Epidemiological insights about hemophilia B by analysis of German hospital quality reports

S. Wahler1, A. Müller2 (1Hamburg, Germany, 2München, Germany)

Bleeding disorders, coagulation and fibrinolytic factors
Date: 17.02.2017,
Time: 17:15 - 18:15

Objective: Hemophilia B (HB), also called factor IX (FIX) deficiency or Christmas disease, is a genetic disorder caused by missing or defective FIX, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation. The birth prevalence of HB is around one in 20,000 live male births worldwide. The main medication to treat HB is concentrated FIX products of plasmatic or recombinant origin. Both treatments have a specific procedure code in Germany. Since 2008 the Paul-Ehrlich-Institute (PEI) has published annual reports of overall epidemiology and factor use. We have taken this further by examining the frequency of in-hospital HB cases and in-hospital use of FIX.

Methods: Data from the German DRG Institute (InEK), Statistical Office (DESTATIS) and hospital quality reports for 2000-2014 were analyzed for cases of hemophilia B and treatments with FIX. Reference was taken from the corresponding reports of the PEI. Statistical analysis was performed using Microsoft-Excel and Access version 2013.

Results: The number of hospital cases with a main diagnosis of HB (D67, ICD10-GM) decreased from 109 (2000), 75 (2003), 70 (2008), to 58 (2014, -47%). In contrast, the number of patients increased slightly from 537 in 2008 to 596 in 2014. The mean age of hospitalized patients (30.3 +/- 23.9 years) and the gender distribution (95% male) remained stable over time. The average length of stay did not differ between age groups, but was shorter for male (5.4 days) than for female patients (7.7 days). The number of cases with a secondary diagnosis of HB also decreased over time. In 2014, it was 403 patients with an average age of 42.4 years. The decrease in cases was not associated with a decrease in in-hospital FIX usage; it was stable around 5m units annually or 8.6% of all FIX used in Germany. In contrast to overall use, there was a growing preference in-hospital for use recombinant FIX (rFIX in 2014: 37%) over plasmatic.

Conclusion: The rate of hospitalization of patients with Hemophilia B decreased in the last decade. Treatment shifted to the ambulatory sector. But the use of FIX in hospital remained stable due to a trend for much higher use per case. In addition, the in-hospital share of recombinant FIX is increasing versus plasmatic FIX, which is not found in the ambulatory sector.