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Costs and utilization of hemophilia A and B patients with and without inhibitors
Edward P Armstrong1, Daniel C Malone, Sangeeta Krishnan
1Strategic Therapeutics, Tucson, AZ, USA, and College of Pharmacy, University of Arizona , Tucson, AZ , USA.
Insights
Patients with hemophilia and inhibitors incur significantly higher healthcare costs, with annual expenses three times greater than those without inhibitors. Hemophilia B patients generally face lower costs and less severe disease compared to hemophilia A patients.
Area of Science:
- Hematology
- Health Economics
- Pharmacoeconomics
Background:
- Hemophilia A and B are inherited bleeding disorders.
- Inhibitors complicate treatment and increase healthcare utilization.
- Understanding cost drivers is crucial for resource allocation.
Purpose of the Study:
- To evaluate and compare health system costs over five years for hemophilia A and B patients with and without inhibitors.
- To identify factors influencing cost differences in hemophilia care.
Main Methods:
- Retrospective observational study using US electronic health records and claims data (2006-2011).
- Identified hemophilia A and B patients, classifying inhibitor presence by bypassing agent use.
- Categorized disease severity (mild, moderate, severe) based on clotting factor levels.
Main Results:
- Seven patients (five with hemophilia A, two with hemophilia B) had inhibitors.
- Patients with inhibitors had higher hospitalization rates (100%) compared to those without (41.3% for hemophilia A, 42.3% for hemophilia B).
- Median annual costs were $325,780 for patients with inhibitors, $98,334 for hemophilia A without inhibitors, and $23,265 for hemophilia B without inhibitors.
Conclusions:
- Inhibitors, though infrequent, significantly increase hospitalizations and annual costs (3-fold higher) in hemophilia patients.
- Hemophilia B patients generally present with less severe disease and lower annual costs than hemophilia A patients or those with inhibitors.
Objective:
To evaluate the health system costs among patients with hemophilia A and B with and without inhibitors over 5 years.
Methods:
This was a retrospective, observational study utilizing medical and pharmacy electronic medical records and administrative encounters/claims data tracking US patients between 2006-2011. Patients with diagnosis codes for hemophilia A and B were identified. Patients with inhibitors were characterized by utilization of bypassing agents activated prothrombin complex concentrate or factor VIIa on two or more distinct dates. Severity was classified as mild, moderate, or severe based on laboratory tests of clotting factor.
Results:
There were 160 hemophilia A patients and 54 hemophilia B patients identified. From this group, seven were designated as patients with inhibitors (five with hemophilia A and two with hemophilia B). Hemophilia A patients without inhibitors reported 65 (41.9%) as being severe, 19 (12.3%) as moderate, and 71 (45.8%) as mild. Hemophilia B patients without inhibitors reported nine (17.3%) had severe, 13 (25.0%) had moderate, and 30 (57.7%) had mild hemophilia. All patients with inhibitors had been hospitalized in the previous 5 years compared to 64 (41.3%) with hemophilia A without inhibitors and 22 (42.3%) with hemophilia B without inhibitors. The median aggregate cost per year (including factor and health resource use) was $325,780 for patients with inhibitors compared to $98,334 for hemophilia A patients without inhibitors and $23,265 for hemophilia B patients without inhibitors.
Conclusions:
The results suggest that, while the frequency of inhibitors within the hemophilia cohort was low, there was a higher frequency of hospitalizations, and the associated median aggregate costs per year were 3-fold higher than those patients without inhibitors. In contrast, hemophilia B patients experience less severe disease and account for lower aggregate yearly costs compared to either patients with hemophilia A or patients with inhibitors.
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