforementioned BCVA subgroups. During 12-months of follow-up, mean BCVA change-from-baseline was +0.7 (±6.25) letters. Sixty-four percent of eyes were within a 5-letter change from baseline, whilst 13% showed a decline in VA (≥5-letters loss) (Figure 1). An apparent improvement in VA (≥15-letters gain) was observed in 1% of eyes. However, review of available historical VA data in these patients revealed presence of aberrant baseline BCVA readings.
In keeping with this chronic degenerative disease, there was little change in mean VA during 12-months of follow-up, with apparent VA gain in a few rare cases attributed potentially to aberrant baseline readings. This highlights the utility of historical data, and importance of conducting robust prospective natural history studies prior to interventional trials.
This is an abstract that was submitted for the 2018 ARVO Annual Meeting, held in Honolulu, Hawaii, April 29 - May 3, 2018.
Figure 1. Histogram of Month 12 change-from-baseline VA in CHM patients.
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