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

Lacy, Gabrielle D.; Abalem, Maria Fernanda; Andrews, Chris; Popova, Lilia; Santos, Erin P.; Yu, Gina; Rakine, Hanan; Baig, Natasha; Ehrlich, Joshua R.; Fahim, Abigail T.; Branham, Kari; Stelmack, Joan A.; Swenor, Bonnielin K.; Dagnelie, Gislin; Musch, David C.; Jayasundera, Thiran

doi:10.1016/j.ajo.2020.08.032

Cited by 38 publications

(39 citation statements)

References 23 publications

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“…Item response theory analysis for the MVAQ was performed using a methodology similar to the development of the Michigan Retinal Degeneration Questionnaire. 12 Unidimensional domains were identified by factor analysis and then fit to a graded response model using the Cai Metropolis-Hastings Robbins-Monro algorithm 13 in the mirt package in R software. 14 Patientlevel vision anxiety is represented by u, which describes the latent trait measured by a singular domain.…”

Section: Approval From the University Of Michigan Institu-mentioning

confidence: 99%

Effects of duration and number of symptoms on vision-related anxiety in patients with Inherited Retinal Diseases

Popova

Abuzaitoun

Abalem

et al. 2022

Ophthalmic Genetics

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Section: Approval From the University Of Michigan Institu-mentioning

confidence: 99%

Effects of duration and number of symptoms on vision-related anxiety in patients with Inherited Retinal Diseases

Popova

Abuzaitoun

Abalem

et al. 2022

Ophthalmic Genetics

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“…The Michigan Retinal Degeneration Questionnaire and Michigan Vision-related Anxiety Questionnaire are patient-reported outcome measures for adult patients with IRD to assess their vision ability and associated psychosocial health, respectively [ 8 , 9 ]. These IRD-specific measures will begin to be incorporated into clinical trials studying voretigene neparvovec-rzyl as part of their outcomes and other observational studies.…”

Section: Discussionmentioning

confidence: 99%

“…That said, until recently IRD-specific PROMs did not exist. The Michigan Retinal Degeneration Questionnaire (MRDQ) and Michigan Vision-related Anxiety Questionnaire (MVAQ) were recently developed adult IRD-tailored PROMs that assess vision ability and associated psychosocial health, respectively [ 8 , 9 ]. The MRDQ generates an ability score from adult patient-reported visual function in domains representative of physiological visual function pathways and thereby can record a patient’s perspective on emerging therapeutics for these domains by using theta scores (item response theory) derived using a graded response model.…”

Section: Introductionmentioning

confidence: 99%

The State of Patient-Reported Outcome Measures for Pediatric Patients with Inherited Retinal Disease

et al. 2022

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Patient-reported outcome measures (PROMs) are questionnaires that assess health outcomes meaningful to the patient. PROMs have multiple applications, such as supporting clinicians’ decision-making for patient care, understanding the impact of disease on patient functioning, and evaluating the efficacy of therapeutics. Though PROMs were developed for various eye conditions, no PROM was tailored to pediatric patients with inherited retinal disease (IRD). Hence, a literature search was conducted using MEDLINE and Embase to identify PROMs potentially relevant to this patient population. This review evaluated selected pediatric PROMs against the US Food and Drug Administration (FDA) guidelines and found restricted use in the context of IRD. As there is a need for PROMs tailored to pediatric patients with IRD, we provide a perspective on applying the International Society for Pharmacoeconomics and Outcomes Research and FDA standards on the development of PROMs specific to IRD. Supplementary Information The online version contains supplementary material available at 10.1007/s40123-022-00514-x.

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“…120 Recently, the Michigan Retinal Degeneration Questionnaire was also validated as a PRO for patients with IRD, employing 59 items in 7 domains. 121 Whilst there remains no consensus on the most appropriate PRO tools in IRD and whether they need to be disease/genotype specific given the extreme clinical heterogeneity of IRD, they provide clinically meaningful information for both patients and researchers and are an integral assessment to fully evaluate treatment efficacy and calculate costeffectiveness.…”

Section: Patient-reported Outcome Measuresmentioning

confidence: 99%

Functional evaluation in inherited retinal disease

et al. 2021

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Functional assessments are a fundamental part of the clinical evaluation of patients with inherited retinal diseases (IRDs). Their importance and impact have become increasingly notable, given the significant breadth and number of clinical trials and studies investigating multiple avenues of intervention across a wide range of IRDs, including gene, pharmacological and cellular therapies. Moreover, the fact that many clinical trials are reporting improvements in vision, rather than the previously anticipated structural stability/slowing of degeneration, makes functional evaluation of primary relevance. In this review, we will describe a range of methods employed to characterise retinal function and functional vision, beginning with tests variably included in the clinic, such as visual acuity, electrophysiological assessment and colour discrimination, and then discussing assessments often reserved for clinical trials/research studies such as photoaversion testing, full-field static perimetry and microperimetry, and vision-guided mobility testing; addressing perimetry in greatest detail, given it is commonly a primary outcome metric. We will focus on how these tests can help diagnose and monitor particular genotypes, also noting their limitations/challenges and exploring analytical methodologies for better exploiting functional measurements, as well as how they facilitate patient inclusion and stratification in clinical trials and serve as outcome measures.

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

Cited by 38 publications

References 23 publications

Effects of duration and number of symptoms on vision-related anxiety in patients with Inherited Retinal Diseases

Effects of duration and number of symptoms on vision-related anxiety in patients with Inherited Retinal Diseases

The State of Patient-Reported Outcome Measures for Pediatric Patients with Inherited Retinal Disease

Functional evaluation in inherited retinal disease

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