Understanding the relationship between the 32-item motor function measure and daily activities from an individual with spinal muscular atrophy and their caregivers’ perspective: a two-part study

BMC Neurology

Sharan Randhawa¹  Rob Arbuckle¹  Jessica Flynn¹  Angela De Martini²  Lida Yang²  Sonia Reif²  Rosangel Cruz³  Jill Jarecki³  Tina Duong⁴  Aurelie Barriere⁵  Carole Vuillerot⁶  Johannes Reithinger⁷  Fani Petridis⁷  Hannah Staunton⁸  Jessica Braid⁸  Mencia De Lemus⁹  Ria Broekgaarden^1,10  Nicole Gusset^1,11 

[1] Adelphi Values, Patient-Centered Outcomes, Adelphi Mill, Bollington, Cheshire, UK;Charles River Associates Inc, Zurich, Switzerland;CureSMA, Elk Grove Village, IL, USA;Department of Neurology and Neurological Sciences, Stanford University, Stanford, CA, USA;Department of Pediatric Physical Medicine and Rehabilitation, Hôpital Mère Enfant, CHU-Lyon, Lyon University, Lyon, France;Department of Pediatric Physical Medicine and Rehabilitation, Hôpital Mère Enfant, CHU-Lyon, Lyon University, Lyon, France;Neuromyogen Institute, CNRS UMR 5310 – INSERM U1217, Université de Lyon, Lyon, France;F. Hoffmann-La Roche Ltd, Basel, Switzerland;Roche Products Limited, Welwyn Garden City, UK;SMA Europe Freiburg, Freiburg, Germany;FundAME, Madrid, Spain;SMA Europe Freiburg, Freiburg, Germany;SMA Europe and Vereniging Spierziekten Nederland, Baarn, The Netherlands;SMA Europe Freiburg, Freiburg, Germany;SMA Schweiz, Swiss Patient Organisation for Spinal Muscular Atrophy, Heimberg, Switzerland;

关键词: Spinal muscular atrophy;    32-item motor function measure;    Content validity;    Qualitative interviews;    Quantitative online survey;    Clinical meaningfulness;    Patient relevance;   

DOI  :  10.1186/s12883-021-02166-z

来源: Springer

【 摘 要 】

BackgroundThe 32-item Motor Function Measure (MFM32) is a clinician-reported outcome measure used to assess the functional abilities of individuals with neuromuscular diseases, including those with spinal muscular atrophy (SMA). This two-part study explored the relationship between the functional abilities assessed in the MFM32 and activities of daily living (ADLs) from the perspective of individuals with Type 2 and Type 3 (non-ambulant and ambulant) SMA and their caregivers through qualitative interviews and a quantitative online survey.MethodsIn-depth, semi-structured, qualitative interviews were conducted with individuals with SMA and caregivers from the US. Subsequently, a quantitative online survey was completed by individuals with SMA or their caregivers from France, Germany, Italy, Poland, Spain, Canada, the United States (US) and the UK. In both parts of the study, participants were asked to describe the ADLs considered to be related to the functional abilities assessed in the MFM32. Results from the qualitative interviews informed the content of the quantitative online survey.ResultsQualitative interviews were conducted with 15 adult participants, and 217 participants completed the quantitative online survey. From the qualitative interviews, all of the functional abilities assessed in the patient-friendly MFM32 were deemed as related to one or more ADL. The specific ADLs that participants considered related to the patient-friendly MFM32 items could be grouped into 10 key ADL domains: dressing, mobility/transferring, self-care, self-feeding, reaching, picking up and holding objects, physical activity, writing and technology use, social contact/engagement, toileting and performing work/school activities. These results were confirmed by the quantitative online survey whereby the ADLs reported to be related to each patient-friendly MFM32 item were consistent and could be grouped into the same 10 ADL domains.ConclusionThis study provides in-depth evidence from the patient/caregiver perspective supporting the relevance of the patient-friendly MFM32 items to the ADLs of individuals with Type 2 and Type 3 SMA.

【 授权许可】

CC BY