Evidence map›Paper›PMID 34158306›Full record

ArticleBMJ open2021

Perspectives of people with inherited retinal diseases on ocular gene therapy in Australia: protocol for a national survey.

Heather G Mack, Fred K Chen, John Grigg, Robyn Jamieson, John De Roach, Fleur O'Hare, Alexis Ceecee Britten-Jones, Myra McGuinness, Nicole Tindill, Lauren Ayton and 1 more

Open access · goldAbstract readClinical Trial Protocol
PubMed Full text at PMC Publisher
In one paragraph

Article in BMJ open, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
0.7field-weighted citation impact, top 31% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

8 citing papers in PubMed, 9 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. Article
  5. Article
  6. Review
  7. Will our cardiomyopathy patients accept gene therapy?Netherlands heart journal : monthly journal of the Netherlands Society of Cardiology and the Netherlands Heart Foundation · 2022
    Review
  8. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

11 authors at 4 institutions in 1 country.

Heather G MackCentre for Eye Research Australia Ltd, The University of Melbourne, East Melbourne, Victoria, Australia hmack@eyesurgery.com.au.ORCID 0000-0001-9756-1098
Fred K ChenCentre for Ophthalmology and Visual Science, Lions Eye Institute, University of Western Australia, Nedlands, Western Australia, Australia.
John GriggDepartment of Ophthalmology, The University of Sydney, Sydney, New South Wales, Australia.
Robyn JamiesonChildren's Medical Research Institute, University of Sydney, Sydney, New South Wales, Australia.
John De RoachThe Australian Inherited Retinal Disease Registry and DNA Bank, Perth, Western Australia, Australia.
Fleur O'HareCentre for Eye Research Australia Ltd, The University of Melbourne, East Melbourne, Victoria, Australia.
Alexis Ceecee Britten-JonesCentre for Eye Research Australia Ltd, The University of Melbourne, East Melbourne, Victoria, Australia.ORCID 0000-0002-1101-2870
Myra McGuinnessCentre for Eye Research Australia Ltd, The University of Melbourne, East Melbourne, Victoria, Australia.ORCID 0000-0002-5422-040X
Nicole TindillRoyal Victorian Eye and Ear Hospital, East Melbourne, Victoria, Australia.
Lauren AytonCentre for Eye Research Australia Ltd, The University of Melbourne, East Melbourne, Victoria, Australia.
Australian Ocular Gene Therapy Group
The University of Melbourne · AUThe University of Sydney · AULions Eye Institute · AUThe Royal Victorian Eye & Ear Hospital · AU

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionVoretigene neparvovec-rzyl (Luxturna) was approved by the Australian Therapeutic Goods Administration on 4 August 2020 for the treatment of biallelic mutations in the METHODS AND ANALYSIS: A new 'Attitudes to Gene Therapy for the Eye' tool will be developed following consultation with people with inherited retinal disease (IRD) and content matter experts. Australians with IRD or their guardians will be asked to complete an internet-based survey comprising existing quality of life and visual function instruments and items for the newly proposed tool. We expect to recruit 500 survey participants from patient support groups, the practices of Australian ophthalmologists who are specialists in IRD and Australian ophthalmic research institutions. Launch is anticipated early 2021. Responses will be analysed using item response theory methodology. ETHICS AND DISSEMINATION: This study has received ethics approval from the University of Melbourne (#2057534). The results of the study will be published in a peer-reviewed journal and will be presented at relevant conferences. Organisations involved in recruitment, and the Patient Engagement Advisory committee will assist the research team with dissemination of the study outcomes.

Indexed as

Leber Congenital AmaurosisRetinal DiseasesAdultAustraliaGenetic TherapyHumansQuality of Lifegeneticsmedical retinavitreoretinal

Identifiers

PMID34158306
PMCPMC8220456
OpenAlexW3175609343

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.