Abstract：
To describe people with hemophilia B (PWHB) in the US who experience bleeds despite factor replacement therapy and to quantify the associated burden from the third-party payer perspective.
Observational study of adult male PWHB treated with factor IX replacement therapy identified from the PharMetrics Plus claims data from 2010 to 2019.
Patients with medically recorded bleeds (MRBs) were identified using diagnostic codes. Rates and rate ratios of inpatient admissions, emergency department (ED) visits, and outpatient visits among PWHB with and without MRBs were estimated. The presence of comorbidities was identified using diagnostic codes, and the analysis was stratified by age group.
There were 345 PWHB with MRBs and 252 without MRBs. More than half of PWHB with MRBs (56.8%) had 1 or more comorbidity vs 39.3% of PWHB without MRBs. The prevalence of anxiety and depression was high in PWHB, regardless of bleed status and age group, whereas the prevalence of other comorbidities increased with age group. The rate of all-cause inpatient admissions for PWHB with MRBs was 14.8 per 100 person-years (95% CI, 12.8-17.1), 2.5 times higher than for PWHB without MRBs. The rate of all-cause ED visits for PWHB with MRBs was 67.6 per 100 person-years (95% CI, 63.2-72.3), 2.7 times higher than for those without MRBs.
This study reports significant resource use and clinical burden among PWHB who seek medical care. PWHB with MRBs had considerable all-cause resource use compared with PWHB without MRBs. The prevalence of mental illness was consistently high across all age groups.
Observational study of adult male PWHB treated with factor IX replacement therapy identified from the PharMetrics Plus claims data from 2010 to 2019.
Patients with medically recorded bleeds (MRBs) were identified using diagnostic codes. Rates and rate ratios of inpatient admissions, emergency department (ED) visits, and outpatient visits among PWHB with and without MRBs were estimated. The presence of comorbidities was identified using diagnostic codes, and the analysis was stratified by age group.
There were 345 PWHB with MRBs and 252 without MRBs. More than half of PWHB with MRBs (56.8%) had 1 or more comorbidity vs 39.3% of PWHB without MRBs. The prevalence of anxiety and depression was high in PWHB, regardless of bleed status and age group, whereas the prevalence of other comorbidities increased with age group. The rate of all-cause inpatient admissions for PWHB with MRBs was 14.8 per 100 person-years (95% CI, 12.8-17.1), 2.5 times higher than for PWHB without MRBs. The rate of all-cause ED visits for PWHB with MRBs was 67.6 per 100 person-years (95% CI, 63.2-72.3), 2.7 times higher than for those without MRBs.
This study reports significant resource use and clinical burden among PWHB who seek medical care. PWHB with MRBs had considerable all-cause resource use compared with PWHB without MRBs. The prevalence of mental illness was consistently high across all age groups.
摘要：
目的：描述美国B型血友病（PWHB）患者尽管接受因素替代治疗，但仍出现出血，并从第三方支付者的角度量化相关负担。
方法：从2010年至2019年的PharMetricsPlus索赔数据中确定的接受IX因子替代疗法治疗的成年男性PWHB的观察性研究。
方法：使用诊断代码对有医学记录的出血（MRB）的患者进行鉴定。住院率和比率，急诊科（ED）访问，估计了有和没有MRB的PWHB的门诊量。使用诊断代码识别合并症的存在，分析按年龄组分层。
结果：有345个带有MRB的PWHB和252个没有MRB的PWHB。超过一半的具有MRB的PWHB（56.8％）具有1种或更多种合并症，而没有MRB的PWHB为39.3％。PWHB的焦虑和抑郁患病率较高，不管出血状况和年龄组，而其他合并症的患病率随年龄组增加。PWHB合并MRB的全因住院率为14.8/100人年（95％CI，12.8-17.1），比没有MRB的PWHB高2.5倍。对于具有MRB的PWHB的全因ED访视率为每100人年67.6（95％CI，63.2-72.3），比没有MRB的高2.7倍。
结论：本研究报告了寻求医疗护理的PWHB的显著资源使用和临床负担。与没有MRB的PWHB相比，具有MRB的PWHB具有相当大的全因资源使用。在所有年龄组中，精神疾病的患病率一直很高。
方法：从2010年至2019年的PharMetricsPlus索赔数据中确定的接受IX因子替代疗法治疗的成年男性PWHB的观察性研究。
方法：使用诊断代码对有医学记录的出血（MRB）的患者进行鉴定。住院率和比率，急诊科（ED）访问，估计了有和没有MRB的PWHB的门诊量。使用诊断代码识别合并症的存在，分析按年龄组分层。
结果：有345个带有MRB的PWHB和252个没有MRB的PWHB。超过一半的具有MRB的PWHB（56.8％）具有1种或更多种合并症，而没有MRB的PWHB为39.3％。PWHB的焦虑和抑郁患病率较高，不管出血状况和年龄组，而其他合并症的患病率随年龄组增加。PWHB合并MRB的全因住院率为14.8/100人年（95％CI，12.8-17.1），比没有MRB的PWHB高2.5倍。对于具有MRB的PWHB的全因ED访视率为每100人年67.6（95％CI，63.2-72.3），比没有MRB的高2.7倍。
结论：本研究报告了寻求医疗护理的PWHB的显著资源使用和临床负担。与没有MRB的PWHB相比，具有MRB的PWHB具有相当大的全因资源使用。在所有年龄组中，精神疾病的患病率一直很高。
