Comparison And Influencing Factors Ot Health State Utility Measures In Hemophilia A Patients

BackgroundHemophilia A(HA)is an invisible genetic hemorrhagic disorder with X chromosome linkage manifested by coagulation factor deficiency or synthesis disorder.It accounts for 85%in hemophilia population.The incidence of HA in male population is about 1/5000.The most common clinical manifestation of this disease is joint bleeding.Repeated joint bleeding can lead to joint deformity and even disability.The quality of life of patients are seriously affected.Carrying out effective health utility value is the key and foundation of health economic evaluation and health technology evaluation,which affects health decision-making.At present,there are few studies on the measurement of HA health utilities in China.ObjectivesIn this study,two children and adolescents health utilities scales(EQ-5D-Y,CHU9D)and two adults health utilities scales(EQ-5D-5L,SF-6Dv2)were used to evaluate measurement properties,measure health utility values at different ages and explore the influencing factors of the health utility values in HA patients.Then this study could provide an appropriate reference for selecting health utility scales for HA patients.MethodsIn this study,a combination of online and offline survey was used to investigate HA patients aged 7 years and older from October 2020 to July 2021.The questionnaire included socio-demographic characteristics,disease characteristics information,and health utility scales.The measurement properties were evaluated by reliability,validity,sensitivity and agreement.Reliability was evaluated by Cronbach’α and internal consistency of each dimension.Validity included convergent validity,known-group validity.Sensitivity was evaluated by ceiling effect,floor effect,effect size,relative efficiency,the area under the Receiver Operating Characteristic curve(AUC).Intraclass correlation coefficient and Bland-Altman(B-A)plots were used to compare agreement analysis.ResultsA total of 211 HA patients were investigated.209 patients were included in the analysis.The number of HA patients aged 7～16 years was 71.The average age was 11.26±2.70.The number of HA patients aged 17 years and older was 138.The average age was 35.18±11.15.HA patients were mainly severe(67.0%)and rural household registration(64.6%).Nearly 80%of the adult patients had a high school education or less,and more than half were unemployed(55.8%).Cronbach’s α between EQ-5Q-Y and CHU9D,EQ-5D-5L and SF-6Dv2 were all above 0.7.Most of the instruments showed moderate correlations among dimensions,indicating good consistency of the scales.Both EQ-5D-Y and CHU9D can distinguish differences in the quality of life of patients with different ages,learning status and so on.EQ-5D-5L and SF-6Dv2 can distinguish the differences of quality of life of patients with different household registration types,learning status,and so on.These all indicates that both scales have good discriminative validity.EQ-5D-Y and CHU9D,EQ-5D-5L and SF-6Dv2 have good convergent validity.Exploratory factor analysis showed that EQ-5D-5L and SF-6Dv2 had a large number of common factors and a high degree of consistency.All scales had good sensitivity,but there were differences in sensitivity levels.CHU9D was more sensitive than EQ-5D-Y on the whole.Overall,SF-6Dv2 performed better than EQ-5D-5L in sensitivity.The ICC coefficient between EQ-5D-Y and CHU9D was 0.758,and EQ-5D-5L and SF-6Dv2 was 0.834.B-A plots showed that these scales were still not refundable.Among HA patients aged 7～16 years,the average utility value of EQ-5D-Y was 0.836±0.141.The average utility value of CHU9D was 0.770±0.219.Whether use of prothrombin complex,the number of mild to moderate bleeding within one year were two common factors influencing two health utility scales.Among HA patients aged 17 and above,the average utility value of EQ-5D-5L was 0.439±0.389.The average utility value of SF-6Dv2 was 0.343±0.309.Education,unemployment or losing job,with or without inhibitors and severe bleeding were common influencing factors of the two health scales.ConclusionsFour instruments(EQ-5D-Y and CHU9D,EQ-5D-5L and SF-6Dv2)all had good reliability,validity,sensitivity and agreement.But CHU9D performed better than EQ-5D-Y,SF-6Dv2 performed better than EQ-5D-5L.Therefore,this study recommended CHU9D to measure the quality of life in HA patients aged 7～16 years,and SF-6Dv2 to measure quality of life in HA patients aged 17 years and above.Though the influencing factors of scales were different,and bleeding was the common influencing factor in HA patients.