June 2017
Volume 58, Issue 8
Open Access
ARVO Annual Meeting Abstract  |   June 2017
How do patients rate their subjective symptoms after CNGA3 gene therapy:
First application of the instrument A3-PRO
Author Affiliations & Notes
  • Barbara Wilhelm
    Centre for Ophthalmology, STZ eyetrial, Tuebingen, Germany
  • Andreas Koegel
    Institute Schreier, Tübingen, Tübingen, Germany
  • Nadine Kahle
    University Eye Hospital, Centre for Ophthalmology, University Hospitals Tübingen, Tübingen, Germany
  • Tobias Peters
    University Eye Hospital, Centre for Ophthalmology, University Hospitals Tübingen, Tübingen, Germany
  • Stylianos Michalakis
    Center for Integrated Protein Science Munich (CIPSM) at the Department of Pharmacy - Center for Drug Research, Ludwig-Maximilians-Universität München, München, Germany, Germany
  • Martin Biel
    Center for Integrated Protein Science Munich (CIPSM) at the Department of Pharmacy - Center for Drug Research, Ludwig-Maximilians-Universität München, München, Germany, Germany
  • Mathias Seeliger
    Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospitals Tübingen, Tübingen, Germany
  • Eberhart Zrenner
    Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospitals Tübingen, Tübingen, Germany
  • Bernd Wissinger
    Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospitals Tübingen, Tübingen, Germany
  • Susanne Kohl
    Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospitals Tübingen, Tübingen, Germany
  • Marius Ueffing
    Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospitals Tübingen, Tübingen, Germany
  • Karl Ulrich Bartz-Schmidt
    University Eye Hospital, Centre for Ophthalmology, University Hospitals Tübingen, Tübingen, Germany
  • Dominik M Fischer
    University Eye Hospital, Centre for Ophthalmology, University Hospitals Tübingen, Tübingen, Germany
  • Ditta Zobor
    Institute for Ophthalmic Research, Centre for Ophthalmology, University Hospitals Tübingen, Tübingen, Germany
  • Footnotes
    Commercial Relationships   Barbara Wilhelm, None; Andreas Koegel, None; Nadine Kahle, None; Tobias Peters, None; Stylianos Michalakis, Eyeserv GmbH (P); Martin Biel, Eyeserv GmbH (P); Mathias Seeliger, Eyeserv GmbH (P); Eberhart Zrenner, Eyeserv GmbH (P); Bernd Wissinger, None; Susanne Kohl, None; Marius Ueffing, None; Karl Ulrich Bartz-Schmidt, None; Dominik Fischer, None; Ditta Zobor, None
  • Footnotes
    Support  Tistou and Charlotte Kerstan Stiftung, Germany, RD-Cure Project
Investigative Ophthalmology & Visual Science June 2017, Vol.58, 4678. doi:
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      Barbara Wilhelm, Andreas Koegel, Nadine Kahle, Tobias Peters, Stylianos Michalakis, Martin Biel, Mathias Seeliger, Eberhart Zrenner, Bernd Wissinger, Susanne Kohl, Marius Ueffing, Karl Ulrich Bartz-Schmidt, Dominik M Fischer, Ditta Zobor; How do patients rate their subjective symptoms after CNGA3 gene therapy:
      First application of the instrument A3-PRO. Invest. Ophthalmol. Vis. Sci. 2017;58(8):4678.

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      © ARVO (1962-2015); The Authors (2016-present)

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Abstract

Purpose : There are limitations of wide-spread standard measures for vision-oriented quality of life like the VFQ25. Specific symptoms in low vision caused by hereditary disorders and their short-term variations during an interventional trial may not be sufficiently reflected. This is especially true for Achromatopsia, a hereditary retinal degeneration with severe visual impairment in daily life, its leading symptom being glare in normal daylight conditions. Our aim was to develop a patient reported outcome (PRO) measure as a complement to the VFQ25 in the first gene therapy trial in CNGA3-linked Achromatopsia.

Methods : A3-PRO is a short questionnaire to measure the patients’ subjective visual performance and its development during therapy in a clinical trial, using five criteria – each represented by a single item. In order to also consider the possibility of sporadic deteriorations we provided a symmetric response scale, although distinctive declines were not expected. The patients rate the development of all aspects on a scale ranging from “almost always worse” (-4) to “almost always better” (+4) in nine indicated steps, including “unchanged” (0) as middle category. The questions are read by an interviewer, so the questionnaire contains a brief instruction and supplementary explanations to each item. The inventory is completed by a simple five-level scale of the patient’s overall satisfaction with the therapy. The questions were deduced from the key symptoms identified in an observational trial in the target cohort.

Results : A3-PRO was easy to assess for both patient and interviewer. The time need was appr. five minutes per test. Interim results of the ongoing trial (n=9; 1 f, 8 m; age range: 24 to 59 y) are reported (NCT02610582). The questionnaire was able to reflect intra- and interindividual changes of subjective symptoms over the time of the study in all three dosage groups. Our preliminary findings need to be confirmed in the final analysis.

Conclusions : A3-PRO is an easy applicable tool for the investigation of Achromatopsia. It provides valuable quantitative data on patient perception of symptom development after treatment and is superior to open, non-standardized questions in the clinical trial setting.

This is an abstract that was submitted for the 2017 ARVO Annual Meeting, held in Baltimore, MD, May 7-11, 2017.

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