Effectiveness of PAH-specific treatment is comparable in PAH-CTD and IPAH: data from the Polish National Registry (BNP-PL)

Abstract Purpose Pulmonary arterial hypertension (PAH) is a rare disease with poor prognosis. The same treatment is recommended in patients with PAH associated with connective tissue disease (CTD) and idiopathic PAH (IPAH). However, the effectiveness of PAH-specific treatment in these both groups is inconsistent. Methods The study incorporated data from the prospective, multicentre polish registry (BNP-PL) by comparison of baseline vs. 1-year follow-up clinical, laboratory, echocardiographic and hemodynamic parameters in PAH-CTD vs. IPAH group. The all-cause 1-year mortality was also analyzed. Results 624 patients were included in the study: 176 (28%) PAH-CTD and 448 (72%) IPAH. 48 (8% of the total 624) patients died during 1-year follow up: 32 (7%) with IPAH and 16 (9%) with PAH-CTD (p = 0.4). The effectiveness of treatment was examined in 421 patients: 115 (27%) PAH-CTD and 306 (73%) IPAH. The improvement of treatment was effective in both groups in terms of WHO FC (p < 0.001 in both groups), 4-strata risk model (p < 0.001 in both groups), six minute walking test (6MWT, p < 0.001 in IPAH, p = 0.001 in PAH-CTD), Δ6MWT (p = 0.19 between PAH-CTD and IPAH), tricuspid annular plane systolic excursion (p < 0.001 in both groups), NT- proBNP (p < 0.001 in IPAH and p < 0.01 in PAH-CTD), mean pulmonary arterial pressure (p = 0.043 in IPAH and p = 0.01 in PAH-CTD), pulmonary vascular resistance (p < 0.001 in both groups) and cardiac output (p < 0.001 in IPAH and p = 0.034 in PAH-CTD). Conclusions PAH specific treatment in PAH-CTD patients is as effective as in IPAH patients during 1-year observation. 1-year all-cause mortality was not significantly higher in PAH-CTD patients compared to IPAH patients.