Staunton, Hannah;
Cleanthous, Sophie;
Teodoro, Vanda;
Barrett, Louise;
Braid, Jessica;
Ewens, Bethany;
Cano, Stefan;
... Mayhew, Anna; + view all
(2023)
A Mixed-method Approach to Develop an Ambulatory Module of the SMA Independence Scale.
Journal of Neuromuscular Diseases
, 10
(6)
pp. 1093-1109.
10.3233/JND-230096.
Preview |
Text
jnd_2023_10-6_jnd-10-6-jnd230096_jnd-10-jnd230096.pdf - Published Version Download (579kB) | Preview |
Abstract
Background: Limited qualitative data exist on the symptoms and impacts of spinal muscular atrophy (SMA) experienced by ambulant individuals. An ambulant module of the SMA Independence Scale (SMAIS) was developed to quantify the assistance required to perform everyday mobility-related activities. // Objective: The objective of this study was to develop a patient-centered module that provides key insights into what constitutes independence for ambulant and near-ambulant individuals with SMA. // Methods: A stepwise, mixed-method approach was used. Semi-structured interviews were conducted in three waves with individuals with SMA and caregivers of children with SMA who were ambulant or near-ambulant (can walk ≥5 steps with support). Wave 1 interviews (n = 20) focused on concept elicitation. Wave 2 and 3 interviews (n = 15, both) involved completion and cognitive debriefing of items generated based on Wave 1 interviews. Therapeutic area experts were consulted throughout all key steps of the study. In particular, feedback was provided for item refinement and response option decisions. A macro-level preliminary, exploratory analysis, using Rasch Measurement Theory (RMT), provided insight on measurement properties. // Results: Wave 1 resulted in 42 mobility and 11 instrumental activity of daily living (iADL) items. During Wave 2, participants defined independence as completing a task with supportive aids but without help from another person, leading to item refinement and modifications to the response scale. Lack of conceptual relevance and ceiling effects led to the removal of all iADL items after Wave 2, and 41 mobility items were tested in Wave 3. Final exploratory RMT and item refinement to reduce overlap led to a 27-item set related to mobility tasks. // Conclusions: Our study provides preliminary support for using the 27-item SMAIS–Ambulatory Module for ambulant or near-ambulant individuals with SMA. Larger-scale analyses to further assess the psychometric properties of the scale are warranted.
| Type: | Article |
|---|---|
| Title: | A Mixed-method Approach to Develop an Ambulatory Module of the SMA Independence Scale |
| Location: | Netherlands |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.3233/JND-230096 |
| Publisher version: | https://doi.org/10.3233/JND-230096 |
| Language: | English |
| Additional information: | Copyright © 2023 – The authors. Published by IOS Press. This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial License (CC BY-NC 4.0). |
| Keywords: | Spinal muscular atrophy, patient-reported outcome measures, psychometrics, activities of daily living |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10183817 |
Archive Staff Only
 |
View Item |
