High Health Care Burden Among People with Hemophilia A Experiencing Bleeding or Joint Complications
Hemophilia A, an X-linked genetic disorder resulting in deficient clotting factor VIII, remains a condition marked by significant morbidity and cost burden despite advances in therapy. People with hemophilia A (PwHA) frequently experience spontaneous bleeding episodes, most often in muscles and joints, which can lead to long-term arthropathy. Factor VIII replacement therapy is the standard of care, yet it requires frequent administration, is costly, and may lose effectiveness in those who develop inhibitors. The introduction of emicizumab has expanded treatment options, but challenges persist in controlling bleeding events and preventing joint damage. Understanding the real-world health care resource utilization (HCRU) associated with bleeding and joint complications is critical for improving patient outcomes and reducing system-wide costs.
This cross-sectional study analyzed US claims data from PharMetrics Plus® between 2010 and 2019, focusing on adult men with hemophilia A who were treated with factor VIII replacement therapy or emicizumab (after its 2018 approval for non-inhibitor patients). A total of 1961 PwHA met inclusion criteria. Patients were categorized into subgroups based on the presence or absence of medically recorded bleeding (MRB) and joint disorders (arthropathy). Health care utilization—including inpatient hospitalizations, emergency department (ED) visits, outpatient encounters, and pharmacy use—was compared across subgroups using multivariate regression models.
More than half of the PwHA (53%) experienced MRB, while 18% had joint disorders. Across both categorizations, those with MRB or joint disease demonstrated significantly greater clinical burden and health care utilization. PwHA with MRB had higher rates of inpatient admissions, ED visits, outpatient visits, and pharmacy dispensing compared with those without MRB. Bleeding-related events were particularly costly: they accounted for nearly half of all inpatient hospitalizations and 40% of ED visits. Similarly, PwHA with joint disorders required more frequent hospitalizations, outpatient visits, and pain-relief medications than those without joint involvement. The study also revealed higher comorbidity prevalence in affected subgroups, particularly anxiety, depression, chronic liver disease, HIV, and hepatitis C. These findings underscore the complex interplay of physical, psychological, and infectious disease comorbidities in this population.
The results highlight a pressing need for treatment strategies that not only control acute bleeding but also reduce cumulative joint damage. Effective prophylaxis has the potential to lessen health care use, improve quality of life, and decrease costs associated with hospitalization and long-term joint complications. The high prevalence of pain medication use and mental health comorbidities further suggests that holistic care approaches—including psychosocial support and pain management—are essential. Moreover, payer and policy stakeholders should note that PwHA with recurrent bleeding and arthropathy contribute disproportionately to overall resource consumption, suggesting that investments in preventive therapies may yield long-term economic benefit.
This real-world analysis confirms that PwHA with bleeding episodes or joint disorders experience substantially greater disease and economic burden than their peers without these complications. By reducing bleeding frequency and preventing joint deterioration, emerging and established treatment strategies may ease both clinical and health care resource burdens. Continued research and investment in therapies targeting prevention are vital to improving patient outcomes and ensuring sustainable care models for hemophilia A.
