BACKGROUND: Pulmonary arterial hypertension (PAH) is common in congenital heart disease (CHD). Because clinical-trial data on PAH associated with CHD (PAH-CHD) remain limited, registry data on the long-term course are essential. This analysis aimed to update information from the COMPERA-CHD registry on management strategies based on real-world data. METHODS: The prospective international pulmonary hypertension registry COMPERA has since 2007 enrolled more than 10,000 patients. COMPERA-CHD is a sub-registry for patients with PAH-CHD. RESULTS: A total of 769 patients with PAH-CHD from 62 specialized centers in 12 countries were included into COMPERA-CHD from January 2007 through September 2020. At the last follow-up in 09/2020, patients [mean age 45.3±16.8 years; 512 (66%) female] had either post-tricuspid shunts (n=359; 46.7%), pre-tricuspid shunts (n=249; 32.4%), complex CHD (n=132; 17.2%), congenital left heart or aortic valve or aortic disease (n=9; 1.3%), or miscellaneous CHD (n=20; 2.6%). The mean 6-minute walking distance was 369±121 m, and 28.2%, 56.0%, and 3.8% were in WHO functional class I/II, III or IV, respectively (12.0% unknown). Compared with the previously published COMPERA-CHD data, after 21 months of follow-up, the number of included PAH-CHD patients increased by 91 (13.4%). Within this group the number of Eisenmenger patients rose by 39 (16.3%), the number of "Non-Eisenmenger PAH" patients by 45 (26.9%). Currently, among the 674 patients from the PAH-CHD group with at least one follow-up, 450 (66.8%) received endothelin receptor antagonists (ERA), 416 (61.7%) PDE-5 inhibitors, 85 (12.6%) prostacyclin analogues, and 36 (5.3%) the sGC stimulator riociguat. While at first inclusion in the COMPERA-CHD registry, treatment was predominantly monotherapy (69.3%), this has shifted to favoring combination therapy in the current group (53%). For the first time, the nature, frequency, and treatment of significant comorbidities requiring supportive care and medication are described. CONCLUSIONS: Analyzing "real life data" from the international COMPERA-CHD registry, we present a comprehensive overview about current management modalities and treatment concepts in PAH-CHD. There was an trend towards more aggressive treatment strategies and combination therapies. In the future, particular attention must be directed to the "Non-Eisenmenger PAH" group and to patients with complex CHD, including Fontan patients. TRIAL REGISTRATION: www.clinicaltrials.gov, study identifier: Clinicaltrials.gov NCT01347216.
- Kaemmerer, A. S.
- Gorenflo, M.
- Huscher, D.
- Pittrow, D.
- Ewert, P.
- Pausch, C.
- Delcroix, M.
- Ghofrani, H. A.
- Hoeper, M. M.
- Kozlik-Feldmann, R.
- Skride, A.
- Stähler, G.
- Vizza, C. D.
- Jureviciene, E.
- Jancauskaite, D.
- Gumbiene, L.
- Ewert, R.
- Dähnert, I.
- Held, M.
- Halank, M.
- Skowasch, D.
- Klose, H.
- Wilkens, H.
- Milger, K.
- Jux, C.
- Koestenberger, M.
- Scelsi, L.
- Brunnemer, E.
- Hofbeck, M.
- Ulrich, S.
- Vonk Noordegraaf, A.
- Lange, T. J.
- Bruch, L.
- Konstantinides, S.
- Claussen, M.
- Löffler-Ragg, J.
- Wirtz, H.
- Apitz, C.
- Neidenbach, R.
- Freilinger, S.
- Nemes, A.
- Opitz, C.
- Grünig, E.
- Rosenkranz, S.
Keywords
- Congenital heart disease (CHD)
- Eisenmenger syndrome
- pulmonary hypertension
- registry
- targeted treatment
