Development and validation of the modified-comprehensive Kumamoto Score: a multi-organ assessment tool for hereditary transthyretin amyloidosis - PubMed
4 hours ago
- #multi-organ assessment
- #hereditary transthyretin amyloidosis
- #clinical validation
- Development and validation of the modified-comprehensive Kumamoto Score (mKS) for hereditary transthyretin amyloidosis, a multi-organ assessment tool.
- The mKS includes four organ-specific subscales: peripheral neuropathy (0-258), cardiomyopathy (0-195), eye (0-60), and central nervous system involvement (0-45), with a total range of 0-558.
- Prospective validation with 76 patients showed strong correlations with existing clinical scores, biomarkers, and quality of life measures, and weak intercorrelations between subscales confirming independent organ assessment.
- Conclusions highlight the mKS as a validated tool with enhanced early-stage sensitivity for use in the era of disease-modifying therapies.