Among patients with severe hemophilia A (SHA), both the financial and clinical burden remain high despite widespread efforts to coordinate care through hemophilia treatment centers (HTCs), according to research published in eJHaem.

HA is an X-linked coagulation disorder characterized by factor VIII (FVIII) deficiency. The disorder is primarily diagnosed in male patients, and is linked with bleeding of variable severity.

SHA, however, is diagnosed in up to approximately 66% of patients with HA, and is characterized by particularly low FVIII levels and clinically serious bleeding episodes, which may arise spontaneously or through injury. While FVIII replacement therapy may improve outcomes among some patients, this treatment paradigm is linked with inhibitor resistance, with potentially severe implications for patient health and quality of life.

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The variable presentation and potential responses to therapy have led to differing recommendations for subsets of patients with SHA. Patients with inhibitor antibodies, for example, or those with hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV) require specialized care. For this study, researchers aimed to evaluate patient characteristics, healthcare resource used at HTCs, and annual costs of treatment among individuals with SHA.

All data were obtained from the American Thrombosis and Hemostasis Network (ATHN)’s ATHNdataset. Overall, of 40,630 patients identified in the ATHNdataset, and 3677 with SHA were included. Among these, all were male, 789 were included in the inhibitor subpopulation, and 505, 182, and 885 patients had HIV, HBC, and HCV, respectively.

Analysis showed that patients with SHA had a mean 2.8 visits to an HTC per year, as well as 0.9 comprehensive care visits, 1.1 telephone calls, 0.5 office visits, and 0.1 procedures, including surgery. The data also showed, however, that 23.3% of patients had no HTC visits, and 33.8% had no comprehensive care visit in the 1 year leading up to data collection.

Annual treatment costs varied by treatment strategy, and ranged from $613,220 to $934,301. “This study additionally highlighted the economic burden of current hemophilia treatment, further emphasizing the importance of tailored disease management in SHA,” the authors wrote in their report.

Disclosure: The study author(s) declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of authors’ disclosures. 


Recht M, He C, Chen E, Cheng D, Solari P, Hinds D. Resource utilization and treatment costs of patients with severe hemophilia A: real-world data from the ATHNdataset. EJHaem. 2022;3(2):341-352. doi:10.1002/jha2.412