192P A community-based experience with the new 12-Tier functional ability scale for Evolving Spinal Muscular Atrophy (EVOLVE-SMA).

With the development of SMN-specific treatments, the conventional "Type" classification of SMA, based upon the untreated natural history of progressive degeneration, is now anachronistic. This scale, the Functional Ability Scale for Evolving SMA (EVOLVE-SMA), addresses the need for a broader classification of SMA, offering potential benefits for enhanced therapy assessment, refined clinical trial criteria, biomarker development, and population-based therapy impact assessment. Participants with SMA, greater than 2 years of age were recruited at a community-based event to complete a survey. Following participant self-assignment, a clinician team provided a blinded participant classification. One hundred sixty-eight participants with SMA or their caregivers (57.7% self-report; 42.3% caregiver report) completed the survey. Using a 10-point Likert scale, prospective participants reported favorable likability (mean 7.1) and that the scale represented their functional ability (mean 6.9). Eleven clinician team members (physical therapists and physicians) provided letter classifications following the participant completing the survey with excellent reliability (ICC 0.995, p<0.001). Reliability established among raters and participants' self-assignment demonstrated excellent overall agreement (ICC 0.924, p<.001). The average time for participants to complete the survey and clinicians to select a classification was 10 minutes showing the feasibility. We demonstrate the feasibility and reliability of the EVOLVE-SMA scale. EVOLVE-SMA is sufficiently granular to assess small differences, can be reliably administered by either patients or providers, and is meaningful to the patient experience, and broadly applicable across the full range of residual functional impairment that patients manifest. EVOLVE-SMA holds promise as a new outcome of value for future research and resource allocation, and it assists individual care by replacing the existing "type" system of SMA nosology. [ABSTRACT FROM AUTHOR]