Specialized orthotic care to improve functioning in adults with neuromuscular disorders: results of an open-label blinded end-point randomized controlled trial

BACKGROUND: People with neuromuscular disorders (NMD) are often provided with lower limb orthoses to improve walking. Guideline-based provision of lower limb orthoses in expert settings (i.e. specialized orthotic care) may improve treatment outcomes by better matching the orthotic properties to address walking problems in neuromuscular disorders (NMD). AIM: We assessed the (cost)-effectiveness of specialized orthotic care compared to usual orthotic care in adults with NMD. DESIGN: Randomized controlled trial with an economic evaluation. SETTING: Outpatient rehabilitation settings. POPULATION: Adults with plantar flexor and/or quadriceps weakness due to NMD with an indication for a lower limb orthosis. METHODS: Participants were randomly assigned to specialized orthotic care (N.=31), or usual orthotic care (N.=30). Primary endpoints were personal goal attainment (at 24 weeks follow-up), and walking energy cost at comfortable speed (change from baseline to 24 weeks follow-up). Secondary endpoints included comfortable walking speed, spatiotemporal gait parameters, gait biomechanics, stability, physical functioning, (fear of) falling, fatigue and satisfaction. RESULTS: Personal goal attainment was significantly higher for specialized orthotic care (P=0.011). Walking energy cost did not differ between groups (P=0.140) but decreased significantly with -11.8% (-0.55 J/kg/m; 95% CI: -0.99 to -0.11) following specialized, but not following usual orthotic care (-0.08 J/kg/m; 95% CI: -0.32 to 0.26). Secondarily, changes in step length, walking speed, physical functioning, and satisfaction were in favor of specialized orthotic care (P≤0.042). Specialized orthotic care was likely cost-effective from a societal and healthcare perspective. CONCLUSIONS: Specialized orthotic care was beneficial in attaining personal goals, and likely cost-effective compared to usual orthotic care. CLINICAL REHABILITATION IMPACT: Guideline-based provision of lower limb orthoses in expert settings could enhance functioning of adults with NMD and generate societal cost savings.