Researchers explored the theme of meaningful change through the 32-item Motor Function Measure (MFM32) and advocated its importance in the lives of patients with spinal muscular atrophy (SMA) type 2 and 3, as published in Frontiers in Neurology.
SMA is divided into 4 subtypes, from the most severe to the least. The wide range of clinical presentations and speed of muscular deterioration have prompted scientists to produce a number of clinical outcome assessments to more accurately capture what a patient with SMA is uniquely experiencing.
“The MFM32 is validated for use in individuals with neuromuscular disorders, including those with Types 2 and 3 SMA and has been found to be better targeted to weaker patients with a more progressed disease,” Duong and colleagues wrote.
Read more about SMA etiology
The MFM32 has been the subject of multiple studies itself and has been praised for its validity, reliability, and responsiveness. However, Duong and colleagues felt there was room for the theme of meaningful change to be reflected more prominently in the scale, which emphasizes differences between the experience of individual patients instead of just between different categories of patients.
They hence set out to evaluate patient-centered meaningful change at the individual level using the MFM32. They accomplished this by conducting qualitative semistructured interviews that were supplemented by an online survey, as well as conducting an independent analysis of clinical trial data to estimate meaningful change thresholds. In their efforts, the research team focused on SMA type 2 and 3.
Based on patient and caregiver feedback, Duong and colleagues discovered that maintaining functional ability was an important outcome for patients with SMA type 2 and 3. The quantitative analyses performed demonstrated that an approximate 3 point improvement in the MFM32 total score represented a meaningful change for patients with SMA at an individual level.
“Overall, the qualitative and quantitative findings from this study support the importance of examining a range of meaningful change thresholds on the MFM32 including ≥0 points change reflecting stabilization or improvement and ≥3 points change reflecting a higher threshold of improvement,” Duong et al concluded.
Duong T, Staunton H, Braid J, et al. A patient-centered evaluation of meaningful change on the 32-item Motor Function Measure in spinal muscular atrophy using qualitative and quantitative data. Front Neurol. Published online January 17, 2022. doi:10.3389/fneur.2021.770423