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

Autor: Pera MC; Pediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy., Coratti G; Pediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy., Forcina N; Pediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy., Mazzone ES; Pediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy., Scoto M; Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, UK., Montes J; Department of Neurology, Columbia University Medical Center, New York, NY, USA., Pasternak A; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., Mayhew A; John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle, UK., Messina S; Department of Clinical and Experimental Medicine and Nemo Sud Clinical Center, University of Messina, Messina, Italy., Sframeli M; Department of Clinical and Experimental Medicine and Nemo Sud Clinical Center, University of Messina, Messina, Italy., Main M; Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, UK., Lofra RM; John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, Newcastle, UK., Duong T; Stanford University, Palo Alto, CA, USA., Ramsey D; Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, UK., Dunaway S; Department of Neurology, Columbia University Medical Center, New York, NY, USA., Salazar R; Department of Neurology, Columbia University Medical Center, New York, NY, USA., Fanelli L; Pediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy., Civitello M; Nemours Children's Hospital, University of Central Florida College of Medicine, Orlando, USA., de Sanctis R; Pediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy., Antonaci L; Pediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy., Lapenta L; Pediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy., Lucibello S; Pediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy., Pane M; Pediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy., Day J; Stanford University, Palo Alto, CA, USA., Darras BT; Department of Neurology, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., De Vivo DC; Department of Neurology, Columbia University Medical Center, New York, NY, USA., Muntoni F; Dubowitz Neuromuscular Centre, UCL Institute of Child Health & Great Ormond Street Hospital, London, UK., Finkel R; Nemours Children's Hospital, University of Central Florida College of Medicine, Orlando, USA., Mercuri E; Pediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy. eumercuri@gmail.com.
Jazyk: angličtina
Zdroj: BMC neurology [BMC Neurol] 2017 Feb 23; Vol. 17 (1), pp. 39. Date of Electronic Publication: 2017 Feb 23.
DOI: 10.1186/s12883-017-0790-9
Abstrakt: 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.
Databáze: MEDLINE
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