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

Maria Carmela Pera, Giorgia Coratti, Nicola Forcina, Elena Stacy Mazzone, Mariacristina Scoto, Jacqueline Montes, Amy Pasternak, Anna Mayhew, Sonia Messina, Maria Sframeli, Marion Main, Robert Muni Lofra, Tina Duong, Danielle Ramsey, Sally Dunaway, Rachel Salazar, Lavinia Fanelli, Matthew Civitello, Roberto De Sanctis, Laura AntonaciLeonardo Lapenta, Simona Lucibello, Marika Pane, John Day, Basil T. Darras, Darryl C. De Vivo, Francesco Muntoni, Richard Finkel, Eugenio Maria Mercuri

Risultato della ricerca: Contributo in rivistaArticolo in rivista

40 Citazioni (Scopus)


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.
Lingua originaleEnglish
pagine (da-a)39-39
Numero di pagine1
RivistaBMC Neurology
Stato di pubblicazionePubblicato - 2017


  • Activities of Daily Living
  • Adolescent
  • Adult
  • Caregivers
  • Carers
  • Child
  • Clinical trials
  • Female
  • Focus Groups
  • Humans
  • Male
  • Muscular Atrophy, Spinal
  • Neurology (clinical)
  • Outcome Assessment (Health Care)
  • Patients
  • Quality of life
  • Severity of Illness Index
  • Spinal Muscular Atrophies of Childhood
  • Spinal muscular atrophy
  • Young Adult


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