Resource utilization and treatment costs of patients with severe hemophilia A: Real-world data from the ATHNdataset.

Hemophilia A is characterized by unpredictable spontaneous bleeds and chronic comorbidities. However, limited data exists at the national level into detailed management patterns related to patient clinical characteristics, representative real-world dosing and treatment frequency, and costs. To assess and characterize the US severe hemophilia A (SHA) population, including subgroups of patients, in terms of clinical and demographic characteristics, healthcare resource utilization received at hemophilia treatment centers (HTCs), and projected annual costs of treatment utilizing data from the ATHNdataset of the American Thrombosis and Hemostasis Network (ATHN). Adult male people with SHA (PwSHA) (FVIII < 1%) were identified in the ATHNdataset between January 2013 and September 2019. This retrospective cohort study described patients' demographic and clinical characteristics, clinical history, as well as the HTC-related health resource utilization (HRU), treatment utilization, and projected annual treatment costs of US PwSHA received over the most recent year. Results are reported for the overall population and for three mutually exclusive subpopulations of patients: PwSHA with a history of and/or current inhibitors, PwSHA without a history of inhibitors but with (or a history of) one or more transfusion-transmitted infections (hepatitis B virus [HBV], hepatitis C virus [HCV], or human immunodeficiency virus [HIV]), and PwSHA without a history of inhibitors or of transfusion-transmitted infections (HBV, HCV, or HIV). Of the overall PwSHA cohort (

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© 2022 The Authors. eJHaem published by British Society for Haematology and John Wiley & Sons Ltd.

Déclaration de conflit d'intérêts

This study was funded by BioMarin. Hinds, Chen, and Solari are or were employees of BioMarin and own stock/stock options at the time of the analysis. Solari is currently an employee of Spark Therapeutics. Cheng, He, and Recht are, or were at the time of this study, employees of American Thrombosis and Hemostasis Network (ATHN), which has received ATHNdataset licensing and other fees from BioMarin.  Research funding to Dr. Recht's employers has come from Bayer, BioMarin, CSL Behring, Genentech, Grifols, Hema Biologics, LFB, Novo Nordisk, Octapharma, Pfizer, Sanofi, Spark, Takeda, and uniQure.  Dr. Recht has worked as a consultant for Catalyst Biosciences, CSL Behring, Genentech, Hema Biologics, Kedrion, Novo Nordisk, Pfizer, Sanofi, Takeda, and uniQure.  Dr. Recht sits on the board of directors of the Foundation for Women and Girls with Blood Disorders and Partners in Bleeding Disorders.  Dr. Recht is an employee of the American Thrombosis and Hemostasis Network and Oregon Health and Science University.