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

卷:21

Jessica Flynn¹  Sharan Randhawa¹  Rob Arbuckle¹  Angela De Martini²  Lida Yang²  Sonia Reif²  Jill Jarecki³  Rosangel Cruz³  Tina Duong⁴  Carole Vuillerot⁵  Aurelie Barriere⁵  Johannes Reithinger⁶  Fani Petridis⁶  Jessica Braid⁷  Hannah Staunton⁷  Mencia De Lemus⁸  Nicole Gusset⁸  Ria Broekgaarden⁸ 

[1] Adelphi Values, Patient-Centered Outcomes;

[2] Charles River Associates Inc;

[3] CureSMA;

[4] Department of Neurology and Neurological Sciences, Stanford University;

[5] Department of Pediatric Physical Medicine and Rehabilitation, Hôpital Mère Enfant, CHU-Lyon, Lyon University;

[6] F. Hoffmann-La Roche Ltd;

[7] Roche Products Limited;

[8] SMA Europe Freiburg;

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

DOI  :  10.1186/s12883-021-02166-z

来源: DOAJ

【 摘 要 】

Abstract Background The 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. Methods In-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. Results Qualitative 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. Conclusion This 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】

Unknown