Discussion on the relationship between the distribution characteristics of TCM syndrome types and related objective indicators in hepatolenticular degeneration

Abstract

Hepatolenticular degeneration is a rare treatable autosomal recessive inherited copper metabolism disorder with a diverse clinical phenotype and an exceptionally complex pathogenesis. Early definitive phenotypic diagnosis and targeted treatment are major challenges worldwide. In this study, we strictly followed the "National Standards of the People's Republic of China - Terminology of Traditional Chinese Medicine Clinical Diagnosis and Treatment (Syndrome Part)," "Chinese Medicine Nomenclature," and the clinical investigation-determined traditional Chinese medicine syndrome differentiation standards at Anhui University of Chinese Medicine to select 6 of the most common traditional Chinese medicine syndrome differentiations. This study retrospectively analyzed 107 patients admitted between 2019 and 2023 with Wilson's disease based on real-world data. After testing for normal distribution and homogeneity of variance, corresponding analysis of variance was selected, followed by post hoc multiple comparisons. Of the selected 25 objective influencing factors, 22 exhibited normal distribution, while red blood cells, hemoglobin, and type IV collagen did not pass the homogeneity of variance test. After analysis of variance, the factors ceruloplasmin (CP) and copper oxidase (SCO) showed significant differences among patients with different traditional Chinese medicine syndromes (P < .05), with partial η2 for CP being 0.13 > 0.06 and for SCO being 0.143 > 0.14. Post hoc multiple comparison results indicated significant differences in CP and SCO among patients with certain traditional Chinese medicine syndromes (P < .05). There were significant differences in the factors CP and SCO among patients with different traditional Chinese medicine syndromes. Significant differences were observed in the copper blue protein factor between damp-heat syndrome and liver and kidney deficiency syndrome, liver and kidney deficiency syndrome and liver and kidney yin deficiency syndrome, liver and kidney deficiency syndrome and phlegm heat and wind syndrome, as well as liver and kidney deficiency syndrome and syndrome of phlegm and blood stasis (P < .05). Significant differences were also found in the SCO factor between damp-heat syndrome and liver and kidney deficiency syndrome, liver and kidney deficiency syndrome and liver and kidney yin deficiency syndrome, liver and kidney deficiency syndrome and phlegm heat and wind syndrome, and liver and kidney deficiency syndrome and syndrome of phlegm and blood stasis (P < .05).