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000259692 1001_ $$0P:(DE-2719)9002138$$aBuchholz, Maresa$$b0$$eFirst author$$udzne
000259692 245__ $$aPatient-reported, health economic and psychosocial outcomes in patients with Friedreich ataxia (PROFA): protocol of an observational study using momentary data assessments via mobile health app.
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000259692 520__ $$aFriedreich ataxia (FA) is the most common hereditary ataxia in Europe, characterised by progressively worsening movement and speech impairments with a typical onset before the age of 25 years. The symptoms affect the patients' health-related quality of life (HRQoL) and psychosocial health. FA leads to an increasing need for care, associated with an economic burden. Little is known about the impact of FA on daily lives and HRQoL. To fill that gap, we will assess patient-reported, psychosocial and economic outcomes using momentary data assessment via a mobile health application (app).The PROFA Study is a prospective observational study. Patients with FA (n=200) will be recruited at six European study centres (Germany, France and Austria). We will interview patients at baseline in the study centre and subsequently assess the patients' health at home via mobile health app. Patients will self-report ataxia severity, HRQoL, speech and hearing disabilities, coping strategies and well-being, health services usage, adverse health events and productivity losses due to informal care on a daily to monthly basis on the app for 6 months. Our study aims to (1) validate measurements of HRQoL and psychosocial health, (2) assess the usability of the mobile health app, and (3) use descriptive and multivariate statistics to analyse patient-reported and economic outcomes and the interaction effects between these outcomes. Insights into the app's usability could be used for future studies using momentary data assessments to measure outcomes of patients with FA.Ethical approval has been obtained from the Ethics Committee of the University Medicine of Greifswald, (BB096/22a, 26 October 2022) and from all local ethics committees of the participating study sites. Findings of the study will be published in peer-reviewed journals, presented at relevant international/national congresses and disseminated to German and French Patient Advocacy Organizations.ClinicalTrials.gov Registry (NCT05943002); Pre-results.
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000259692 650_7 $$2Other$$aFriedreich ataxia
000259692 650_7 $$2Other$$aRare diseases
000259692 650_7 $$2Other$$ahealth and informal care
000259692 650_7 $$2Other$$ahealth economics
000259692 650_7 $$2Other$$am-health app assessment
000259692 650_7 $$2Other$$apatient-reported outcomes
000259692 650_7 $$2Other$$aspeech and hearing disabilities
000259692 650_2 $$2MeSH$$aHumans
000259692 650_2 $$2MeSH$$aAdult
000259692 650_2 $$2MeSH$$aFriedreich Ataxia
000259692 650_2 $$2MeSH$$aQuality of Life
000259692 650_2 $$2MeSH$$aMobile Applications
000259692 650_2 $$2MeSH$$aTelemedicine: methods
000259692 650_2 $$2MeSH$$aPatient Reported Outcome Measures
000259692 650_2 $$2MeSH$$aObservational Studies as Topic
000259692 7001_ $$0P:(DE-2719)9001915$$aWeber, Niklas$$b1$$udzne
000259692 7001_ $$aBorel, Stephanie$$b2
000259692 7001_ $$aSayah, Sabrina$$b3
000259692 7001_ $$00000-0003-3454-6266$$aXie, Feng$$b4
000259692 7001_ $$aSchulz, Jörg B$$b5
000259692 7001_ $$aReetz, Kathrin$$b6
000259692 7001_ $$aBoesch, Sylvia$$b7
000259692 7001_ $$0P:(DE-2719)2810704$$aKlopstock, Thomas$$b8$$udzne
000259692 7001_ $$aKarin, Ivan$$b9
000259692 7001_ $$0P:(DE-2719)2810795$$aSchöls, Ludger$$b10$$udzne
000259692 7001_ $$0P:(DE-2719)9001510$$aGrobe-Einsler, Marcus$$b11$$udzne
000259692 7001_ $$0P:(DE-2719)2810314$$aKlockgether, Thomas$$b12$$udzne
000259692 7001_ $$aDavies, Elin Haf$$b13
000259692 7001_ $$aSchmeder, Madeleine$$b14
000259692 7001_ $$aNadke, Andreas$$b15
000259692 7001_ $$0P:(DE-2719)2810763$$aMichalowsky, Bernhard$$b16$$eLast author$$udzne
000259692 773__ $$0PERI:(DE-600)2599832-8$$a10.1136/bmjopen-2023-075736$$gVol. 13, no. 8, p. e075736 -$$n8$$pe075736$$tBMJ open$$v13$$x2044-6055$$y2023
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