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        Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy

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        Author
        Pera, Maria C
        Coratti, Giorgia
        Forcina, Nicola
        Mazzone, Elena S
        Scoto, Mariacristina
        Montes, Jacqueline
        Pasternak, Amy
        Mayhew, Anna
        Messina, Sonia
        Sframeli, Maria
        Main, Marion
        Lofra, Robert Muni
        Duong, Tina
        Ramsey, Danielle
        Dunaway, Sally
        Salazar, Rachel
        Fanelli, Lavinia
        Civitello, Matthew
        de Sanctis, Roberto
        Antonaci, Laura
        Lapenta, Leonardo
        Lucibello, Simona
        Pane, Marika
        Day, John
        Darras, Basil T
        De Vivo, Darryl C
        Muntoni, Francesco
        Finkel, Richard
        Mercuri, Eugenio
        Attention
        2299/19732
        Abstract
        BACKGROUND: 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.
        Publication date
        2017-02-23
        Published in
        BMC Neurology
        Published version
        https://doi.org/10.1186/s12883-017-0790-9
        License
        http://creativecommons.org/licenses/by/4.0/
        Other links
        http://hdl.handle.net/2299/19732
        Relations
        School of Health and Social Work
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