TY - JOUR
T1 - The Michigan Retinal Degeneration Questionnaire
T2 - A Patient-Reported Outcome Instrument for Inherited Retinal Degenerations
AU - Lacy, Gabrielle D.
AU - Abalem, Maria Fernanda
AU - Andrews, Chris A.
AU - Popova, Lilia T.
AU - Santos, Erin P.
AU - Yu, Gina
AU - Rakine, Hanan Y.
AU - Baig, Natasha
AU - Ehrlich, Joshua R.
AU - Fahim, Abigail T.
AU - Branham, Kari H.
AU - Stelmack, Joan A.
AU - Swenor, Bonnielin K.
AU - Dagnelie, Gislin
AU - Musch, David C.
AU - Jayasundera, K. Thiran
N1 - Funding Information:
All authors have completed and submitted the ICMJE form for disclosure of potential conflicts of interest. Michigan Retinal Degeneration Questionnaire is copyrighted material of the University of Michigan. All rights reserved. For licensing information, contact the University of Michigan Technology Transfer Office at techtransfer@umich.edu . Funding/Support: This research was supported by the National Institute of Health grants TL1TR002242 (G.D.L.), K23EY027848 (J.R.E.), K12EY022299 (A.T.F.), and K23EY026985 (K.T.J.). The authors would like to thank Lindsay Godsey, Adam R. Holmes, Milan K. Patel, and Naheed W. Khan for their contributions to this work. Author Contributions: G.D.L.: Conceptualization, methodology, investigation, writing, visualization, and project administration. M.F.A.: Conceptualization, methodology, investigation, writing, supervision, and project administration. C.A.A.: Methodology, formal analysis, investigation, writing, visualization, and funding acquisition. L.T.P., E.P.S., G.Y., H.Y.R., N.B.: Conceptualization and investigation. J.R.E.: Conceptualization, validation, and writing—review and editing. A.T.F., K.H.B.: Investigation and resources. J.A.S., B.K.S., G.D.: Validation and writing—review and editing. D.C.M.: Conceptualization, methodology, validation, and writing—review and editing. K.T.J.: Conceptualization, methodology, investigation, resources, writing, supervision, project administration, and funding acquisition. Financial Disclosures: C.A.A. and D.C.M. are consultants to Applied Genetics Technologies Corporation, who wish to make use of this patient-reported outcome (or a defined subset) for their clinical trials in planning. This manuscript has not been published elsewhere and has not been submitted simultaneously for publication elsewhere. All authors attest that they meet the current ICMJE criteria for authorship.
Funding Information:
All authors have completed and submitted the ICMJE form for disclosure of potential conflicts of interest. Michigan Retinal Degeneration Questionnaire is copyrighted material of the University of Michigan. All rights reserved. For licensing information, contact the University of Michigan Technology Transfer Office at techtransfer@umich.edu. Funding/Support: This research was supported by the National Institute of Health grants TL1TR002242 (G.D.L.), K23EY027848 (J.R.E.), K12EY022299 (A.T.F.), and K23EY026985 (K.T.J.). The authors would like to thank Lindsay Godsey, Adam R. Holmes, Milan K. Patel, and Naheed W. Khan for their contributions to this work. Author Contributions: G.D.L.: Conceptualization, methodology, investigation, writing, visualization, and project administration. M.F.A.: Conceptualization, methodology, investigation, writing, supervision, and project administration. C.A.A.: Methodology, formal analysis, investigation, writing, visualization, and funding acquisition. L.T.P. E.P.S. G.Y. H.Y.R. N.B.: Conceptualization and investigation. J.R.E.: Conceptualization, validation, and writing—review and editing. A.T.F. K.H.B.: Investigation and resources. J.A.S. B.K.S. G.D.: Validation and writing—review and editing. D.C.M.: Conceptualization, methodology, validation, and writing—review and editing. K.T.J.: Conceptualization, methodology, investigation, resources, writing, supervision, project administration, and funding acquisition. Financial Disclosures: C.A.A. and D.C.M. are consultants to Applied Genetics Technologies Corporation, who wish to make use of this patient-reported outcome (or a defined subset) for their clinical trials in planning. This manuscript has not been published elsewhere and has not been submitted simultaneously for publication elsewhere. All authors attest that they meet the current ICMJE criteria for authorship.
Publisher Copyright:
© 2020 Elsevier Inc.
PY - 2021/2
Y1 - 2021/2
N2 - Purpose: To create a psychometrically validated patient-reported outcome measure for inherited retinal degenerations. Design: Qualitative and quantitative patient-reported outcome (PROs) questionnaire development using item response theory validation. Methods: One hundred twenty-eight patients with a diagnosis of an inherited retinal degeneration at the Kellogg Eye Center (University of Michigan) were recruited and administered a 166-item questionnaire comprising 7 expert-defined domains. The questionnaire was re-administered 4-16 days later to a subset of 25 participants to assess test-retest variability. Graded response models were fit by Cai's Metropolis-Hastings Robbins-Monro algorithm using the R (version 3.6.3) package mirt. Model data were fit to assess questionnaire dimensionality, to estimate item information, and to score participants. Poorly functioning items were removed, and the model was refit to create the final questionnaire. Results: The psychometrically validated PROs measure was reduced to a 59-item questionnaire measuring 7 unidimesnional domains: central vision, color vision, contrast sensitivity, scotopic function, photopic peripheral vision, mesopic peripheral vision, and photosensitivity. A total of 39 items were removed because of poor factor loading, low item information, poor person-ability differentiation, or high item-level interdependence. This novel questionnaire produces a reliable domain score for person ability that does not show significant test-retest variability across repeated administration. Conclusions: The final PRO questionnaire, known as the Michigan Retinal Degeneration Questionnaire, is psychometrically validated and available for use in the evaluation of patients with inherited retinal degenerations.
AB - Purpose: To create a psychometrically validated patient-reported outcome measure for inherited retinal degenerations. Design: Qualitative and quantitative patient-reported outcome (PROs) questionnaire development using item response theory validation. Methods: One hundred twenty-eight patients with a diagnosis of an inherited retinal degeneration at the Kellogg Eye Center (University of Michigan) were recruited and administered a 166-item questionnaire comprising 7 expert-defined domains. The questionnaire was re-administered 4-16 days later to a subset of 25 participants to assess test-retest variability. Graded response models were fit by Cai's Metropolis-Hastings Robbins-Monro algorithm using the R (version 3.6.3) package mirt. Model data were fit to assess questionnaire dimensionality, to estimate item information, and to score participants. Poorly functioning items were removed, and the model was refit to create the final questionnaire. Results: The psychometrically validated PROs measure was reduced to a 59-item questionnaire measuring 7 unidimesnional domains: central vision, color vision, contrast sensitivity, scotopic function, photopic peripheral vision, mesopic peripheral vision, and photosensitivity. A total of 39 items were removed because of poor factor loading, low item information, poor person-ability differentiation, or high item-level interdependence. This novel questionnaire produces a reliable domain score for person ability that does not show significant test-retest variability across repeated administration. Conclusions: The final PRO questionnaire, known as the Michigan Retinal Degeneration Questionnaire, is psychometrically validated and available for use in the evaluation of patients with inherited retinal degenerations.
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U2 - 10.1016/j.ajo.2020.08.032
DO - 10.1016/j.ajo.2020.08.032
M3 - Article
C2 - 32858027
AN - SCOPUS:85096389832
SN - 0002-9394
VL - 222
SP - 60
EP - 68
JO - American journal of ophthalmology
JF - American journal of ophthalmology
ER -