BACKGROUND: Reports on the clinical meaningfulness of outcome measures in @entity2057 ( @entity2057 ) are rare. In this two-part study, our aim was to explore @entity1 ' and caregivers' views on the clinical relevance of the Hammersmith Functional Motor Scale Expanded- (HFMSE). METHODS: First, we used focus groups including @entity2057 @entity1 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 @entity1 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 @entity1 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 @entity1 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 @entity2057 clinical trials because the individual items and the detected changes have clear content validity and clinical meaningfulness for @entity1 and their caregivers.

Content validity and clinical meaningfulness of the HFMSE in XXXX .

@entity1 :: ('9606', 'Species') :: ['child', 'patients'] @entity2057 :: ('MESH:D009133', 'Disease') :: ['SMA', 'spinal muscular atrophy']
@entity2057 :: (MESH:D009133,Disease) :: ['spinal muscular atrophy']
