Costs and Hospitalizations Associated with Hemophilia

San Antonio—Patients with hemophilia who develop inhibitors had a lower frequency of hospitalizations and had treatment costs 3 times lower than people who did not develop inhibitors, according to a retrospective, observational study.

The authors found that hemophilia B patients had less severe disease and lower costs than hemophilia A patients or patients with inhibitors. Results were presented at the NASP meeting in a poster presentation. The poster was titled Costs and Utilization of Hemophilia A and B Patients with and without Inhibitors.

The authors noted that hemophilia, a congenital bleeding disorder, results from clotting factor deficiency and often requires patients to use clotting factor replacement therapy to deal with bleeding complications and joint damage. When patients develop inhibitors to the replacement clotting factors, they receive alternative treatments such as immune tolerance induction and bypass agents, which increases costs.

In this study, the authors examined electronic medical records and administrative claims data of patients in the United States between 2006 and 2011. They used the US Department of Defense database for military servicemen and servicewomen and their families. Patients were eligible if they had an International Classification of Diseases, 9th Revision code for hemophilia A or B and received clotting factors. They were excluded if they had von Willebrand disease.

In all, 214 people met the inclusion criteria, including 7 with inhibitors: 5 with hemophilia A and 2 with hemophilia B. Of the patients, 74.8% had hemophilia A and 25.2% had hemophilia B. The authors characterized patients in the inhibitors group if they took activated prothrombin complex or factor VIIa on 2 or more dates. They classified mild hemophilia as greater than 5% to 40% normal factor activity, moderate hemophilia as 1% to 5% normal factor activity, and severe hemophilia as <1% normal factor activity.

Of the hemophilia A patients without inhibitors, 41.9% had severe disease, 12.3% had moderate disease, and 45.8% had mild disease. Of the hemophilia B patients without inhibitors, 17.3% had severe disease, 25% had moderate disease, and 57.7% had mild disease.

The median age was 2 years for patients with inhibitors, 6 years for hemophilia A patients without inhibitors, and 8.5 years for hemophilia B patients without inhibitors. Patients with inhibitors had a median of 1.4 bleeding events per year compared with 0.3 for mild, 0.4 for moderate, and 0.2 for severe noninhibitor patients.

Each patient with inhibitors was hospitalized in the previous 5 years, compared with fewer than 50% of patients without inhibitors. The median aggregate cost per year was more than $300,000 for patients with inhibitors, approximately $100,000 for hemophilia A patients without inhibitors, and less than $50,000 for hemophilia B patients without inhibitors.

The authors mentioned some limitations to their analysis, including that patients with inhibitors were children, so the results may not be relevant for an adult population. Further, the database only included claims from a hospital, outpatient, or pharmacy setting. The analysis did not include bleeding treated in people’s homes.

Biogen Idec funded this study.