Clinical Burden and Healthcare Resource Use Among Adults Living with Hemophilia A: An Observational Study.

IF 2 Q3 PHARMACOLOGY & PHARMACY
Drugs - Real World Outcomes Pub Date : 2025-12-01 Epub Date: 2025-09-01 DOI:10.1007/s40801-025-00516-8
Lisa Young, Lu Ban, Yong Chen, Patrick F Fogarty
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Abstract

Introduction: People with hemophilia A (PwHA) may suffer from repeated bleeding in muscles and joints. However, few studies have reported disease burden and healthcare resource utilization (HCRU) in PwHA experiencing bleeding events and/or joint disorders in a real-world setting. The aim of this study was to examine disease burden and HCRU in PwHA via two different categorizations: PwHA with and without medically recorded bleeding (MRB), and PwHA with and without joint disorders.

Methods: This was an observational cross-sectional study of adult male PwHA treated with factor VIII replacement therapy, identified from PharMetrics Plus® claims data between 2010 and 2019. Comorbidities prevalence and HCRU rates were described. Rate ratios (RRs) of HCRU between PwHA with and without MRB, and PwHA with and without joint disorders were estimated using multivariate adjusted Poisson or negative binomial regressions.

Results: There were 1961 PwHA identified. Of those, 1045 had MRB and 352 had arthropathy. PwHA with MRB showed a higher prevalence of comorbidities and HCRU compared with those without MRB, including inpatient admissions, emergency department visits, outpatient visits, factor VIII replacement therapy, and pain relief medications use. PwHA with MRB also had high bleeding-related HCRU; bleeding-related events accounted for 46% of inpatient hospitalizations. Similarly, PwHA with joint disorders had a higher comorbidity burden and HCRU than those without joint disorders.

Conclusions: PwHA with concurrent bleeding/joint disorders had higher clinical burden compared with those without MRB or joint disorders. Treatment approaches to reduce bleeding episodes, and consequently joint damage, in PwHA may reduce clinical burden.

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A型血友病成人患者的临床负担和医疗资源使用:一项观察性研究
血友病A (PwHA)患者可能会出现肌肉和关节反复出血。然而,很少有研究报道在现实世界中经历出血事件和/或关节疾病的PwHA的疾病负担和医疗资源利用(HCRU)。本研究的目的是通过两种不同的分类来检查PwHA的疾病负担和HCRU: PwHA伴有和不伴有医学记录的出血(MRB), PwHA伴有和不伴有关节疾病。方法:这是一项观察性横断面研究,研究对象是接受因子VIII替代疗法治疗的成年男性PwHA,研究数据来自2010年至2019年的PharMetrics Plus®索赔数据。描述了合并症患病率和HCRU率。采用多变量校正泊松或负二项回归估计患有和不患有MRB的PwHA以及患有和不患有关节疾病的PwHA之间HCRU的比率比(rr)。结果:共检出PwHA 1961例。其中,1045人患有MRB, 352人患有关节病。与没有MRB的患者相比,合并MRB的PwHA显示出更高的合并症和HCRU患病率,包括住院、急诊科就诊、门诊就诊、因子VIII替代治疗和止痛药使用。合并MRB的PwHA也有较高的出血相关HCRU;出血相关事件占住院患者的46%。同样,伴有关节疾病的PwHA患者的合并症负担和HCRU高于无关节疾病患者。结论:合并出血/关节疾病的PwHA患者比无MRB或关节疾病的患者有更高的临床负担。治疗方法减少出血发作,从而减少关节损伤,在PwHA可能减轻临床负担。
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来源期刊
Drugs - Real World Outcomes
Drugs - Real World Outcomes PHARMACOLOGY & PHARMACY-
CiteScore
3.60
自引率
5.00%
发文量
49
审稿时长
8 weeks
期刊介绍: Drugs - Real World Outcomes targets original research and definitive reviews regarding the use of real-world data to evaluate health outcomes and inform healthcare decision-making on drugs, devices and other interventions in clinical practice. The journal includes, but is not limited to, the following research areas: Using registries/databases/health records and other non-selected observational datasets to investigate: drug use and treatment outcomes prescription patterns drug safety signals adherence to treatment guidelines benefit : risk profiles comparative effectiveness economic analyses including cost-of-illness Data-driven research methodologies, including the capture, curation, search, sharing, analysis and interpretation of ‘big data’ Techniques and approaches to optimise real-world modelling.
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