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dc.contributor.authorRamsey, Danielle
dc.contributor.authorScoto, Mariacristina
dc.contributor.authorMayhew, Anna
dc.contributor.authorMain, Marion
dc.contributor.authorMazzone, Elena S
dc.contributor.authorMontes, Jacqueline
dc.contributor.authorde Sanctis, Roberto
dc.contributor.authorDunaway Young, Sally
dc.contributor.authorSalazar, Rachel
dc.contributor.authorGlanzman, Allan M
dc.contributor.authorPasternak, Amy
dc.contributor.authorQuigley, Janet
dc.contributor.authorMirek, Elizabeth
dc.contributor.authorDuong, Tina
dc.contributor.authorGee, Richard
dc.contributor.authorCivitello, Matthew
dc.contributor.authorTennekoon, Gihan
dc.contributor.authorPane, Marika
dc.contributor.authorPera, Maria Carmela
dc.contributor.authorBushby, Kate
dc.contributor.authorDay, John
dc.contributor.authorDarras, Basil T
dc.contributor.authorDe Vivo, Darryl
dc.contributor.authorFinkel, Richard
dc.contributor.authorMercuri, Eugenio
dc.contributor.authorMuntoni, Francesco
dc.date.accessioned2018-02-06T17:28:45Z
dc.date.available2018-02-06T17:28:45Z
dc.date.issued2017-02-21
dc.identifier.citationRamsey , D , Scoto , M , Mayhew , A , Main , M , Mazzone , E S , Montes , J , de Sanctis , R , Dunaway Young , S , Salazar , R , Glanzman , A M , Pasternak , A , Quigley , J , Mirek , E , Duong , T , Gee , R , Civitello , M , Tennekoon , G , Pane , M , Pera , M C , Bushby , K , Day , J , Darras , B T , De Vivo , D , Finkel , R , Mercuri , E & Muntoni , F 2017 , ' Revised Hammersmith Scale for Spinal Muscular Atrophy: A SMA specific clinical outcome assessment tool ' , PLoS ONE , vol. 12 , no. 2 , e0172346 . https://doi.org/10.1371/journal.pone.0172346
dc.identifier.issn1932-6203
dc.identifier.otherPURE: 11213354
dc.identifier.otherPURE UUID: 0d794fde-d354-4281-ba94-ecd868412730
dc.identifier.otherPubMed: 28222119
dc.identifier.otherScopus: 85013487380
dc.identifier.otherORCID: /0000-0003-1275-2640/work/32942874
dc.identifier.urihttp://hdl.handle.net/2299/19730
dc.descriptionThis is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
dc.description.abstractRecent translational research developments in Spinal Muscular Atrophy (SMA), outcome measure design and demands from regulatory authorities require that clinical outcome assessments are 'fit for purpose'. An international collaboration (SMA REACH UK, Italian SMA Network and PNCRN USA) undertook an iterative process to address discontinuity in the recorded performance of the Hammersmith Functional Motor Scale Expanded and developed a revised functional scale using Rasch analysis, traditional psychometric techniques and the application of clinical sensibility via expert panels. Specifically, we intended to develop a psychometrically and clinically robust functional clinician rated outcome measure to assess physical abilities in weak SMA type 2 through to strong ambulant SMA type 3 patients. The final scale, the Revised Hammersmith Scale (RHS) for SMA, consisting of 36 items and two timed tests, was piloted in 138 patients with type 2 and 3 SMA in an observational cross-sectional multi-centre study across the three national networks. Rasch analysis demonstrated very good fit of all 36 items to the construct of motor performance, good reliability with a high Person Separation Index PSI 0.98, logical and hierarchical scoring in 27/36 items and excellent targeting with minimal ceiling. The RHS differentiated between clinically different groups: SMA type, World Health Organisation (WHO) categories, ambulatory status, and SMA type combined with ambulatory status (all p < 0.001). Construct and concurrent validity was also confirmed with a strong significant positive correlation with the WHO motor milestones rs = 0.860, p < 0.001. We conclude that the RHS is a psychometrically sound and versatile clinical outcome assessment to test the broad range of physical abilities of patients with type 2 and 3 SMA. Further longitudinal testing of the scale with regards change in scores over 6 and 12 months are required prior to its adoption in clinical trials.en
dc.format.extent19
dc.language.isoeng
dc.relation.ispartofPLoS ONE
dc.rightsOpen
dc.titleRevised Hammersmith Scale for Spinal Muscular Atrophy: : A SMA specific clinical outcome assessment toolen
dc.contributor.institutionSchool of Health and Social Work
dc.description.statusPeer reviewed
dc.relation.schoolSchool of Health and Social Work
dc.description.versiontypeFinal Published version
dcterms.dateAccepted2017-02-21
rioxxterms.versionVoR
rioxxterms.versionofrecordhttps://doi.org/10.1371/journal.pone.0172346
rioxxterms.licenseref.urihttp://creativecommons.org/licenses/by/4.0/
rioxxterms.typeJournal Article/Review
herts.preservation.rarelyaccessedtrue
herts.rights.accesstypeOpen


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