Literature DB >> 32571121

Content generation for patient-reported outcome measures for retinal degeneration therapeutic trials.

Gabrielle D Lacy1, Maria Fernanda Abalem1,2, Lilia T Popova1, Erin P Santos1, Gina Yu1, Hanan Y Rakine1, Julie M Rosenthal1, Joshua R Ehrlich1, David C Musch1,3, K Thiran Jayasundera1.   

Abstract

PURPOSE: Generate content for a patient-reported outcome (PRO) measure for use in future clinical trials for inherited retinal degenerations.
METHODS: Patients at the University of Michigan Kellogg Eye Center with a clinical diagnosis of inherited retinal degeneration with varying phenotypes were recruited for interviews. First, in-depth interviews were performed to solicit a wide range of patient experiences pertaining to visual function. Coders qualitatively analyzed the transcripts from these interviews using Atlas.ti software (Version 8.1.3 (522)) to draft questionnaire items. Next, the questionnaire was tested and refined based on participant feedback in cognitive interviews and administrator feedback in the pilot survey administration (pilot interviews).
RESULTS: A total of 55 participants with a clinical diagnosis of inherited retinal degeneration were interviewed throughout the three study phases: in-depth interviews (n = 26), cognitive interviews (n = 16), and pilot interviews (n = 13). Coded items were analyzed for frequency of occurrence and related themes, then organized into common domains. Within each domain, PRO items were drafted to address the functional limitations or adaptations experienced by patients.
CONCLUSIONS: Items for a PRO measure have been drafted and evaluated for interpretability in the target inherited retinal degeneration patient population. Content validity for the items was established through a process of in-depth interviews, cognitive interviews, and pilot interviews.

Entities:  

Keywords:  Michigan retinal degeneration questionnaire; Patient-reported outcomes; gene therapy; inherited retinal degeneration; retinal dystrophy

Mesh:

Year:  2020        PMID: 32571121      PMCID: PMC7503769          DOI: 10.1080/13816810.2020.1776337

Source DB:  PubMed          Journal:  Ophthalmic Genet        ISSN: 1381-6810            Impact factor:   1.803


  26 in total

1.  Perceived and actual performance of daily tasks: relationship to visual function tests in individuals with retinitis pigmentosa.

Authors:  J P Szlyk; W Seiple; G A Fishman; K R Alexander; S Grover; C L Mahler
Journal:  Ophthalmology       Date:  2001-01       Impact factor: 12.079

2.  Whiners and deniers - what does self-rated health measure?

Authors:  Audrey Layes; Yukiko Asada; George Kepart
Journal:  Soc Sci Med       Date:  2011-12-03       Impact factor: 4.634

3.  Development and Psychometric Assessment of Novel Item Banks for Hereditary Retinal Diseases.

Authors:  Mallika Prem Senthil; Jyoti Khadka; John De Roach; Tina Lamey; Terri McLaren; Isabella Campbell; Eva K Fenwick; Ecosse L Lamoureux; Konrad Pesudovs
Journal:  Optom Vis Sci       Date:  2019-01       Impact factor: 1.973

4.  Impact of Retinitis Pigmentosa on Quality of Life, Mental Health, and Employment Among Young Adults.

Authors:  Anne Elisabeth Chaumet-Riffaud; Philippe Chaumet-Riffaud; Anaelle Cariou; Céline Devisme; Isabelle Audo; José-Alain Sahel; Saddek Mohand-Said
Journal:  Am J Ophthalmol       Date:  2017-02-22       Impact factor: 5.258

5.  The COSMIN checklist for assessing the methodological quality of studies on measurement properties of health status measurement instruments: an international Delphi study.

Authors:  Lidwine B Mokkink; Caroline B Terwee; Donald L Patrick; Jordi Alonso; Paul W Stratford; Dirk L Knol; Lex M Bouter; Henrica C W de Vet
Journal:  Qual Life Res       Date:  2010-02-19       Impact factor: 4.147

6.  Worse-than-usual visual fields measured in retinitis pigmentosa related to episodically decreased general health.

Authors:  Ava Katherine Bittner; Jennifer A Haythornthwaite; Marie Diener-West; Gislin Dagnelie
Journal:  Br J Ophthalmol       Date:  2012-12-04       Impact factor: 4.638

7.  The meaning of vaguely quantified frequency response options on a quality of life scale depends on respondents' medical status and age.

Authors:  Stefan Schneider; Arthur A Stone
Journal:  Qual Life Res       Date:  2016-04-12       Impact factor: 4.147

Review 8.  Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium.

Authors:  Debra A Thompson; Robin R Ali; Eyal Banin; Kari E Branham; John G Flannery; David M Gamm; William W Hauswirth; John R Heckenlively; Alessandro Iannaccone; K Thiran Jayasundera; Naheed W Khan; Robert S Molday; Mark E Pennesi; Thomas A Reh; Richard G Weleber; David N Zacks
Journal:  Invest Ophthalmol Vis Sci       Date:  2015-02-09       Impact factor: 4.799

9.  Copy-number variation contributes 9% of pathogenicity in the inherited retinal degenerations.

Authors:  Erin Zampaglione; Benyam Kinde; Emily M Place; Daniel Navarro-Gomez; Matthew Maher; Farzad Jamshidi; Sherwin Nassiri; J Alex Mazzone; Caitlin Finn; Dana Schlegel; Jason Comander; Eric A Pierce; Kinga M Bujakowska
Journal:  Genet Med       Date:  2020-02-10       Impact factor: 8.822

10.  Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial.

Authors:  Robert E MacLaren; Markus Groppe; Alun R Barnard; Charles L Cottriall; Tanya Tolmachova; Len Seymour; K Reed Clark; Matthew J During; Frans P M Cremers; Graeme C M Black; Andrew J Lotery; Susan M Downes; Andrew R Webster; Miguel C Seabra
Journal:  Lancet       Date:  2014-01-16       Impact factor: 79.321

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  2 in total

1.  The Michigan Vision-Related Anxiety Questionnaire: A Psychosocial Outcomes Measure for Inherited Retinal Degenerations.

Authors:  Gabrielle D Lacy; Maria Fernanda Abalem; Chris A Andrews; Rebhi Abuzaitoun; Lilia T Popova; Erin P Santos; Gina Yu; Hanan Y Rakine; Natasha Baig; Joshua R Ehrlich; Abigail T Fahim; Kari H Branham; Bonnielin K Swenor; Paul R Lichter; Gislin Dagnelie; Joan A Stelmack; David C Musch; K Thiran Jayasundera
Journal:  Am J Ophthalmol       Date:  2020-12-09       Impact factor: 5.258

2.  The Michigan Retinal Degeneration Questionnaire: A Patient-Reported Outcome Instrument for Inherited Retinal Degenerations.

Authors:  Gabrielle D Lacy; Maria Fernanda Abalem; Chris A Andrews; Lilia T Popova; Erin P Santos; Gina Yu; Hanan Y Rakine; Natasha Baig; Joshua R Ehrlich; Abigail T Fahim; Kari H Branham; Joan A Stelmack; Bonnielin K Swenor; Gislin Dagnelie; David C Musch; K Thiran Jayasundera
Journal:  Am J Ophthalmol       Date:  2020-08-26       Impact factor: 5.258

  2 in total

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