Current therapy and outcome of Eisenmenger syndrome: data of the German National Register for congenital heart defects

Abstract

Aims: We aimed to assess the contemporary outcome of Eisenmenger syndrome (ES), delineate the use of disease targeting therapies (DTT) in these patients and to investigate the effect of treatment on outcome in the community.

Methods and results: Patients with ES were systematically identified from the German National Register for Congenital Heart Defects. Data on underlying diagnosis, medical therapy, and survival were collected. The impact of DTT on survival was assessed using time-dependant Cox analysis. Overall, 153 ES patients were included (mean age 34.0 ± 13.3 years, 46% females). Of these, 88 (57.5%) were treated with at least one DTT (76.1% Bosentan, 20.5% Sildenafil) while 17.6% were on dual DTT. In addition, 24.8% of patients received digoxin, 10.5% angiotensin-converting enzyme-inhibitors/angiotensin receptor blockers, and 17.6% β-blockers. Moreover, 17.6% of patients were treated with oral anticoagulants, while 23.5% of patients received Aspirin. The survival rate at 1, 5, and 10 years of follow-up was only 92, 75, and 57% in the entire cohort, and was even worse in treatment naive ES patients (survival rate 86, 60, and 34% at 1, 5, and 10 years). Use of DTT was independently associated with a better survival (hazard ratio 0.42, P= 0.015).

Conclusion: This study illustrates the alarmingly poor survival prospects of Eisenmenger patients by community-based data even in the current era with advanced DTT and in a country with a wealthy health system. Treatment naive ES patients had especially high mortality rates approaching 60-70% at 10 years of follow-up. Treatment with DTT was associated with better survival.

Keywords: Adult congenital heart disease; Advanced therapy; Eisenmenger syndrome; Registry.

Figure 1

Distribution of therapy. The Figure illustrates that overall 57.5% of patients were treated with at least one disease targeting therapy drug. Of those patients treated with disease targeting therapies, over 95% of patients were on Bosentan or Sildenafil.

Figure 2

Kaplan–Meier survival function of all Eisenmenger patients included in the current study. (A) Time scale based on follow-up time in the National Register. (B) Time scale based on patients' age.

Figure 3

Survival of treatment naïve patients compared with those treated with at least one disease targeting drug modelled based on the results of time-dependant Cox analysis.

Figure 4

Survival of patients on disease targeting monotherapy compared with those treated with dual therapy modelled based on the results of time-dependant Cox analysis.