Qualitative interviews to determine movement task selection of patients with Limb-girdle muscular dystrophy


Pre-Clinical Research

Poster Number: 19


Mindy Leffler MEd, Kristina Davis PhD, Marielle Contesse PhD, Alyssa Burns PhD


1. Casimir, 2. Casimir, 4. Casimir

Currently, there are no clinician-rated assessments of movement compensation that capture finer, functional changes of Limb-girdle muscular dystrophies (LGMD) disease progression. The Duchenne Video Assessment (DVA) is a standardized, at-home video capture tool, used to quantify quality of movement as an indication of disease severity and progression. The purpose of this study was to adapt the DVA and create the Limb-girdle video assessment (LGVA), by identifying LGMD movement tasks that may show clinically relevant changes in compensatory movements.

LGMD expert physical therapists (PTs; n=5) were recruited to participate in qualitative interviews to discuss 1) their experiences with LGMD, 2) spontaneous movement task suggestions that may be sensitive to change and 3) applicability of DVA tasks to LGMD.

PTs expressed that current clinic outcome measures may not detect patient reported changes (i.e, fatigue, muscle pain, movement difficulty). All 5 PTs spontaneously listed: climbing stairs, standing up from the floor, standing up from a chair, eating and dressing (t-shirt on and off) as key movement tasks to evaluate treatment benefit and disease progression. After review of DVA videos PTs confirmed the DVA movement tasks were relevant to capture in the LGMD population. Functional subgroup branching based on ambulation was recommended to determine the relevance of the task and the sensitivity to change in the short term. The three groups are mild: ambulatory and can still stand from floor independently, moderate: ambulatory and cannot stand from floor independently and severe; non-ambulatory.

PTs suggest the LGVA may be an effective tool in evaluating compensatory movement patterns and detect clinically meaningful changes in LGMD disease progression and confirmed the subset of tasks from the DVA relevant to LGMD patients. Future research is needed to assess the LGMD-specific compensatory patterns.