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dc.contributor.authorPera, Maria C
dc.contributor.authorCoratti, Giorgia
dc.contributor.authorForcina, Nicola
dc.contributor.authorMazzone, Elena S
dc.contributor.authorScoto, Mariacristina
dc.contributor.authorMontes, Jacqueline
dc.contributor.authorPasternak, Amy
dc.contributor.authorMayhew, Anna
dc.contributor.authorMessina, Sonia
dc.contributor.authorSframeli, Maria
dc.contributor.authorMain, Marion
dc.contributor.authorLofra, Robert Muni
dc.contributor.authorDuong, Tina
dc.contributor.authorRamsey, Danielle
dc.contributor.authorDunaway, Sally
dc.contributor.authorSalazar, Rachel
dc.contributor.authorFanelli, Lavinia
dc.contributor.authorCivitello, Matthew
dc.contributor.authorde Sanctis, Roberto
dc.contributor.authorAntonaci, Laura
dc.contributor.authorLapenta, Leonardo
dc.contributor.authorLucibello, Simona
dc.contributor.authorPane, Marika
dc.contributor.authorDay, John
dc.contributor.authorDarras, Basil T
dc.contributor.authorDe Vivo, Darryl C
dc.contributor.authorMuntoni, Francesco
dc.contributor.authorFinkel, Richard
dc.contributor.authorMercuri, Eugenio
dc.date.accessioned2018-02-06T17:29:01Z
dc.date.available2018-02-06T17:29:01Z
dc.date.issued2017-02-23
dc.identifier.citationPera , M C , Coratti , G , Forcina , N , Mazzone , E S , Scoto , M , Montes , J , Pasternak , A , Mayhew , A , Messina , S , Sframeli , M , Main , M , Lofra , R M , Duong , T , Ramsey , D , Dunaway , S , Salazar , R , Fanelli , L , Civitello , M , de Sanctis , R , Antonaci , L , Lapenta , L , Lucibello , S , Pane , M , Day , J , Darras , B T , De Vivo , D C , Muntoni , F , Finkel , R & Mercuri , E 2017 , ' Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy ' , BMC Neurology , vol. 17 , no. 1 , pp. 39 . https://doi.org/10.1186/s12883-017-0790-9
dc.identifier.issn1471-2377
dc.identifier.otherPURE: 11264932
dc.identifier.otherPURE UUID: 25359eac-4b53-4935-8db8-af5c3b3d6174
dc.identifier.otherPubMed: 28231823
dc.identifier.otherPubMedCentral: PMC5324197
dc.identifier.otherScopus: 85013743931
dc.identifier.otherORCID: /0000-0003-1275-2640/work/32942873
dc.identifier.urihttp://hdl.handle.net/2299/19732
dc.description© The Author(s). 2017 Open Access This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated
dc.description.abstractBACKGROUND: Reports on the clinical meaningfulness of outcome measures in spinal muscular atrophy (SMA) are rare. In this two-part study, our aim was to explore patients' and caregivers' views on the clinical relevance of the Hammersmith Functional Motor Scale Expanded- (HFMSE). METHODS: First, we used focus groups including SMA patients and caregivers to explore their views on the clinical relevance of the individual activities included in the HFMSE. Then we asked caregivers to comment on the clinical relevance of possible changes of HFMSE scores over time. As functional data of individual patients were available, some of the questions were tailored according to their functional level on the HFMSE. RESULTS: Part 1: Sixty-three individuals participated in the focus groups. This included 30 caregivers, 25 patients and 8 professionals who facilitated the discussion. The caregivers provided a comparison to activities of daily living for each of the HFMSE items. Part 2: One hundred and forty-nine caregivers agreed to complete the questionnaire: in response to a general question, 72% of the caregivers would consider taking part in a clinical trial if the treatment was expected to slow down deterioration, 88% if it would stop deterioration and 97% if the treatment was expected to produce an improvement. Caregivers were informed of the first three items that their child could not achieve on the HFMSE. In response 75% indicated a willingness to take part in a clinical trial if they could achieve at least one of these abilities, 89% if they could achieve two, and 100% if they could achieve more than 2. CONCLUSIONS: Our findings support the use of the HFMSE as a key outcome measure in SMA clinical trials because the individual items and the detected changes have clear content validity and clinical meaningfulness for patients and their caregivers.en
dc.format.extent10
dc.language.isoeng
dc.relation.ispartofBMC Neurology
dc.subjectspinal muscular atrophy
dc.subjectquality of life
dc.subjectcarers
dc.subjectclinical trials
dc.titleContent validity and clinical meaningfulness of the HFMSE in spinal muscular atrophyen
dc.contributor.institutionSchool of Health and Social Work
dc.description.statusPeer reviewed
rioxxterms.versionVoR
rioxxterms.versionofrecordhttps://doi.org/10.1186/s12883-017-0790-9
rioxxterms.typeJournal Article/Review
herts.preservation.rarelyaccessedtrue


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