Evidence map›Paper›PMID 40299280›Full record

ArticleAdvances in therapy2025

The Impacts of Caregiving for Patients with X-Linked Retinitis Pigmentosa (XLRP): Findings from the EXPLORE XLRP-2 Study.

Michel Weber, Francesco Parmeggiani, Dominique Bremond-Gignac, Avril Daly, Marjolein Lahaye, Andrew Lotery, Nabin Paudel, Markus Ritter, Enrique Rodríguez de la Rúa, Ygal Rotenstreich and 5 more

Abstract readMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Article in Advances in therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
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

1 citing paper in PubMed.

  1. 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

15 authors.

Michel Weber *Ophthalmology Department, University Hospital Centre (CHU) de Nantes, Nantes, France.
Francesco Parmeggiani *Department of Translational Medicine, University of Ferrara, Ferrara, Italy.
Dominique Bremond-GignacOphthalmology Department, Necker Enfants Malades University Hospital, AP-HP, Paris Cité University, Paris, France.
Avril DalyRetina International, Dublin, Ireland.
Marjolein LahayeJanssen-Cilag B.V., Breda, the Netherlands.
Andrew LoteryFaculty of Medicine, University of Southampton, Southampton, UK.
Nabin PaudelRetina International, Dublin, Ireland.
Markus RitterDepartment of Ophthalmology, Medical University of Vienna, Vienna, Austria.
Enrique Rodríguez de la RúaDepartment of Ophthalmology, University Hospital Virgen Macarena, Seville, Spain.
Ygal RotenstreichThe Goldschleger Eye Institute, Sheba Medical Center, Tel Hashomer, Israel.
Eeva-Marja SankilaDepartment of Ophthalmology, University of Helsinki and Helsinki University Hospital, Helsinki, Finland.
Katarina StinglCenter for Ophthalmology, University Eye Hospital, University of Tübingen, Tübingen, Germany.
Jacqueline Van DenderenJanssen-Cilag B.V., Breda, the Netherlands.
Tom DeneeJanssen-Cilag B.V., Breda, the Netherlands.
Katalin PungorJanssen-Cilag GmbH, Neuss, Germany. kpungor@its.jnj.com.ORCID http://orcid.org/0000-0003-4679-2050

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionInformal caregivers play an important part in the healthcare of patients with chronic diseases, including those leading to visual impairment. X-linked retinitis pigmentosa (XLRP) is considered one of the most severe forms of retinitis pigmentosa and causes declines in vision starting in childhood, ultimately progressing to legal blindness in adulthood. Caregivers are expected to play an increasing role in patient care, but real-world impacts of XLRP on caregivers are poorly evaluated.

methodsEXPLORE XLRP-2 was an exploratory, multicentre, non-interventional study. Cross-sectional surveys were used to gather experiences directly from caregivers across Europe and Israel by both validated and newly developed caregiver-reported outcome surveys.

resultsSeventy caregivers of patients with XLRP associated with retinitis pigmentosa GTPase regulator (RPGR) mutations were enrolled, of whom 68 were included in analyses; 87.7% of caregivers were female and mean (standard deviation [SD]) age was 49.4 (11.7) years. They were most commonly either spouses (50.8%) or parents (41.5%) of patients. Caregivers spent a mean (SD) 28.7 (34.5) hours per week caring for patients. Of the 72.3% of caregivers who were employed, 34.8% worked part time; of the 27.7% of caregivers who were unemployed, 33.3% cited caregiving responsibilities as a cause for unemployment; 23.1% and 46.2% of caregivers reported any level of depression and anxiety, respectively, with few additional impacts captured by the surveys.

conclusionsSome caregivers reported employment and mental health impacts in this study. However, despite many hours spent per week caring for patients with XLRP, the surveys did not reflect the expected burden experienced by caregivers, highlighting the need for further research in this field.

Indexed as

CaregiversGenetic Diseases, X-LinkedRetinitis PigmentosaAdultAgedCross-Sectional StudiesEuropeEye ProteinsFemaleHumansIsraelMaleMiddle AgedQuality of LifeEye ProteinsRPGR protein, humanBurdenCaregiverImpactInherited retinal dystrophyReal-worldSurveyX-linked retinitis pigmentosa

Identifiers

PMID40299280
PMCPMC12085331

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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.