July 2018
Volume 59, Issue 9
Open Access
ARVO Annual Meeting Abstract  |   July 2018
The Natural History of the Progression of Choroideremia (NIGHT) Study: Longitudinal Changes in Visual Acuity over 12 Months
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
Author Affiliations & Notes
  • Mark E Pennesi
    Ophthalmology, Casey Eye Institute - OHSU, Portland, Oregon, United States
  • Byron L Lam
    Bascom Palmer Eye Institute, University of Miami Health System, Pinecrest, Florida, United States
  • M Dominik Fischer
    Centre for Opthalmology, University of Tubingen, Tubingen, Germany
  • Eeva-Marja Kaarina Sankila
    Department of Ophthalmology, Helsinki University, Helsinki, Finland
  • Frank G. Holz
    Ophthalmology, University of Bonn, Bonn, Germany
  • Robert E MacLaren
    Nuffield Laboratory of Ophthalmology, University of Oxford, Oxford, United Kingdom
  • David G Birch
    Retina Foundation of the Southwest, Dallas, Texas, United States
  • Ian M MacDonald
    Department of Ophthalmology and Visual Sciences, University of Alberta, Edmonton, Alberta, Canada
  • Graeme Black
    Central Manchester University Hospitals Trust, Manchester Centre for Genomic Medicine, Manchester, United Kingdom
  • Carel C B Hoyng
    Department of Ophthalmology, Radboud University Medical Center, Njimegen, Netherlands
  • Neil M Bressler
    Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, Maryland, United States
  • Stephen H. Tsang
    Department of Ophthalmology and of Pathology and Cell Biology, Columbia University, New York, New York, United States
  • Kimberly E Stepien
    Department of Ophthalmology & Visual Sciences, University of WI-Madison, Madison, Wisconsin, United States
  • Michael S Ip
    Doheny Eye Institute, Doheny Image Reading Center, Los Angeles, California, United States
  • Vedran Pavlovic
    Nightstar Therapeutics, London, United Kingdom
  • Aniz Girach
    Nightstar Therapeutics, London, United Kingdom
  • Footnotes
    Commercial Relationships   Mark Pennesi, Nightstar Therapeutics (F); Byron Lam, Nightstar Therapeutics (F); M Dominik Fischer, Nightstar (C), Nightstar (F); Eeva-Marja Sankila, Nightstar Therapeutics (F); Frank Holz, Nightstar (F); Robert MacLaren, Nightstar (F), Nightstar (I), Nightstar Therapeutics (C); David Birch, Nightstar (F), Nightstar Therapeutics (C); Ian MacDonald, Nightstar (F); Graeme Black, Nightstar (F), Nightstar (C); Carel Hoyng, Nightstar (F); Neil Bressler, Nightstar (F); Stephen Tsang, Nightstar (F); Kimberly Stepien, Nightstar (F); Michael Ip, Nightstar (C), Nightstar (F); Vedran Pavlovic, Nightstar (E); Aniz Girach, Nightstar (E)
  • Footnotes
    Support  None
Investigative Ophthalmology & Visual Science July 2018, Vol.59, 3898. doi:https://doi.org/
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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)

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Abstract

Purpose : There are very few prospective natural history studies in Choroideremia (CHM). The NIGHT study is a prospective, multi-center, observational study in CHM (NCT03359551).

Methods : 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.

Results : 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.

Conclusions : 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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Figure 1. Histogram of Month 12 change-from-baseline VA in CHM patients.

Figure 1. Histogram of Month 12 change-from-baseline VA in CHM patients.

Figure 1. Histogram of Month 12 change-from-baseline VA in CHM patients.
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This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License.
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Copyright © 2015 Association for Research in Vision and Ophthalmology.
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